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Thalidomide in systemic mastocytosis: results from an open‐label, multicentre, phase II study
Author(s) -
Gruson Bérengère,
Lortholary Olivier,
Canioni Danielle,
Chandesris Olivia,
Lanternier Fanny,
Bruneau Julie,
Grosbois Bernard,
Livideanu Cristina,
Larroche Claire,
Durieu Isabelle,
Barete Stéphane,
Sevestre Henri,
Diouf Momar,
Chaby Guillaume,
Marolleau JeanPierre,
Dubreuil Patrice,
Hermine Olivier,
Damaj Gandhi
Publication year - 2013
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.12265
Subject(s) - systemic mastocytosis , medicine , thalidomide , neutropenia , purine analogue , gastroenterology , quality of life (healthcare) , mast cell , phases of clinical research , bone marrow , surgery , chemotherapy , immunology , purine , biochemistry , chemistry , nursing , multiple myeloma , enzyme
Summary 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 ( A ssociation F rançaise pour les I nitiatives de R echerche sur le M astocyte et L es M astocytoses), Q uality of L ife or H amilton 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.