Thymectomy Trial in Non-Thymomatous Myasthenia Gravis Patients Receiving Prednisone Therapy

• ClinicalTrials.gov Identifier: NCT00294658
• Recruitment Status: Completed
• First Posted: February 22, 2006
• Results First Posted: January 16, 2017
• Last Update Posted: May 23, 2017
• Sponsor: University of Alabama at Birmingham
• Collaborator: National Institute of Neurological Disorders and Stroke (NINDS)
• Information provided by (Responsible Party): Gary Cutter, PhD, University of Alabama at Birmingham

• Study Type: Interventional
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Single (Outcomes Assessor); Primary Purpose: Treatment
• Condition: Myasthenia Gravis
• Interventions: Procedure: thymectomy plus prednisone; Drug: prednisone alone
• Enrollment: 126

Participant Flow

Recruitment Details	The first enrollment of two patients started with July 26, 2006 and the last patient was recruited on November 28, 2012. Of the 126 randomized patients, 96 were from centers outside the US and 30 were from US centers.
Pre-assignment Details	After informed consent signed, 66 patients were randomized to extended transsternal thymectomy with prednisone treatment and 60 patients were assigned to take prednisone without surgery. Within the 126 subject that started, 15 dropped out before 3 year follow-up.

Arm/Group Title	Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description	Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy had been performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen had been taken every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Period Title: Overall Study
Started	66	60
Completed	60	51
Not Completed	6	9
Reason Not Completed
Death	0	1
Lost to Follow-up	4	0
Physician Decision	1	2
Pregnancy	0	1
Withdrawal by Subject	0	4
Found to have invasive thymoma	1	0
Patient and family were not satisfied	0	1

Baseline Characteristics

Arm/Group Title		Thymectomy Plus Prednisone	Prednisone Alone	Total
Arm/Group Description		Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.	Total of all reporting groups
Overall Number of Baseline Participants		66	60	126
Baseline Analysis Population Description		[Not Specified]
Age, Continuous; Median (Full Range); Unit of measure: Years
	Number Analyzed	66 participants	60 participants	126 participants
		32; (18 to 63)	33; (18 to 64)	32; (18 to 64)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	66 participants	60 participants	126 participants
	Female	50 75.8%	39 65.0%	89 70.6%
	Male	16 24.2%	21 35.0%	37 29.4%
Race/Ethnicity, Customized; Measure Type: Number; Unit of measure: Participants	Number Analyzed	66 participants	60 participants	126 participants
Asian		6	4	10
Black		7	6	13
Hispanic		17	17	34
Non-Hispanic		31	30	61
Other		5	3	8
Region of Enrollment; Measure Type: Number; Unit of measure: Participants	Number Analyzed	66 participants	60 participants	126 participants
Argentina		10	11	21
United States		16	14	30
Japan		1	1	2
United Kingdom		2	2	4
Thailand		3	2	5
Canada		9	9	18
Netherlands		1	0	1
Brazil		3	2	5
Poland		1	1	2
Italy		6	4	10
Mexico		1	0	1
South Africa		4	4	8
Australia		2	2	4
Chile		5	5	10
Germany		2	3	5
Therapy at enrollment - Pyridostigimine; Measure Type: Number; Unit of measure: Participants	Number Analyzed	66 participants	60 participants	126 participants
Yes		60	56	116
No		5	1	6
Missing		1	3	4
Therapy at enrollment - Glucocorticoid; Measure Type: Number; Unit of measure: Participants	Number Analyzed	66 participants	60 participants	126 participants
Yes		49	47	96
No		16	10	26
Missing		1	3	4
Previous therapy - Intravenous immue globulin; Measure Type: Number; Unit of measure: Participants	Number Analyzed	66 participants	60 participants	126 participants
Yes		12	13	25
No		53	44	97
Missing		1	3	4
Previous therapy - Plasma exchange; Measure Type: Number; Unit of measure: Participants	Number Analyzed	66 participants	60 participants	126 participants
Yes		9	7	16
No		56	50	106
Missing		1	3	4
MGFA class [1]; Measure Type: Number; Unit of measure: Participants	Number Analyzed	66 participants	60 participants	126 participants
IIa		25	25	50
IIb		18	14	32
III		21	20	41
IV		2	1	3
		[1] Measure Description: Class IIa. Predominantly affecting limb, axial muscles, or both. May also have lesser involvement of oropharyngeal muscles. Class IIb. Predominantly affecting oropharyngeal, respiratory muscles, or both. May also have lesser or equal involvement of limb, axial muscles, or both. Class III. Moderate weakness affecting muscles other than ocular muscles; may also have ocular muscle weakness of any severity. Class IV. Severe weakness affecting muscles other than ocular muscles; may also have ocular muscle weakness of any severity. Higher class was considered worse outcome.
Duration of disease; Median (Full Range); Unit of measure: Years
	Number Analyzed	66 participants	60 participants	126 participants
		1.08; (0.02 to 4.41)	1.14; (0.15 to 4.38)	1.11; (0.02 to 4.41)
QMG score [1]; Mean (Standard Deviation); Unit of measure: Units on a scale
	Number Analyzed	66 participants	60 participants	126 participants
		11.40 (5.12)	12.35 (4.90)	11.84 (5.02)
		[1] Measure Description: Myasthenia Gravis (QMG) test. QMG total scores range from 0 to 39, with higher scores indicating more severe disease.
Prednisone use at baseline; Measure Type: Number; Unit of measure: Participants	Number Analyzed	66 participants	60 participants	126 participants
Yes		49	47	96
No		16	10	26
Missing		1	3	4
Prednisone Dosage at baseline (mg) [1]; Mean (Standard Deviation); Unit of measure: Mg
	Number Analyzed	66 participants	60 participants	126 participants
		43.43 (28.92)	42.49 (23.52)	42.97 (26.28)
		[1] Measure Description: Prednisone dosage unit as mg, patient has been taken prednisone every other day

