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Assessment of Allogeneic Hematopoietic Cell Transplantation in Medicare Beneficiaries With Multiple Myeloma

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ClinicalTrials.gov Identifier: NCT03127761
Recruitment Status : Recruiting
First Posted : April 25, 2017
Last Update Posted : September 13, 2017
Sponsor:
Collaborator:
National Marrow Donor Program
Information provided by (Responsible Party):
Center for International Blood and Marrow Transplant Research

Brief Summary:

Multiple myeloma (MM) is the second most common hematologic malignancy in adults. The current standard of care for MM patients fit to undergo high dose conditioning chemotherapy is an autologous HCT (autoHCT). Allogeneic HCT (alloHCT) is the only potentially curative therapy available to patients with MM. However, the significant morbidity and mortality of this procedure historically limited its application in older patients.

Thus, although potentially curative, standard risk MM patients have excellent prognoses in the era of novel therapies which reduces the overall benefit of alloHCT. However, because the outcomes for high-risk MM remain poor despite the best available standard therapies (overall survival of 24-36 months), initial data suggest that alloHCT should be explored in this subset.


Condition or disease Intervention/treatment
Multiple Myeloma Other: Allogeneic Hematopoietic Stem Cell Transplant

Detailed Description:

Multiple myeloma (MM) is the second most common hematologic malignancy in adults. Overall survival (OS) in MM has improved significantly in the last 15 years with the emergence of novel therapies such as thalidomide, bortezomib and lenalidomide. The median life expectancy of patients with MM treated in the current era is more than 6 years, while SEER data from a slightly earlier time period (2008-12) estimated the 5 year survival at 48.5%. However, prognosis is not uniform and varies considerably based on a presenting features and response to therapy.

The current standard of care for MM patients fit to undergo high dose conditioning chemotherapy is an autologous HCT (autoHCT). There is controversy regarding the timing of autoHCT after initial novel therapy induction with randomized trials showing similar OS whether done early or delayed to time of relapse as salvage therapy. However, more recent trials comparing early versus delayed transplant support the benefit of early upfront autoHCT.

Allogeneic HCT (alloHCT) is the only potentially curative therapy available to patients with MM. However, the significant morbidity and mortality of this procedure historically limited its application in older patients. Current data from the Center for International Blood and Marrow Research (CIBMTR) show transplant-related mortality rates of 23 (20-26)% at 5 years with myeloablative conditioning.

Thus, although potentially curative, standard risk MM patients have excellent prognoses in the era of novel therapies which reduces the overall benefit of alloHCT. However, because the outcomes for high-risk MM remain poor despite the best available standard therapies (overall survival of 24-36 months), initial data suggest that alloHCT should be explored in this subset.


Study Type : Observational
Estimated Enrollment : 544 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Assessment of Allogeneic Hematopoietic Cell Transplantation in Medicare Beneficiaries With Multiple Myeloma: A Study to Develop Evidence of Effectiveness for the Centers for Medicare and Medicaid Services (CMS)
Actual Study Start Date : July 25, 2017
Estimated Primary Completion Date : May 2027
Estimated Study Completion Date : April 2028

Resource links provided by the National Library of Medicine


Group/Cohort Intervention/treatment
Allogeneic HCT
Prospectively enrolled cohort of patients receiving allogeneic hematopoietic cell transplantation for multiple myeloma
Other: Allogeneic Hematopoietic Stem Cell Transplant
This observational study will compare outcomes of prospectively enrolled HCT recipients with outcomes of a cohort of matched autoHCT controls.

Historical autoHCT
Historical cohort of patients with autologous hematopoietic cell transplantation between 2010 and 2016



Primary Outcome Measures :
  1. Compare five-year survival [ Time Frame: 5 years post transplant ]
    Compare five-year overall survival between the alloHCT cohort and an age and disease risk matched cohort of autoHCT patients


Secondary Outcome Measures :
  1. Progression-free survival (PFS) [ Time Frame: 5 years post transplant ]
    five-year PFS probabilities between the AlloHCT cohort and an age and disease risk matched cohort of autoHCT patients

  2. Relapse or progression [ Time Frame: 5 years post transplant ]
    Myeloma recurrence or progression will be defined per International Myeloma Working Group (IMWG) guidelines

  3. Transplant related mortality [ Time Frame: 5 years post transplant ]
    Death from any cause within 28 days after alloHCT or death in the absence of progression/relapse of MM after day 28 post transplant

  4. Incidence of acute GVHD [ Time Frame: 5 years post transplant ]
    Occurrence of Grade I, II and III/IV skin, gastrointestinal, or liver abnormalities fulfilling the Consensus criteria of Grades II-IV acute GVHD

  5. Incidence of chronic GVHD [ Time Frame: 5 years post transplant ]
    Occurrence of symptoms in any organ system fulfilling the criteria of chronic GVHD



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
A prospective cohort study of approximately 544 patients receiving allogeneic HCT for multiple myeloma in CIBMTR centers in the US matched to a historical control patients treated with autoHCT between 2010 and 2016
Criteria

Inclusion Criteria:

  • Medicare beneficiary
  • Stage II or III multiple myeloma and/or primary plasma cell leukemia
  • Eligible to receive an allogeneic HCT from any suitable allogeneic donor (as determined by the transplant center) including umbilical cord blood
  • Will receive allogeneic HCT at a US transplant center
  • Agree to submit comprehensive clinical data on their pre- and post-transplant clinical status and outcomes to the CIBMTR

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03127761


Contacts
Contact: Sue Logan 763-406-8575 slogan@nmdp.org

Locations
United States, Minnesota
Center for International Blood and Marrow Transplant Research Recruiting
Minneapolis, Minnesota, United States, 55401
Contact: Sue Logan       slogan@nmdp.org   
Sponsors and Collaborators
Center for International Blood and Marrow Transplant Research
National Marrow Donor Program
Investigators
Study Chair: Anita D'Souza, MD, MS CIBMTR, Medical College of Wisconsin
Study Chair: Parameswaran Hari, MD, MS CIBMTR, Medical College of Wisconsin

Responsible Party: Center for International Blood and Marrow Transplant Research
ClinicalTrials.gov Identifier: NCT03127761     History of Changes
Other Study ID Numbers: 17-CMS-MM
First Posted: April 25, 2017    Key Record Dates
Last Update Posted: September 13, 2017
Last Verified: September 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Center for International Blood and Marrow Transplant Research:
Multiple Myeloma

Additional relevant MeSH terms:
Multiple Myeloma
Neoplasms, Plasma Cell
Neoplasms by Histologic Type
Neoplasms
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Paraproteinemias
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases