title: Thalidomide in advanced mastocytosis.
abstract: Mastocytosis is an acquired orphan disease characterized by the abnormal accumulation of mast cells responsible for organ failure and systemic symptoms. Cytoreductive drugs have been shown to be effective, but have rarely resulted in complete or long-term remission. We report two patients with advanced systemic mastocytosis (SM) who were treated successfully with thalidomide, given at the maximal tolerated dosage. B and C-findings as well as clinical symptoms rapidly improved. After a follow-up of more than 1 year, the patients remained in partial remission. Thalidomide seems to be an active drug in advanced SM. However, clinical trials are warranted to define its efficacy and safety profiles.
authors.affiliation: Department of Haematology, Centre Hospitalier Universitaire, Amiens, France. damaj.gandhi@chu-amiens.fr
authors.forename: Gandhi
authors.initials: G
authors.lastname: Damaj
authors.forename: Emmanuelle
authors.initials: E
authors.lastname: Bernit
authors.forename: David
authors.initials: D
authors.lastname: Ghez
authors.forename: Jean-François
authors.initials: JF
authors.lastname: Claisse
authors.forename: Nicolas
authors.initials: N
authors.lastname: Schleinitz
authors.forename: Jean-Robert
authors.initials: JR
authors.lastname: Harlé
authors.forename: Danielle
authors.initials: D
authors.lastname: Canioni
authors.forename: Olivier
authors.initials: O
authors.lastname: Hermine
mesh_terms: D000368:Aged; D020533:Angiogenesis Inhibitors / Q000627:therapeutic use; D001854:Bone Marrow Cells / Q000473:pathology; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008297:Male; D034721:Mastocytosis, Systemic / Q000188:drug therapy* / Q000473:pathology; D008875:Middle Aged; D013792:Thalidomide / Q000627:therapeutic use*
chemical_list: D020533:Angiogenesis Inhibitors; D007166:Immunosuppressive Agents; D013792:Thalidomide