Outcome Measures

1. Primary Outcome

Title	Time-weighted Average Quantitative Myasthenia Gravis Weakness Score Over 3 Years
Description	Myasthenia Gravis (QMG) test. QMG total scores range from 0 to 39 for a given visit, with higher scores indicating more severe disease. The time weighted average is a calculation that provides an integrated measure of the outcome over the time of followup. The denominator that was used to compute the time-weighted average for the Quantitative Myasthenia Gravis (QMG) score and the prednisone dose was the number of days from randomization to the last visit. Computations used the trapezoidal method where in the QMG score is multiplied by the number of days at this level from one visit to the next and added up over the entire followup experience and divided by the total number of days from randomization.
Time Frame	baseline, month 3, 4, 6 and every 3 months through 36 months

Outcome Measure Data

Analysis Population Description

Four participants in each group did not provide the information to enable calculation of the time-weighted Quantitative Myasthenia Gravis Weakness Score over 3 years.

Arm/Group Title	Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:	Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed	62	56
Mean (Standard Deviation); Unit of Measure: units on a scale
	6.15 (4.09)	8.99 (4.93)

2. Primary Outcome

Title	Time-weighted Average Alternate-day Prednisone Dose (mg) Measured Over 3 Years
Description	Participants reported alternate-day prednisone dose (mg) intake from baseline through withdrawn or completed 3 years follow up. The prednisone dosages had been weighted over the days of reporting period.
Time Frame	baseline, month 1 , 2 , 3, 4, 6 and every 3 months through 36 months

Outcome Measure Data

Analysis Population Description

Five participants in Thymectomy plus prednisone and 4 in Prednisone alone group did not provide the information to enable calculation of the time-weighted average alternate-day prednisone dose (mg) over 3 years.

Arm/Group Title	Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:	Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed	61	56
Mean (Standard Deviation); Unit of Measure: mg
	32 (23)	54 (29)

3. Secondary Outcome

Title	Subgroup Analyses of Time-weighted Average Quantitative Myasthenia Gravis Score by Prednisone Use at Enrollment
Description	Myasthenia Gravis (QMG) test. QMG total scores range from 0 to 39 for a given visit, with higher scores indicating more severe disease.
Time Frame	baseline, month 3, 4, 6 and every 3 months through 36 months

Outcome Measure Data

Analysis Population Description

Four participants in Thymectomy plus prednisone and 5 in Prednisone alone group did not provide information to enable the calculation of Time-weighted average Quantitative Myasthenia Gravis Score by prednisone use at enrollment over 3 years.

Arm/Group Title		Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:		Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed		62	55
Mean (Standard Deviation); Unit of Measure: units on a scale
Not prednisone naïve	Number Analyzed	47 participants	46 participants
		6.30 (3.89)	9.10 (5.06)
Prednisone naïve	Number Analyzed	15 participants	9 participants
		5.66 (4.79)	8.84 (4.60)

4. Secondary Outcome

Title	Subgroup Analyses of Time-weighted Average Quantitative Myasthenia Gravis Score by Sex
Description	Myasthenia Gravis (QMG) test. QMG total scores range from 0 to 39 for a given visit, with higher scores indicating more severe disease.
Time Frame	baseline, month 3, 4, 6 and every 3 months through 36 months

Outcome Measure Data

Analysis Population Description

Four participants in each group did not provide the information to enable calculation of the time-weighted Quantitative Myasthenia Gravis Weakness Score over 3 years.

Arm/Group Title		Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:		Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed		62	56
Mean (Standard Deviation); Unit of Measure: units on a scale
Female	Number Analyzed	46 participants	38 participants
		6.47 (4.13)	9.73 (5.16)
Male	Number Analyzed	16 participants	18 participants
		5.23 (3.95)	7.45 (4.11)

5. Secondary Outcome

Title	Subgroup Analyses of Time-weighted Average Quantitative Myasthenia Gravis Score by Age at Disease Onset
Description	Myasthenia Gravis (QMG) test. QMG total scores range from 0 to 39 for a given visit, with higher scores indicating more severe disease.
Time Frame	baseline, month 3, 4, 6 and every 3 months through 36 months

Outcome Measure Data

Analysis Population Description

Four participants in each group did not provide the information to enable calculation of the time-weighted average Quantitative Myasthenia Gravis Weakness Score over 3 years. Another 2 participants in Thymectomy plus prednisone and 4 in Prednisone alone group did not provide the age at disease onset information.

