Analysis of Efficacy and Prognostic Factors of Lenalidomide Treatment as Part of a Dutch Compassionate Use Program

Evelien Kneppers, Henk M. Lokhorst, Corien M. Eeltink, Gerwin Huls, Marie José Kersten, Jan Koedam, Monique C. Minnema, Marinus H. J. van Oers, Reinier A. P. Raymakers, Martijn R. Schaafsma, Edo Vellenga, Pierre W. Wijermans, Shulamiet Wittebol, Pieter Sonneveld, Sonja Zweegman

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Background and Methods: To obtain efficacy and safety data on lenalidomide treatment outside of clinical trials, we analyzed the clinical data of 114 patients with refractory or relapsed multiple myeloma treated with lenalidomide on a compassionate use basis. The recommended treatment consisted of lenalidomide 25 mg given on days 1-21 of a 28-day cycle, in combination with dexamethasone. A median of 3 previous lines of therapy were given, including thalidomide in 91%. Most patients were treated until progression or intolerable toxicity. Results: The median number of cycles was 7 (range, 1-21 + cycles) with a maximum response after a median of 3 cycles (range, 1-10 cycles). The overall response rate was 69%, including complete response in 6%, very good partial response in 19%, and partial response in 44%. The response rate was not influenced by previous thalidomide and/or bortezomib treatment. The median time to progression (TTP) was 9 months and the median overall survival (OS) was 22 months. A significantly longer TTP was observed in patients who previously underwent allogeneic stem cell transplantation (12.5 months vs. 8 months; P = .036). Overall survival was significantly affected by performance status (P <.0001). Lenalidomide toxicity was predominantly hematologic (37%; Common Toxicity Criteria >= 3) and the incidence of venous thrombotic events was low (5%) using the recommended prophylaxis. Conclusion: This analysis confirms that, outside clinical prospective trials, treatment with lenalidomide is highly effective and feasible in heavily pretreated patients with multiple myeloma
Original languageEnglish
Pages (from-to)138-143
JournalClinical lymphoma, myeloma & leukemia
Issue number2
Publication statusPublished - 2010

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