title: Low-dose thalidomide ameliorates cytopenias and splenomegaly in myelofibrosis with myeloid metaplasia: a phase II trial.
abstract: OBJECTIVE
  A phase II dose-escalation trial was conducted to ascertain low-dose thalidomide safety and response in patients with advanced myelofibrosis with myeloid metaplasia (MMM).
  
  
  METHODS
  Thalidomide was administered together with current therapy to 63 patients, starting at 50 mg daily and increasing to 400 mg as tolerated.
  
  
  RESULTS
  Half of the patients sustained daily doses more than 100 mg and the drop-out rate was 51% at 6 months: the drop-out rate was lower in patients with high baseline fatigue score. At efficacy analysis, anemia was ameliorated in 22% of the patients and transfusions were eliminated in 39% of transfusion-dependent patients. Platelet count increased by 50 x 10(9)/L or more in 22% of patients with an initial count lower than 100 x 10(9)/L. Splenomegaly decreased by more than 50% of the initial size in 19% of patients. Reduction of an overall disease severity score occurred in 31% of patients and was associated with a significant reduction of fatigue. Disease severity amelioration was independently predicted by a high baseline myeloproliferative index (ie, large splenomegaly, thrombocytosis, or leukocytosis).
  
  
  CONCLUSIONS
  Low-dose thalidomide displays an acceptable toxicity profile and provides an objective and subjective advantage to a relevant portion of MMM patients.
authors.affiliation: Laboratory of Medical Informatics, Unit of Clinical Immunology and Immunohematology, IRCCS Policlinico San Matteo, viale Golgi 19, 27100 Pavia, Italy.
authors.forename: Monia
authors.initials: M
authors.lastname: Marchetti
authors.forename: Giovanni
authors.initials: G
authors.lastname: Barosi
authors.forename: Francesca
authors.initials: F
authors.lastname: Balestri
authors.forename: Gianluca
authors.initials: G
authors.lastname: Viarengo
authors.forename: Sara
authors.initials: S
authors.lastname: Gentili
authors.forename: Sara
authors.initials: S
authors.lastname: Barulli
authors.forename: Jean-Loup
authors.initials: JL
authors.lastname: Demory
authors.forename: Fiorella
authors.initials: F
authors.lastname: Ilariucci
authors.forename: Antonio
authors.initials: A
authors.lastname: Volpe
authors.forename: Dominique
authors.initials: D
authors.lastname: Bordessoule
authors.forename: Alberto
authors.initials: A
authors.lastname: Grossi
authors.forename: Marie Caroline
authors.initials: MC
authors.lastname: Le Bousse-Kerdiles
authors.forename: Andrea
authors.initials: A
authors.lastname: Caenazzo
authors.forename: Alessandro
authors.initials: A
authors.lastname: Pecci
authors.forename: Antonietta
authors.initials: A
authors.lastname: Falcone
authors.forename: Giorgio
authors.initials: G
authors.lastname: Broccia
authors.forename: Cesarina
authors.initials: C
authors.lastname: Bendotti
authors.forename: Fredric
authors.initials: F
authors.lastname: Bauduer
authors.forename: Francesco
authors.initials: F
authors.lastname: Buccisano
authors.forename: Brigitte
authors.initials: B
authors.lastname: Dupriez
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000740:Anemia / Q000517:prevention & control; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage* / Q000009:adverse effects; D007970:Leukopenia / Q000517:prevention & control*; D008297:Male; D008875:Middle Aged; D010976:Platelet Count; D055728:Primary Myelofibrosis / Q000188:drug therapy* / Q000473:pathology; D012449:Safety; D012720:Severity of Illness Index; D013163:Splenomegaly / Q000517:prevention & control*; D013792:Thalidomide / Q000008:administration & dosage* / Q000009:adverse effects; D013921:Thrombocytopenia / Q000517:prevention & control*; D016896:Treatment Outcome
chemical_list: D007166:Immunosuppressive Agents; D013792:Thalidomide