Arm/Group Title		Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:		Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed		60	52
Mean (Standard Deviation); Unit of Measure: units on a scale
Age (years) at Disease Onset < 40	Number Analyzed	42 participants	34 participants
		6.50 (4.41)	9.60 (5.32)
Age (years) at Disease Onset ≥ 40	Number Analyzed	18 participants	18 participants
		5.33 (2.79)	7.85 (3.50)

6. Secondary Outcome

Title	Subgroup Analyses of Time-weighted Average Alternate-day Prednisone Dose (mg) by Prednisone Use at Enrollment
Description	Participants reported alternate-day prednisone dose (mg) intake from baseline through withdrawn or completed 3 years follow up. The prednisone dosages had been weighted over the days of reporting period.
Time Frame	baseline, month 3, 4, 6 and every 3 months through 36 months

Outcome Measure Data

Analysis Population Description

Five participants in Thymectomy plus prednisone and 4 in Prednisone alone group did not provide the information to enable calculation of time-weighted average alternate-day prednisone dose (mg) over 3 years. Another 1 in Prednisone alone group did not provide prednisone use at enrollment information.

Arm/Group Title		Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:		Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed		61	55
Mean (Standard Deviation); Unit of Measure: mg
Not prednisone naïve	Number Analyzed	46 participants	46 participants
		35 (25)	56 (31)
Prednisone naïve	Number Analyzed	15 participants	9 participants
		25 (17)	45 (22)

7. Secondary Outcome

Title	Subgroup Analyses of Time-weighted Average Alternate-day Prednisone Dose (mg) by Sex
Description	Participants reported alternate-day prednisone dose (mg) intake from baseline through withdrawn or completed 3 years follow up. The prednisone dosages had been weighted over the days of reporting period.
Time Frame	baseline, month 3, 4, 6 and every 3 months through 36 months

Outcome Measure Data

Analysis Population Description

Five participants in Thymectomy plus prednisone and 4 in Prednisone alone group did not provide the information to enable calculation of the time-weighted average alternate-day prednisone dose (mg) over 3 years.

Arm/Group Title		Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:		Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed		61	56
Mean (Standard Deviation); Unit of Measure: mg
Female	Number Analyzed	45 participants	37 participants
		33 (25)	54 (27)
Male	Number Analyzed	16 participants	19 participants
		31 (18)	55 (34)

8. Secondary Outcome

Title	Subgroup Analyses of Time-weighted Average Average Alternate-day Prednisone Dose (mg) by Age at Disease Onset
Description	Participants reported alternate-day prednisone dose (mg) intake from baseline through withdrawn or completed 3 years follow up. The prednisone dosages had been weighted over the days of reporting period.
Time Frame	baseline, month 3, 4, 6 and every 3 months through 36 months

Outcome Measure Data

Analysis Population Description

Five participants in Thymectomy plus prednisone and 4 in Prednisone alone group did not provide the information to enable calculation of the time-weighted average alternate-day prednisone dose (mg) over 3 years. Another 2 in Thymectomy plus prednisone group and 4 in Prednisone alone group did not provide age at disease onset information.

Arm/Group Title		Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:		Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed		59	52
Mean (Standard Deviation); Unit of Measure: mg
Age (Years) at Disease Onset < 40	Number Analyzed	41 participants	33 participants
		35 (25)	55 (30)
Age (Years) at Disease Onset ≥ 40	Number Analyzed	18 participants	19 participants
		27 (18)	49 (29)

9. Secondary Outcome

Title	Number of Serious Adverse Events
Description	Number of participant who experienced at least one serious adverse events over 3 years: Thymectomy plus prednisone n=25 (out of 66); Prednisone alone n=33 (out of 60)
Time Frame	baseline to 3 years

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:	Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed	25	33
Measure Type: Number; Unit of Measure: events
	48	93

10. Secondary Outcome

Title	Number of Patients With at Least One Serious Adverse Events
Description	Number of participant who experienced at least one serious adverse events over 3 years: Thymectomy plus prednisone n=25 (out of 66); Prednisone alone n=33 (out of 60)
Time Frame	baseline to 3 years

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:	Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed	66	60
Measure Type: Number; Unit of Measure: participants
	25	33

11. Secondary Outcome

Title	Classification of Serious Adverse Events
Description	[Not Specified]
Time Frame	baseline to 3 years

Outcome Measure Data

Analysis Population Description

One participant might had experienced more than one serious adverse event.

