title: Improvement of Anaemia in Patients with Primary Myelofibrosis by Low-Dose Thalidomide and Prednisone.
abstract: BACKGROUND
  A combination of low-dose thalidomide and corticosteroids is a treatment option for anaemic patients with primary myelofibrosis (PMF) who are not eligible for allogeneic hematopoietic stem cell transplantation.
  
  
  METHODS
  We describe the outcomes of 13 patients with PMF treated with thalidomide 50 mg daily in combination with prednisone 0.5 mg/kg daily. Treatment responses were seen in 10/13 (77%) patients with a median onset of therapeutic effect at 4 weeks (range 3-7 weeks) after treatment initiation. Improvements of anaemia and thrombocytopenia and reduction in splenomegaly were observed in 70%, 38%, and 30% of patients, respectively. Four of six initially transfusion-dependent patients became transfusion independent following the therapy. The median duration of treatment response was 18 months (range 3-35 months). The treatment was well tolerated, with only one patient discontinuing therapy due to toxicity. Responders included both patients with and without JAK2 V617F, and included patients with both newly diagnosed and longstanding PMF.
  
  
  CONCLUSIONS
  Our retrospective analysis confirmed that the therapy with low-doses thalidomide with prednisone in patients with PMF achieves significant response rate in anaemia with low treatment toxicity.
authors.affiliation: 4th Department of Internal Medicine - Hematology, Charles University, Faculty Hospital and Faculty of Medicine in Hradec Králové, Hradec Králové, Czech Republic. belohlavkova@fnhk.cz.
authors.forename: Petra
authors.initials: P
authors.lastname: Bělohlávková
authors.affiliation: 4th Department of Internal Medicine - Hematology, Charles University, Faculty Hospital and Faculty of Medicine in Hradec Králové, Hradec Králové, Czech Republic.
authors.forename: Vladimír
authors.initials: V
authors.lastname: Maisnar
authors.affiliation: 4th Department of Internal Medicine - Hematology, Charles University, Faculty Hospital and Faculty of Medicine in Hradec Králové, Hradec Králové, Czech Republic.
authors.forename: Jaroslava
authors.initials: J
authors.lastname: Voglová
authors.affiliation: Department of Oncology, First Faculty of Medicine and Thomayer Hospital, Prague, Czech Republic.
authors.forename: Tomáš
authors.initials: T
authors.lastname: Buchler
authors.affiliation: 4th Department of Internal Medicine - Hematology, Charles University, Faculty Hospital and Faculty of Medicine in Hradec Králové, Hradec Králové, Czech Republic.
authors.forename: Pavel
authors.initials: P
authors.lastname: Žák
mesh_terms: D000368:Aged; D000369:Aged, 80 and over; D000741:Anemia, Aplastic / Q000097:blood / Q000188:drug therapy*; D004305:Dose-Response Relationship, Drug; D004359:Drug Therapy, Combination; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D010976:Platelet Count; D011241:Prednisone / Q000008:administration & dosage*; D055728:Primary Myelofibrosis / Q000097:blood / Q000188:drug therapy*; D013792:Thalidomide / Q000008:administration & dosage*
chemical_list: D013792:Thalidomide; D011241:Prednisone
keywords: Immunomodulatory agents; Primary myelofibrosis; Thalidomide; Treatment