Lenalidomide Approved for Myeloma Maintenance After HSCT

Zosia Chustecka


February 23, 2017

The myeloma drug lenalidomide (Revlimid, Celgene) has been granted an expanded indication by the US Food and Drug Administration (FDA) and is now approved for use as a maintenance therapy in patients who have received an autologous hematopoietic stem cell transplant (auto-HSCT).

This is the first and only drug with such an indication, the company noted.

The approval is based on data from two controlled studies (the United States–based CALGB 100104 trial and the European-based IFM 2005-02 trial), which included more than 1000 patients who had undergone an auto-HSCT after induction therapy.

The results show that lenalidomide maintenance therapy improved median progression-free survival (PFS), the main study endpoint, compared with no maintenance therapy.

Median PFS was improved by 3.8 years in the US study (5.7 years with lenalidomide vs 1.9 years for no maintenance; hazard ratio [HR], 0.38) and was improved by 1.9 years in the European study (median PFS, 3.9 years vs 2 years; HR, 0.53).

The median overall survival (OS) was also improved, although this was a descriptive analysis because these studies were not powered for OS, the manufacturer noted in a press release. The median OS was improved to 9.3 years compared with 7 years in one study, and up to 8.8 years compared with 7.3 months in the other study.

"Autologous stem cell transplant after induction therapy is part of the continuum of care for transplant-eligible multiple myeloma patients. However, most patients will still see their disease recur or progress after this treatment," Philip McCarthy, MD, director of the Blood and Marrow Transplant Center at Roswell Park Cancer Institute, Buffalo, New York, commented in the press release.

"Lenalidomide maintenance therapy, which has been shown to increase progression-free survival following autologous stem cell transplant in clinical trials, can be considered a standard of care for these patients, " he added.

Other experts have also said that lenalidomide maintenance after auto-HSCT is a standard of care. In fact, it is "clearly a standard of care," according to the conclusion of a meta-analysis from the American Society of Clinical Oncology, as reported at the time by Medscape Medical News.

The pooled results from three randomized trials showed a 26% reduction in the risk for death with lenalidomide, representing an estimated 2.5-year increase in median survival compared with placebo.

Although a survival advantage for lenalidomide as maintenance therapy has not been demonstrated consistently (some studies have not shown a benefit), "in this analysis, there is clear evidence that lenalidomide prolongs the overall survival of the patient," noted Meletios A. Dimopoulos, MD, National and Kapodistrian University of Athens School of Medicine, Greece.

Lenalidomide is available only through a restricted distribution program, Revlimid REMS. The prescribing information notes that patients with myeloma treated with lenalidomide may develop deep-vein thrombosis, pulmonary embolism, myocardial infarction, and stroke, so thromboprophylaxis is recommended.

The most frequently reported adverse reactions, reported in at least 20% of patients receiving lenalidomide in both maintenance studies, were neutropenia, thrombocytopenia, leukopenia, anemia, upper respiratory tract infection, bronchitis, nasopharyngitis, cough, gastroenteritis, diarrhea, rash, fatigue, asthenia, muscle spasm, and pyrexia.

In patients receiving lenalidomide maintenance therapy, hematologic second primary malignancies (SPMs) occurred in 7.5% of patients, compared with 3.3% of patients receiving placebo. The incidence of hematologic plus solid tumor (excluding squamous cell carcinoma and basal cell carcinoma) SPMs was 14.9%, compared with 8.8% in patients receiving placebo, with a median follow-up of 91.5 months.

Patients should be monitored for the development of SPMs, the company notes.

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