Arm/Group Title	Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:	Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed	25	33
Measure Type: Number; Unit of Measure: participants
Life-threatening	1	7
Disability or incapacity	8	2
Medical or surgical intervention	9	5
Death	0	1
Complication due to thytmectomy	1	0
Hospitalization	15	31

12. Secondary Outcome

Title	Hospitalization for Exacerbation of Myasthenia Gravis
Description	[Not Specified]
Time Frame	baseline to 2 years and baseline to 3 years

Outcome Measure Data

Analysis Population Description

Number of participants who had hospitalized over 2 and 3 years

Arm/Group Title	Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:	Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed	15	31
Measure Type: Number; Unit of Measure: participants
Months 0-24	6	17
Months 0-36	6	22

13. Secondary Outcome

Title	Cumulative Number of Hospital Days
Description	Number who had hospitalization: Thymectomy plus prednisone n=15 (out of 66); Prednisone alone n=31 (out of 60)
Time Frame	baseline to 3 years

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:	Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed	15	31
Mean (Standard Deviation); Unit of Measure: days
	8.4 (8.6)	19.2 (24.5)

14. Secondary Outcome

Title	Reason for Hospitalization According to Medical Dictionary for Regulatory Activities Term
Description	Number who had hospitalization: Thymectomy plus prednisone n=15 (out of 66); Prednisone alone n=31 (out of 60)
Time Frame	baseline to 3 years

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:	Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed	15	31
Measure Type: Number; Unit of Measure: events
Gastrointestinal disorder	2	2
Hepatobiliary disorder	0	1
Infection or infestation	4	7
Injury, poisoning, or procedure complication	2	0
Metabolism or nutrition disorder	1	0
Nervous system disorder	8	22
Respiratory, thoracic, or mediastinal disorder	1	2
Surgical or medical procedure	0	7
Vascular disorder	0	1

15. Secondary Outcome

Title	Time-weighted Average Prescribed Alternate Day Prednisone Dose (mg)
Description	Physicians reported prescribed alternate-day prednisone dose (mg) intake from baseline through withdrawn or completed 3 years follow up. The prescribed prednisone dosages had been weighted over the days of reporting period.
Time Frame	baseline-day 20, month 1,2, 3, 4, 6 and every 3 months through 36 months

Outcome Measure Data

Analysis Population Description

Four participants in each group did not provide the information to enable calculation of the time-weighted average prescribed alternate-day prednisone dose (mg) over 3 years.

Arm/Group Title	Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:	Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed	62	56
Mean (Standard Deviation); Unit of Measure: mg
	34.3 (22.7)	55.6 (29.4)

16. Secondary Outcome

Title	Penalized Time-weighted Average Alternative Day Prednisone Dose (mg; Method 1: Penalized Using Maximum Dose Before Azathioprine)
Description	For each participant who took azathioprine, we penalized them by taking the maximum dose of prednisone before azathioprine was added. We then applied the same method to compute the time-weighted alternative day prednisone dose from baseline, month 3, 4, 6 and every 3 months through 36 months.
Time Frame	baseline, month 3, 4, 6 and every 3 months through 36 months

Outcome Measure Data

Analysis Population Description

Five participants in Thymectomy plus prednisone and 4 in Prednisone alone group did not provide the information to enable calculation of the penalized time-weighted average alternate-day prednisone dose (mg) over 3 years.

Arm/Group Title	Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:	Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed	61	56
Mean (Standard Deviation); Unit of Measure: mg
	34.4 (28.2)	64.4 (40.8)

17. Secondary Outcome

Title	Penalized Time-weighted Average Alternative Day Prednisone Dose (mg; Method 2: Penalized Using Dose at Time of Starting Azathioprine)
Description	For each participant who took azathioprine, we penalized them by taking the prednisone dose at the time azathioprine commenced. We then applied the same method to compute the time-weighted alternative day prednisone dose from baseline, month 3, 4, 6 and every 3 months through 36 months.
Time Frame	baseline, month 1 , 2 , 3, 4, 6 and every 3 months through 36 months

Outcome Measure Data

Analysis Population Description

Five participants in Thymectomy plus prednisone and 4 in Prednisone alone group did not provide the information to enable calculation of the penalized time-weighted average alternate-day prednisone dose (mg) over 3 years.

Arm/Group Title	Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:	Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed	61	56
Mean (Standard Deviation); Unit of Measure: mg
	33.3 (27.2)	57.9 (36.9)

18. Secondary Outcome

Title	Time-Weighted Average MG Activity of Daily Living (MG-ADL)
Description	MG Activity of Daily Living total scores range from 0 to 24, with the lower scores indicating better daily living quality of life.
Time Frame	baseline, month 4, 6 and every 3 months through 36 months

Outcome Measure Data

Analysis Population Description

Five participants in each group did not provide the information to enable calculation of the time-weighted average MG activity of daily life over 3 years.

Arm/Group Title	Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:	Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed	61	55
Mean (Standard Deviation); Unit of Measure: units on a scale
	2.24 (2.09)	3.41 (2.58)

19. Secondary Outcome

Title	Time-Weighted Average MG Activity of Daily Living (MG-ADL) at Month 12, 24, and 36
Description	MG Activity of Daily Living total scores range from 0 to 24 by visit, with the lower scores indicating better daily living quality of life.
Time Frame	Month 12, 24, and 36

Outcome Measure Data

Analysis Population Description

Participants were in and out at month 12, 24 and 36 visit.

