Efficacy, safety, and cost of pomalidomide in relapsed and refractory multiple myeloma

Guéneau, Paul; Chrétien, Marie‐Lorraine; Cransac-Miet, Amelie; Aho, Ludwig Serge; Lafon, Ingrid; Favennec, Camille; Guy, Julien; Caillot, Denis; Boulin, Mathieu

doi:10.1111/ejh.13039

Cited by 14 publications

(9 citation statements)

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“…This has been confirmed in an observational study that described prescribing practices of pomalidomide in a cohort of 63 consecutive MM patients. After a median follow-up of 28 months, 6% of patients had discontinued pomalidomide following severe adverse events, and 38% of patients had required a dose decrease following adverse events [15].…”

Section: Discussionmentioning

confidence: 99%

Adherence to immunomodulatory drugs in patients with multiple myeloma

et al. 2019

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Background Immunomodulatory drugs (thalidomide, lenalidomide and pomalidomide; IMID) are widely used in the treatment of multiple myeloma patients. To date, few data are available on IMID adherence in multiple myeloma patients. The aim of our study was to evaluate IMID adherence and to compare two indirect methods to measure IMID adherence in multiple myeloma patients: a specific questionnaire and the medication possession ratio (MPR). Another aim was to explore this specific questionnaire for the assessment of IMID adherence in multiple myeloma patients. Methods All consecutive multiple myeloma patients, with at least two consecutive dispensations of thalidomide, lenalidomide or pomalidomide in our hospital were included in this prospective study. IMID adherence was measured using a specific questionnaire and the medication possession ratio. Relationship between the questionnaire scores and variables of interest was evaluated by multiple linear regression with a robust variance estimator. Findings Sixty-three patients were included in our study. The mean questionnaire score was 8.2±1.2 and the mean medication possession ratio value was 0.97±0.06. A total of 76% of patients were considered adherent according to the questionnaire ( i . e . score ≥ 8), 94% according to the medication possession ratio ( i . e . MPR ≥ 0.90), and 70% according to the questionnaire and the medication possession ratio. No statistically significant linear association was observed between the questionnaire score and any variables of interest including medication possession ratio. All Cronbach’s alpha were relatively low (range 0.0342–0.2443), showing a low correlation of the different questions with the questionnaire score. Conclusions Our study is the first prospective study evaluating IMID adherence in multiple myeloma patients in real life. The high adherence to IMIDs reported here, regardless of the drug, is encouraging considering the efficacy, toxicity and elevated cost of IMIDs. The specific questionnaire should be used with caution to evaluate IMID adherence.

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Section: Discussionmentioning

confidence: 99%

Adherence to immunomodulatory drugs in patients with multiple myeloma

et al. 2019

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“…In a British series of 39 patients, including one third of DRMM, the ORR was 41%, with a median PFS of 5.2 months, and OS of 13.1 months [17]. Despite the dismal outcome, Sriskandarajah et al suggested that in some patients PomaD could maintain durable response rather than depth response, translating in longer overall survival [17].…”

Section: Discussionmentioning

confidence: 99%

Clinical Benefit of Long-Term Disease Control with Pomalidomide and Dexamethasone in Relapsed/Refractory Multiple Myeloma Patients

