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

Multiple Myeloma Clinical Trial

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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03127761
• Other study ID #: 17-CMS-MM
• Status: Recruiting
• Start date: July 25, 2017
• Est. completion date: April 2028

Study information

• Verified date: August 2023
• Source: Center for International Blood and Marrow Transplant Research
• Contact: Mona Patel
• Phone: 414-805-0655
• Email: mopatel[@]mcw.edu
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 544
• Est. completion date: April 2028
• Est. primary completion date: May 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Multiple Myeloma
• Neoplasms, Plasma Cell

Intervention

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.

Locations

Country	Name	City	State
United States	Center for International Blood and Marrow Transplant Research	Minneapolis	Minnesota

Sponsors (2)

Lead Sponsor	Collaborator
Center for International Blood and Marrow Transplant Research	National Marrow Donor Program

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Compare five-year survival	Compare five-year overall survival between the alloHCT cohort and an age and disease risk matched cohort of autoHCT patients	5 years post transplant
Secondary	Progression-free survival (PFS)	five-year PFS probabilities between the AlloHCT cohort and an age and disease risk matched cohort of autoHCT patients	5 years post transplant
Secondary	Relapse or progression	Myeloma recurrence or progression will be defined per International Myeloma Working Group (IMWG) guidelines	5 years post transplant
Secondary	Transplant related mortality	Death from any cause within 28 days after alloHCT or death in the absence of progression/relapse of MM after day 28 post transplant	5 years post transplant
Secondary	Incidence of acute GVHD	Occurrence of Grade I, II and III/IV skin, gastrointestinal, or liver abnormalities fulfilling the Consensus criteria of Grades II-IV acute GVHD	5 years post transplant
Secondary	Incidence of chronic GVHD	Occurrence of symptoms in any organ system fulfilling the criteria of chronic GVHD	5 years post transplant