Arm/Group Title		Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:		Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed		66	60
Mean (Standard Deviation); Unit of Measure: units on a scale
Month 12	Number Analyzed	61 participants	54 participants
		1.92 (2.73)	3.33 (3.40)
Month 24	Number Analyzed	59 participants	53 participants
		2.02 (2.78)	3.11 (2.93)
Month 36	Number Analyzed	59 participants	51 participants
		2.14 (2.92)	2.69 (2.80)

20. Secondary Outcome

Title	Azathioprine Use
Description	[Not Specified]
Time Frame	baseline to 3 years

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:	Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed	65	58
Measure Type: Number; Unit of Measure: participants
	11	28

21. Secondary Outcome

Title	Plasma Exchange Use
Description	[Not Specified]
Time Frame	baseline to 3 years

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:	Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed	65	58
Measure Type: Number; Unit of Measure: participants
	10	9

22. Secondary Outcome

Title	Intravenous Immunoglobulin Use
Description	[Not Specified]
Time Frame	baseline to 3 years

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:	Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed	65	58
Measure Type: Number; Unit of Measure: participants
	11	23

23. Secondary Outcome

Title	Minimal Manifestation (MM) Status at Month 12, 24 and 36
Description	Number of participants who were in minimal manifestation status at month 12, 24 and 36.
Time Frame	Month 12, 24 and 36

Outcome Measure Data

Analysis Population Description

Number analyzed: Thymectomy plus prednisone: n=61 (Month 12), 59 (Month 24) , and 58 (Month 36); Prednisone alone n=54 (Month 12), 53 (Month 24), and 51 (Month 36)

Arm/Group Title		Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:		Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed		66	60
Measure Type: Number; Unit of Measure: participants
Month 12	Number Analyzed	61 participants	54 participants
		41	20
Month 24	Number Analyzed	59 participants	53 participants
		39	20
Month 36	Number Analyzed	58 participants	51 participants
		39	24

24. Secondary Outcome

Title	Cumulative Days in Hospital for Myasthenia Gravis Exacerbation
Description	Number of patients with MG exacerbation: Thymectomy plus prednisone=6 (out of 66); Prednisone alone=17 (out of 60)
Time Frame	baseline to 2 years

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:	Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed	6	17
Mean (Standard Deviation); Unit of Measure: days
	5.5 (2.9)	26.4 (28.9)

25. Secondary Outcome

Title	Cumulative Days in Hospital for Myasthenia Gravis Exacerbation
Description	Number of patients with MG exacerbation: Thymectomy plus prednisone=6 (out of 66); Prednisone alone=22 (out of 60)
Time Frame	baseline to 3 years

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:	Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed	6	22
Mean (Standard Deviation); Unit of Measure: days
	8.7 (7.7)	22.5 (27.1)

26. Secondary Outcome

Title	Short Form-36 Standardized Physical Component
Description	Range from 0 to 100, the higher the physical component value, the better the mental health.
Time Frame	Month 0, Month 12, Month 24 and Month 36

Outcome Measure Data

Analysis Population Description

Participants were in and out by visit.

Arm/Group Title		Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:		Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed		66	60
Median (Full Range); Unit of Measure: units on a scale
Month 0	Number Analyzed	65 participants	58 participants
		41.4; (18.2 to 60.0)	37.9; (13.5 to 64.3)
Month 12	Number Analyzed	61 participants	54 participants
		48.4; (11.5 to 64.4)	44.4; (21.2 to 58.8)
Month 24	Number Analyzed	59 participants	53 participants
		50.3; (11.2 to 60.5)	43.0; (25.3 to 59.6)
Month 36	Number Analyzed	58 participants	50 participants
		48.2; (9.8 to 61.8)	44.2; (20.4 to 58.9)

27. Secondary Outcome

Title	Short Form-36 Standardized Mental Component
Description	Range from 0 to 100, the higher the mental component value, the better the mental health.
Time Frame	Month 0, Month 12, Month 24 and Month 36

Outcome Measure Data

Analysis Population Description

Participants were in and out by visit.

Arm/Group Title		Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:		Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed		66	60
Median (Full Range); Unit of Measure: units on a scale
Month 0	Number Analyzed	65 participants	58 participants
		49.1; (14.6 to 65.3)	46.2; (22.4 to 70.7)
Month 12	Number Analyzed	61 participants	54 participants
		39.1; (17.6 to 66.7)	41.7; (7.6 to 64.4)
Month 24	Number Analyzed	59 participants	53 participants
		49.9; (14.4 to 62.3)	46.7; (21.3 to 69.1)
Month 36	Number Analyzed	58 participants	50 participants
		51.7; (29.9 to 65.1)	48.2; (24.1 to 69.2)

28. Secondary Outcome

Title	Treatment Associated Complications (TAC)
Description	Treatment associated complications measured complications occurred by myasthenia gravis patients. Report number of participant with at least one complications by each visit.
Time Frame	Month 0, 1, 2, 3, 4 then every 3 months through Month 36

Outcome Measure Data

Analysis Population Description

Participants were in and out by each visit.