Parisi

Leotta

Romano

et al. 2019

JCM

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Background: We retrospectively analysed relapsed/refractory MM (RRMM) patients treated with pomalidomide and dexamethasone (PomaD) either in real life, or previously enrolled in an interventional (STRATUS, MM-010) or currently enrolled in an observational study (MM-015) to provide further insights on safety and tolerability and clinical efficacy. Methods: Between July 2013 and July 2018, 76 RRMM patients (including 33 double refractory MM) received pomalidomide 4 mg daily given orally on days 1–21 of each 28-day cycle, and dexamethasone 40 mg weekly (≤75 years) or 20 mg weekly for patients aged > 75 years. In nine patients a third agent was added to increase the response: Cyclophosphamide (in two fit patients) or clarithromycin (in seven frail patients). Patients received subcutaneous filgrastim as part of the prophylaxis regimen for neutropenia. Results: A median number of six (range 2–21) PomaD cycles were given. The regimen was well tolerated with grade 3–4 haematological and non-haematological adverse events in 39 (51%) and 25 (33%) patients, respectively. In patients who developed serious AE, pomalidomide dose reduction (11%, 14%) or definitive discontinuation (18%, 23%) were applied. All patients have been evaluated for response within the first two cycles. The disease control rate (DCR), i.e., those patients that had a response equal or better than stable disease (≥ SD), was high (89%), with 44% overall response rate (ORR) after six cycles. The achieved best responses were complete remission (CR, 5%), very good partial remission (VGPR, 4%), partial remission (PR, 35%), minimal response (MR, 7%), and stable disease (SD, 38%). After a median follow up of 19.6 months, median progression free survival was 9.4 months, and overall survival (OS) was 19.02 months. Univariate analysis showed that double refractory patients, or who received more than three previous lines had shorter PFS. At 18 months, regardless of the depth of response, patients with a disease control of at least six months, defined as maintenance of a best clinical and/or biochemical response to treatment for almost six months, had prolonged PFS (35.3% versus 20.6%, p = 0.0003) and OS (81.2% versus 15.9%, p < 0.0001) Conclusions: Our findings indicate that PomaD is a safe and well-tolerated regimen in real-life, associated with prolonged PFS and OS with acceptable toxicity. Moreover, Pd induced disease control in most intensively pre-treated patients and some of them achieved longer PFS than that obtained with the previous treatment.

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“…In the present study, only 21.4% of the patients were able to continue treatment for >1 year. Gueneau et al (16) reported POM effectiveness in the early recurrence phase. However, Palmas et al (17) reported its effectiveness in heavily pretreated patients.…”

Section: Discussionmentioning

confidence: 99%

Tolerability and safety of real‑world use of pomalidomide in patients with relapsed/refractory multiple myeloma

et al. 2018

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Pomalidomide (POM) is a second-generation immunomodulatory agent with proven efficacy in patients with relapsed/refractory multiple myeloma (RRMM) proven to be refractory to previous treatment with lenalidomide (LEN) and bortezomib. We herein conducted a retrospective analysis of 14 RRMM patients receiving POM to determine its tolerability and safety in the clinical setting. The median age of the patients was 72 years (range, 58-84 years), and 85.7% of the patients were aged >70 years. The most frequent treatment dose was 3 mg/day. POM dose reductions were required in 54.5% (6/11) of the patients. The patient data were compared among three age groups (<70, 70-75 and >75 years) and there was only significant difference in daily POM treatment dose. The tolerability of POM must be confirmed, particularly in elderly patients. Dose reduction from 4 to 3 mg occurred during the second cycle in 83.3% (5/6) of the patients. It is important to determine the tolerability of POM in the early phases of treatment. The most frequently reported grade 3/4 hematological adverse events were neutropenia (64.3%), anemia (64.3%) and thrombocytopenia (57.1%). Although the median number of treatment cycles was 4 (range, 1-13), 21.4% (3/14) of the patients with a performance status (PS) of 3 were administered only 1 treatment cycle. The tolerability of POM was low among patients with poor PS and an aggressive treatment introduction should be avoided. However, 21.4% (3/14) of the patients were able to continue treatment for >1 year and some patients received long-term therapy. POM does not require dose modification for renal function, and multiple capsule doses are available, which is an advantage of POM compared with LEN. POM may be administered to late-stage RRMM patients in a real-world clinical setting, but elderly patients or those with poor PS must be treated with caution. In this manner, the treatment options for RRMM patients may be expanded by assessing the tolerability and safety of POM.

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Efficacy, safety, and cost of pomalidomide in relapsed and refractory multiple myeloma

Abstract: The study demonstrated that pomalidomide-dexamethasone regimen has a long-term favorable safety-efficacy profile in RRMM patients. The survival benefit is substantial even in patients with stable disease.

Cited by 14 publications

References 14 publications

Adherence to immunomodulatory drugs in patients with multiple myeloma

Adherence to immunomodulatory drugs in patients with multiple myeloma

Clinical Benefit of Long-Term Disease Control with Pomalidomide and Dexamethasone in Relapsed/Refractory Multiple Myeloma Patients

Tolerability and safety of real‑world use of pomalidomide in patients with relapsed/refractory multiple myeloma

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