title: Thalidomide in systemic mastocytosis: results from an open-label, multicentre, phase II study.
abstract: Mastocytosis can lead to organ failure as well as systemic symptoms that can be disabling, with considerable deterioration in quality of life. Beside symptomatic treatments, interferon-α and purine analogues have been shown to be effective but complete or long-term remission is rarely obtained with these drugs. We conducted a phase II, multicentre, study to investigate thalidomide in severely symptomatic indolent and aggressive systemic mastocytosis. Twenty patients were enrolled of whom 16 were analysed for response. The overall response rate was 56%. Responses were observed in the skin in 61% of patients with a significant decrease in the pruritus score. Mast cell mediator-related symptoms responded in 71% of cases and 25% of aggressive systemic mastocytosis patients had a response in terms of B/C findings (borderline/cytoreduction needed). Bone marrow mast cell infiltration decreased in five of the eight evaluable patients. There was no significant improvement in the AFIRMM (Association Française pour les Initiatives de Recherche sur le Mastocyte et Les Mastocytoses), Quality of Life or Hamilton scores. Grade 3-4 toxicities consisted of peripheral neuropathy (11%) and myelosuppression (neutropenia: 5%; thrombocytopenia: 11%). In conclusion, thalidomide might be useful in mastocytosis and in the treatment of mast cell-related symptoms. It might be considered in selected patients, taking into account the benefit/risk balance and the individual patient evaluation.
authors: Département d'Hématologie, Centre Hospitalier Universitaire (CHU) d'Amiens, Amiens, France.
authors: Bérengère
authors: B
authors: Gruson
authors: Olivier
authors: O
authors: Lortholary
authors: Danielle
authors: D
authors: Canioni
authors: Olivia
authors: O
authors: Chandesris
authors: Fanny
authors: F
authors: Lanternier
authors: Julie
authors: J
authors: Bruneau
authors: Bernard
authors: B
authors: Grosbois
authors: Cristina
authors: C
authors: Livideanu
authors: Claire
authors: C
authors: Larroche
authors: Isabelle
authors: I
authors: Durieu
authors: Stéphane
authors: S
authors: Barete
authors: Henri
authors: H
authors: Sevestre
authors: Momar
authors: M
authors: Diouf
authors: Guillaume
authors: G
authors: Chaby
authors: Jean-Pierre
authors: JP
authors: Marolleau
authors: Patrice
authors: P
authors: Dubreuil
authors: Olivier
authors: O
authors: Hermine
authors: Gandhi
authors: G
authors: Damaj
mesh_terms: D000328:Adult; D000368:Aged; D001853:Bone Marrow / Q000473:pathology; D005221:Fatigue / Q000139:chemically induced; D005260:Female; D005334:Fever / Q000139:chemically induced; D005767:Gastrointestinal Diseases / Q000139:chemically induced; D006402:Hematologic Diseases / Q000139:chemically induced; D006529:Hepatomegaly / Q000209:etiology; D006801:Humans; D008297:Male; D008407:Mast Cells / Q000473:pathology; D034721:Mastocytosis, Systemic / Q000150:complications / Q000188:drug therapy* / Q000473:pathology; D008875:Middle Aged; D009422:Nervous System Diseases / Q000139:chemically induced; D011446:Prospective Studies; D011537:Pruritus / Q000188:drug therapy / Q000209:etiology; D011788:Quality of Life; D012074:Remission Induction; D012720:Severity of Illness Index; D012867:Skin / Q000473:pathology; D013163:Splenomegaly / Q000209:etiology; D013792:Thalidomide / Q000009:adverse effects / Q000627:therapeutic use*
chemical_list: D013792:Thalidomide