Arm/Group Title		Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:		Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed		66	60
Measure Type: Count of Participants; Unit of Measure: Participants
Month 0	Number Analyzed	65 participants	58 participants
		22 33.8%	17 29.3%
Month 1	Number Analyzed	58 participants	56 participants
		15 25.9%	15 26.8%
Month 2	Number Analyzed	61 participants	55 participants
		22 36.1%	20 36.4%
Month 3	Number Analyzed	61 participants	55 participants
		21 34.4%	16 29.1%
Month 4	Number Analyzed	62 participants	56 participants
		23 37.1%	23 41.1%
Month 6	Number Analyzed	61 participants	55 participants
		21 34.4%	22 40.0%
Month 9	Number Analyzed	61 participants	54 participants
		37 60.7%	26 48.1%
Month 12	Number Analyzed	61 participants	54 participants
		35 57.4%	28 51.9%
Month 15	Number Analyzed	60 participants	53 participants
		29 48.3%	24 45.3%
Month 18	Number Analyzed	59 participants	53 participants
		28 47.5%	24 45.3%
Month 21	Number Analyzed	60 participants	52 participants
		22 36.7%	20 38.5%
Mpnth 24	Number Analyzed	59 participants	53 participants
		28 47.5%	23 43.4%
Mpnth 27	Number Analyzed	61 participants	53 participants
		25 41.0%	17 32.1%
Month 30	Number Analyzed	58 participants	53 participants
		23 39.7%	19 35.8%
Month 33	Number Analyzed	61 participants	52 participants
		24 39.3%	23 44.2%
Month 36	Number Analyzed	59 participants	51 participants
		23 39.0%	23 45.1%

29. Secondary Outcome

Title	Treatment Associated Symptoms (TAS)
Description	Treatment associated symptoms measured myasthenia gravis symptoms such as back pain and/or bruises. Report number of participant with at least one treatment associated symptoms by each visit.
Time Frame	Month 0, 1, 2, 3, 4 then every 3 months through Month 36

Outcome Measure Data

Analysis Population Description

Patients were in and out by visit

Arm/Group Title		Thymectomy Plus Prednisone	Prednisone Alone
Arm/Group Description:		Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.	Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Overall Number of Participants Analyzed		66	60
Measure Type: Count of Participants; Unit of Measure: Participants
Month 0	Number Analyzed	65 participants	57 participants
		63 96.9%	53 93.0%
Month 1	Number Analyzed	58 participants	56 participants
		55 94.8%	52 92.9%
Month 2	Number Analyzed	61 participants	55 participants
		61 100.0%	53 96.4%
Month 3	Number Analyzed	61 participants	55 participants
		60 98.4%	54 98.2%
Month 4	Number Analyzed	62 participants	56 participants
		59 95.2%	54 96.4%
Month 6	Number Analyzed	61 participants	55 participants
		60 98.4%	53 96.4%
Month 9	Number Analyzed	61 participants	54 participants
		59 96.7%	50 92.6%
Month 12	Number Analyzed	61 participants	54 participants
		56 91.8%	52 96.3%
Month 15	Number Analyzed	60 participants	53 participants
		56 93.3%	51 96.2%
Month 18	Number Analyzed	59 participants	53 participants
		52 88.1%	51 96.2%
Month 21	Number Analyzed	60 participants	52 participants
		52 86.7%	50 96.2%
Mpnth 24	Number Analyzed	59 participants	53 participants
		52 88.1%	51 96.2%
Mpnth 27	Number Analyzed	61 participants	53 participants
		50 82.0%	50 94.3%
Month 30	Number Analyzed	58 participants	53 participants
		49 84.5%	50 94.3%
Month 33	Number Analyzed	61 participants	51 participants
		51 83.6%	48 94.1%
Month 36	Number Analyzed	60 participants	51 participants
		49 81.7%	47 92.2%

