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

NCT ID: NCT03127761

Last Updated: 2023-08-30

Basic Information

• Recruitment Status: RECRUITING
• Total Enrollment: 544 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2017-07-25
• Study Completion Date: 2028-04-30

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.

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.

Conditions

Multiple Myeloma

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Allogeneic HCT

Prospectively enrolled cohort of patients receiving allogeneic hematopoietic cell transplantation for multiple myeloma

Allogeneic Hematopoietic Stem Cell Transplant

Intervention Type: OTHER

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

No interventions assigned to this group

Interventions

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.

Intervention Type: OTHER

Eligibility 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

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Marrow Donor Program

OTHER

Sponsor Role: collaborator

Center for International Blood and Marrow Transplant Research

NETWORK

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Anita D'Souza, MD, MS

Role: STUDY_CHAIR

CIBMTR, Medical College of Wisconsin

Parameswaran Hari, MD, MS

Role: STUDY_CHAIR

CIBMTR, Medical College of Wisconsin

Locations

Center for International Blood and Marrow Transplant Research

Minneapolis, Minnesota, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Mona Patel

Role: CONTACT

mopatel@mcw.edu

414-805-0655

Facility Contacts

Michael Tierney

Role: primary

DatabaseIRB@NMDP.org

Other Identifiers

17-CMS-MM

Identifier Type: -

Identifier Source: org_study_id