HealthTree Foundation, Lehi, UT
Nathan W. Sweeney , Eduardo Franco Hernandez , Nolan Cole , Scott Ryan Goldsmith , Zachary Crees , Jenny Ahlstrom
Background: Chimeric antigen receptor (CAR) T-cell therapies are in clinical development for multiple myeloma (MM). Patient-reported outcomes (PRO) can provide valuable insights into how patients perceive the relative risks and benefits of these new therapies. This study aimed to evaluate CAR T-cell therapy in relapsed/refractory MM (RRMM) patients. Methods: We utilized HealthTree Cure Hub for Multiple Myeloma to analyze patient-reported data from 17 patients who participated in CAR T-cell clinical trials. In this study, we examined total prior lines of therapy, time to next treatment (TNT), overall survival (OS), patient-reported toxicity and severity, and patient-reported outcomes (myeloma reduction or no myeloma reduction). The Kaplan-Meier model was used to calculate overall survival. The severity of toxicity was assessed using a 1 to 10 scale (1 = minimal and 10 = severe). Results: Our analysis of the 17 patients found a median of 10 lines of therapy prior to CAR T-cell treatment. Ten patients had no new treatments to report at the time of this study, 5 patients reported new treatment with a median TNT of 15.9 months, and for 2 patients we did not have data. The median OS was 24 months (95% CI 21-30 months). The probability that a patient remained alive at 2 years was 0.48 (95% CI: 0.195, 1). Four of the 17 patients died with a median of 22.5 months post-CAR T-cell therapy. Two of these patients died without reporting a change in treatment. There was a total of 36 different side effects reported by patients as a result of the therapy. Table lists the side effects experienced by 2 or more patients and the corresponding average severity. Finally, 76% of patients treated with CAR T-cell therapy reported a reduction in their myeloma, four of these patients had m-protein levels reported and saw an average decrease of 93%. Of the remaining patients, three (18%) reported little to no reduction in their myeloma, and one patient (6%) did not know their response at the time of this analysis. Conclusions: Our investigation of patient-reported results suggests an emerging and viable immunotherapy treatment option for RRMM, with encouraging outcomes and manageable side effects. Future directions include analysis of genetics and treatment options post CAR T-cell therapy. These data highlight the expedited benefit of using PROs via an online platform, like HealthTree Cure Hub, to assess new therapeutics in the research community.
Side Effect | Frequency | Severity Average (1-10) |
---|---|---|
Neutropenia or low lymphocyte | 9 | 3 |
Anemia | 7 | 3.6 |
Fatigue | 7 | 3.9 |
Fever/chills | 7 | 3.1 |
Thrombocytopenia | 6 | 3.7 |
Hypotension | 4 | 1.3 |
Lack of appetite | 4 | 1.8 |
Nausea | 3 | 3.3 |
Decreased weight | 3 | 3 |
Headaches | 3 | 1 |
Diarrhea | 3 | 3.3 |
Low white blood cells - Granulocytes | 3 | 1.3 |
Muscle weakness | 2 | 2.5 |
Bone pain | 2 | 4.5 |
Joint pain | 2 | 4.5 |
Difficulty - speaking | 2 | 3 |
Cough | 2 | 5 |
Dyspnea | 2 | 6.5 |
Pneumonia/bronchitis | 2 | 8.5 |
Gas/bloating | 2 | 1.5 |
Hair loss | 2 | 5.5 |
Upper respiratory Infection | 2 | 7.5 |
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