Multiple Myeloma
Conditions
Keywords
Multiple Myeloma
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.
Interventions
This observational study will compare outcomes of prospectively enrolled HCT recipients with outcomes of a cohort of matched autoHCT controls.
Sponsors
National Marrow Donor Program
Center for International Blood and Marrow Transplant Research
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Compare five-year survival | 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
| Measure | Time frame | Description |
|---|---|---|
| Relapse or progression | 5 years post transplant | Myeloma recurrence or progression will be defined per International Myeloma Working Group (IMWG) guidelines |
| Transplant related mortality | 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 |
| Progression-free survival (PFS) | 5 years post transplant | five-year PFS probabilities between the AlloHCT cohort and an age and disease risk matched cohort of autoHCT patients |
| Incidence of acute GVHD | 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 |
| Incidence of chronic GVHD | 5 years post transplant | Occurrence of symptoms in any organ system fulfilling the criteria of chronic GVHD |
Countries
United States
Contacts
Primary ContactMona Patel
Outcome results
None listed