Adverse Events

Time Frame	Adverse events were reported from baseline to 3 years
Adverse Event Reporting Description	There were two groups: 60 patients who received prednisone only and 66 patients had thymectomy plus prednisone
Arm/Group Title	Thymectomy Plus Prednisone		Prednisone Alone
Arm/Group Description	Procedure: Extended Transsternal Thymectomy plus prednisone treatment thymectomy: The thymectomy will be performed as soon as possible after randomization.		Drug: prednisone alone protocol prednisone: Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
All-Cause Mortality
	Thymectomy Plus Prednisone		Prednisone Alone
	Affected / at Risk (%)		Affected / at Risk (%)
Total	--/--		--/--
Serious Adverse Events
	Thymectomy Plus Prednisone		Prednisone Alone
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	25/66 (37.88%)		33/60 (55.00%)
Blood and lymphatic system disorders
Pancytopenia * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Ear and labyrinth disorders
Deafness * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Gastrointestinal disorders
Abdominal hernia * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Dyspepsia * 1	0/66 (0.00%)	0	1/60 (1.67%)	1
Umbilical hernia * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Vomiting * 1	1/66 (1.52%)	1	1/60 (1.67%)	1
General disorders
Cardiac death * 1	0/66 (0.00%)	0	1/60 (1.67%)	1
Hepatobiliary disorders
Bile duct stone * 1	0/66 (0.00%)	0	1/60 (1.67%)	1
Cholelithiasis * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Infections and infestations
Appendicitis * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Bronchitis bacterial * 1	1/66 (1.52%)	1	3/60 (5.00%)	4
Device related sepsis * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Diverticulitis * 1	0/66 (0.00%)	0	1/60 (1.67%)	4
Gastroenteritis * 1	0/66 (0.00%)	0	1/60 (1.67%)	1
Incision site cellulitis * 1	2/66 (3.03%)	2	0/60 (0.00%)	0
Pneumonia bacterial * 1	1/66 (1.52%)	2	3/60 (5.00%)	3
Pyelonephritis * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Pyelonephritis acute * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Septic shock * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Upper respiratory tract infection bacterial * 1	0/66 (0.00%)	0	1/60 (1.67%)	1
Urinary tract infection * 1	0/66 (0.00%)	0	1/60 (1.67%)	1
Viral upper respiratory tract infection * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Injury, poisoning and procedural complications
Fibula fracture * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Forearm fracture * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Incisional hernia * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Meniscus lesion * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Muscle injury * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Rib fracture * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Tibia fracture * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Metabolism and nutrition disorders
Diabetes mellitus inadequate control * 1	1/66 (1.52%)	2	0/60 (0.00%)	0
Musculoskeletal and connective tissue disorders
Osteonecrosis * 1	0/66 (0.00%)	0	1/60 (1.67%)	1
Osteopenia * 1	0/66 (0.00%)	0	1/60 (1.67%)	1
Osteoporotic fracture * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Breast cancer * 1	0/66 (0.00%)	0	1/60 (1.67%)	1
Nervous system disorders
Myasthenia gravis * 1	6/66 (9.09%)	7	22/60 (36.67%)	43
Myasthenia gravis crisis * 1	4/66 (6.06%)	6	10/60 (16.67%)	13
Phrenic nerve paralysis * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Pregnancy, puerperium and perinatal conditions
Abortion spontaneous * 1	0/66 (0.00%)	0	1/60 (1.67%)	1
Pregnancy * 1	0/66 (0.00%)	0	1/60 (1.67%)	1
Renal and urinary disorders
Renal failure * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Renal impairment * 1	0/66 (0.00%)	0	1/60 (1.67%)	1
Respiratory, thoracic and mediastinal disorders
Diaphragm muscle weakness * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Dyspnoea * 1	0/66 (0.00%)	0	1/60 (1.67%)	1
Hypoxia * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Pleural effusion * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Pleurisy * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Pneumothorax * 1	0/66 (0.00%)	0	1/60 (1.67%)	1
Pulmonary embolism * 1	0/66 (0.00%)	0	1/60 (1.67%)	1
Sleep apnoea syndrome * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Surgical and medical procedures
Eye operation * 1	0/66 (0.00%)	0	1/60 (1.67%)	1
Supportive care * 1	0/66 (0.00%)	0	1/60 (1.67%)	1
Thoracotomy * 1	0/66 (0.00%)	0	1/60 (1.67%)	1
Thymectomy * 1	0/66 (0.00%)	0	7/60 (11.67%)	7
Thyroidectomy * 1	1/66 (1.52%)	1	0/60 (0.00%)	0
Vascular disorders
Hypertensive crisis * 1	0/66 (0.00%)	0	1/60 (1.67%)	1
* Indicates events were collected by non-systematic assessment; 1 Term from vocabulary, MedDRA (12.0)
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	Thymectomy Plus Prednisone		Prednisone Alone
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	55/66 (83.33%)		48/60 (80.00%)
Eye disorders
Cataract * 1	4/66 (6.06%)	14	7/60 (11.67%)	20
Glaucoma * 1	5/66 (7.58%)	22	1/60 (1.67%)	1
Infections and infestations
Herpes zoster * 1	5/66 (7.58%)	6	4/60 (6.67%)	8
Injury, poisoning and procedural complications
Tendon rupture * 1	2/66 (3.03%)	2	3/60 (5.00%)	3
Investigations
Liver function test abnormal * 1	2/66 (3.03%)	2	3/60 (5.00%)	3
Metabolism and nutrition disorders
Abnormal weight gain * 1	32/66 (48.48%)	184	31/60 (51.67%)	141
Diabetes mellitus * 1	6/66 (9.09%)	41	9/60 (15.00%)	44
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain * 1	15/66 (22.73%)	43	4/60 (6.67%)	9
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Skin cancer * 1	1/66 (1.52%)	1	3/60 (5.00%)	3
Psychiatric disorders
Mental disorder * 1	4/66 (6.06%)	4	5/60 (8.33%)	8
Sleep disorder * 1	25/66 (37.88%)	86	22/60 (36.67%)	67
Skin and subcutaneous tissue disorders
Rash * 1	21/66 (31.82%)	58	24/60 (40.00%)	48
Scab * 1	11/66 (16.67%)	44	2/60 (3.33%)	2
Vascular disorders
Hypertension * 1	16/66 (24.24%)	53	16/60 (26.67%)	55
* Indicates events were collected by non-systematic assessment; 1 Term from vocabulary, MedDRA (12.0)

Limitations and Caveats

Potential weakness of this trial include the single-blinded and pill count methodology.

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact

• Name/Title: Dr. Gary Cutter, Professor
• Organization: University of Alabama at Birmingham
• Phone: 205-975-5048
• EMail: cutter@uab.edu

Publications of Results:

Aban IB, Wolfe GI, Cutter GR, Kaminski HJ, Jaretzki A 3rd, Minisman G, Conwit R, Newsom-Davis J; Mgtx Advisory Committee. The MGTX experience: challenges in planning and executing an international, multicenter clinical trial. J Neuroimmunol. 2008 Sep 15;201-202:80-4. doi: 10.1016/j.jneuroim.2008.05.031. Erratum in: J Neuroimmunol. 2009 Dec 10;217(1-2):103.

