title: [Effectiveness and safety of lenalidomide in myelofibrosis patients: a case series from the Spanish compassionate use program].
abstract: OBJECTIVE
  Myelofibrosis (MF) is a chronic myeloproliferative neoplasm (CMPN) characterized by clonal proliferation of the pluripotent hematopoietic germinal cell, fibrosis, and bone marrow angiogenesis, and with extra medullary hematopoiesis. Lenalidomide is an immunomodulatory and antiangiogenic agent that has shown clinical benefit in MF patients in several phase II clinical trials. In this work, we present the results of the retrospective assessment of a series of 32 patients diagnosed with MF that received treatment with lenalidomide within the Spanish program of hospital compassionate use.
  
  
  METHODS
  Multicenter, retrospective study of a case series of MF patients receiving compassionate treatment with lenalidomide.
  
  
  RESULTS
  We gathered information on 32 MF patients that were treated with lenalidomide at 17 Spanish hospitals. The mean age was 68 years (range, 50-83), 72% males. Twenty-six (81%) patients had intermediate-high risk according to Dupriez prognostic score. In 16 patients, the daily dose scheduled was 10 mg/day for 21 days in 28-day cycles; the remaining patients received lower doses or other regimes. In 29 out of 32 patients, response assessment was available. Two patients had complete clinical and hematologic response and 14 had partial response. The most common adverse events observed were neutropenia (56%), thrombocytopenia (50%), and anemia (40%).
  
  
  CONCLUSIONS
  Continuous treatment with lenalidomide is active in MF patients pretreated heavily. Lenalidomide-induced myelosuppression and other associated toxicities are manageable with dose adjustments.
authors.forename: I
authors.initials: I
authors.lastname: Castillo
authors.forename: Ma A
authors.initials: MA
authors.lastname: Ojea
authors.forename: C
authors.initials: C
authors.lastname: Boqué
authors.forename: A
authors.initials: A
authors.lastname: Asensio
authors.forename: Ma M
authors.initials: MM
authors.lastname: Hermosilla
authors.forename: M
authors.initials: M
authors.lastname: Blanes
mesh_terms: D000368:Aged; D000369:Aged, 80 and over; D057176:Compassionate Use Trials; D005260:Female; D006801:Humans; D007155:Immunologic Factors / Q000009:adverse effects / Q000627:therapeutic use*; D000077269:Lenalidomide; D008297:Male; D008875:Middle Aged; D055728:Primary Myelofibrosis / Q000188:drug therapy*; D012189:Retrospective Studies; D013030:Spain; D013792:Thalidomide / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use
chemical_list: D007155:Immunologic Factors; D013792:Thalidomide; D000077269:Lenalidomide