Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups

	Description
Intent-To-Treat	All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, study medications and irradiation.

Participant Flow:   Overall Study

	Intent-To-Treat
STARTED	10
COMPLETED	10
NOT COMPLETED	0

Reporting Groups

	Description
Intent-To-Treat	All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, study medications and irradiation.

Baseline Measures

	Intent-To-Treat
Number of Participants   [units: participants]	10
Age   [units: participants]
<=18 years	10
Between 18 and 65 years	0
>=65 years	0
Age   [units: years] Mean ± Standard Deviation	4.8 ± 7.6
Gender   [units: participants]
Female	3
Male	7
Region of Enrollment   [units: participants]
United States	10

1.  Primary:

Number of Patients Alive (Survival)at 2 Years   [ 2 years ]

2.  Secondary:

Number of Patients Alive at Three Years (Survival)   [ 3 years ]

3.  Secondary:

Number of Patients With Succcessful Engraftment After Transplantation   [ 42 Days ]

4.  Secondary:

Number of Patients With Grade 2-4 Acute Graft Versus Host Disease   [ 100 Days ]

5.  Secondary:

Number of Patients With Chronic Graft Versus Host Disease   [ 2 years ]

6.  Secondary:

Number of Patients With Disease Recurrence   [ 2 years ]