Newsom-Davis J, Cutter G, Wolfe GI, Kaminski HJ, Jaretzki A 3rd, Minisman G, Aban I, Conwit R. Status of the thymectomy trial for nonthymomatous myasthenia gravis patients receiving prednisone. Ann N Y Acad Sci. 2008;1132:344-7. doi: 10.1196/annals.1405.014.

Minisman G, Bhanushali M, Conwit R, Wolfe GI, Aban I, Kaminski HJ, Cutter G. Implementing clinical trials on an international platform: challenges and perspectives. J Neurol Sci. 2012 Feb 15;313(1-2):1-6. doi: 10.1016/j.jns.2011.10.004. Epub 2011 Nov 1. Review.

Kaminski HJ, Kusner LL, Wolfe GI, Aban I, Minisman G, Conwit R, Cutter G. Biomarker development for myasthenia gravis. Ann N Y Acad Sci. 2012 Dec;1275:101-6. doi: 10.1111/j.1749-6632.2012.06787.x. Review.

Wolfe GI, Kaminski HJ, Aban IB, Minisman G, Kuo HC, Marx A, Ströbel P, Mazia C, Oger J, Cea JG, Heckmann JM, Evoli A, Nix W, Ciafaloni E, Antonini G, Witoonpanich R, King JO, Beydoun SR, Chalk CH, Barboi AC, Amato AA, Shaibani AI, Katirji B, Lecky BR, Buckley C, Vincent A, Dias-Tosta E, Yoshikawa H, Waddington-Cruz M, Pulley MT, Rivner MH, Kostera-Pruszczyk A, Pascuzzi RM, Jackson CE, Garcia Ramos GS, Verschuuren JJ, Massey JM, Kissel JT, Werneck LC, Benatar M, Barohn RJ, Tandan R, Mozaffar T, Conwit R, Odenkirchen J, Sonett JR, Jaretzki A 3rd, Newsom-Davis J, Cutter GR; MGTX Study Group. Randomized Trial of Thymectomy in Myasthenia Gravis. N Engl J Med. 2016 Aug 11;375(6):511-22. doi: 10.1056/NEJMoa1602489. Erratum in: N Engl J Med. 2017 May 25;376(21):2097. [Dosage error in article text].

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Lee I, Kuo HC, Aban IB, Cutter GR, McPherson T, Kaminski HJ, Sussman J, Ströbel P, Oger J, Cea G, Heckmann JM, Evoli A, Nix W, Ciafaloni E, Antonini G, Witoonpanich R, King JO, Beydoun SR, Chalk CH, Barboi AC, Amato AA, Shaibani AI, Katirji B, Lecky BRF, Buckley C, Vincent A, Dias-Tosta E, Yoshikawa H, Waddington-Cruz M, Pulley MT, Rivner MH, Kostera-Pruszczyk A, Pascuzzi RM, Jackson CE, Verschuuren JJG, Massey JM, Kissel JT, Werneck LC, Benatar M, Barohn RJ, Tandan R, Mozaffar T, Conwit R, Minisman G, Sonett JR, Wolfe GI; MGTX study group. Minimal manifestation status and prednisone withdrawal in the MGTX trial. Neurology. 2020 Aug 11;95(6):e755-e766. doi: 10.1212/WNL.0000000000010031. Epub 2020 Jul 1.

Wolfe GI, Kaminski HJ, Aban IB, Minisman G, Kuo HC, Marx A, Ströbel P, Mazia C, Oger J, Cea JG, Heckmann JM, Evoli A, Nix W, Ciafaloni E, Antonini G, Witoonpanich R, King JO, Beydoun SR, Chalk CH, Barboi AC, Amato AA, Shaibani AI, Katirji B, Lecky BRF, Buckley C, Vincent A, Dias-Tosta E, Yoshikawa H, Waddington-Cruz M, Pulley MT, Rivner MH, Kostera-Pruszczyk A, Pascuzzi RM, Jackson CE, Verschuuren JJGM, Massey JM, Kissel JT, Werneck LC, Benatar M, Barohn RJ, Tandan R, Mozaffar T, Silvestri NJ, Conwit R, Sonett JR, Jaretzki A 3rd, Newsom-Davis J, Cutter GR; MGTX Study Group. Long-term effect of thymectomy plus prednisone versus prednisone alone in patients with non-thymomatous myasthenia gravis: 2-year extension of the MGTX randomised trial. Lancet Neurol. 2019 Mar;18(3):259-268. doi: 10.1016/S1474-4422(18)30392-2. Epub 2019 Jan 25.

• Responsible Party: Gary Cutter, PhD, University of Alabama at Birmingham
• ClinicalTrials.gov Identifier: NCT00294658
• Other Study ID Numbers: R01NS050733; 1U01NS042685-01A2 ( U.S. NIH Grant/Contract ); CRC ( Other Identifier: NINDS )
• First Submitted: February 21, 2006
• First Posted: February 22, 2006
• Results First Submitted: September 19, 2016
• Results First Posted: January 16, 2017
• Last Update Posted: May 23, 2017