Phase 2
N=41
Autologous Stem Cell Transplant Followed By Maintenance Therapy in Treating Elderly Patients With Multiple Myeloma
Extramedullary Plasmacytoma · Isolated Plasmacytoma of Bone · Light Chain Deposition Disease · Primary Systemic Amyloidosis · Stage I Multiple Myeloma
Bottom Line
View on ClinicalTrials.gov: NCT01849783 ↗Enrolled (actual)
41
Serious AEs
19.5%
Results posted
May 2022
Primary outcome: Primary: Median Progression Free Survival (mPFS) — 76.4 months
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- dexamethasone (Drug); cisplatin (Drug); doxorubicin (Drug); cyclophosphamide (Drug); etoposide (Drug); bortezomib (Drug); thalidomide (Drug); melphalan (Drug); autologous stem cell transplant (Procedure)
- Age
- Older Adult · 65+ yrs
- Sex
- All
- Sponsor
- Margarida Magalhaes-Silverman
- Primary completion
- Sep 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Median Progression Free Survival (mPFS) |
76.4 | — |
| PRIMARY Percentage of Participants With Serious Treatment-Related Complications |
4 | — |
| PRIMARY Percentage of Participants Able to Complete Full Course Therapy |
24 | — |
| SECONDARY Mean Change in Quality-Of-Life Indicators Post-Transplant |
1.3; -0.6; 1.1; 5.4; 4.6; 7.4 | — |
Summary
This phase II trial investigates whether patients greater than or equal to 65 years of age diagnosed with myeloma or another plasma cell malignancy will have better outcomes with transplant followed by maintenance therapy, as primarily measured by progression-free survival, versus non-transplant approaches.
Eligibility Criteria
Inclusion Criteria
- Participants must have had a diagnosis of symptomatic multiple myeloma (MM), MM + amyloidosis, or POEMS (osteosclerotic myeloma: polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, skin changes) requiring treatment; participants with a previous history of smoldering myeloma will be eligible if there is evidence of progressive disease requiring chemotherapy; Note that study participants do not need to have active disease at the time of study entry, as participants may have received up to 12 months of prior chemotherapy, which might have induced a response
- Protein criteria must be present at diagnosis (quantifiable M-component of IgG, IgA, IgD, or IgE and/or urinary kappa or lambda light chain, Bence-Jones protein, or Free Kappa Light Chain or Free Lambda Light Chain) in order to evaluate response. Non-secretory participants are eligible provided the participant has > 20% plasmacytosis OR multiple (≥3) plasmacytomas or lesions on MRI at the time of diagnosis or study enrollment , OR the presence of lesions on PET/CT scan or skeletal survey at diagnosis or study enrollment.
- Participants must have received ≤12 months of prior chemotherapy for this disease without evidence of progressive disease with treatment. Participants may have received prior radiotherapy provided approval has been obtained from the PI. Participants with a history of radiation who have a platelet count = 40% performed
- Participants must have adequate pulmonary function studies (PFTs), >= 50% of predicted on mechanical aspects (forced expiratory volume in 1 second [FEV^1}, forced vital capacity [FVC]) and diffusion capacity (diffusion capacity of the lung for carbon monoxide [DLCO]) >= 50% of predicted (adjusted for hemoglobin); if the participant is unable to complete pulmonary function tests (PFTs) due to disease-related pain or other circumstances that make it difficult to reliably perform PFTs, documentation of pulmonary function adequate for transplant will occur via a CT scan without evidence of major pulmonary disease, and arterial blood gas results
- Participants must have a creatinine 30mL/min/1.73m2
- Participants must have a performance status of 0-2 based on Eastern Cooperative Oncology Group (ECOG) criteria; participants with a poor performance status (3-4) based solely on bone pain will be eligible, provided there is documentation to verify this
- Participants must sign the most current institutional review board (IRB)-approved study (informed consent form) ICF
Exclusion Criteria
- Prior autologous or allogeneic transplant
- Progressive disease on prior treatment
- Platelet count 80% and packed with at least 80% plasma cells) the enrolling investigator must document this
- > Grade 3 neuropathy
- Known hypersensitivity to bortezomib, boron, or mannitol
- Uncontrolled diabetes on appropriate therapy
- Recent (= 3 mg/dl or a GFR <30mL/min/1.73m2.
- Participants must not have a concurrent malignancy unless it can be adequately treated by non-chemotherapeutic intervention; participants may have a history of prior malignancy, provided that he/she has not had any chemotherapy within 365 days of study entry AND that life expectancy exceeds 5 years at the time of study entry
- Participants must not have life-threatening co-morbidities
Data sourced from ClinicalTrials.gov (NCT01849783). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.