title: Treatment of adult systemic mastocytosis with interferon-alpha: results of a multicentre phase II trial on 20 patients.
abstract: Systemic mastocytosis (SM) is characterized by proliferation of mast cells in various organs, which may release a wide variety of mediators, thereby explaining the broad clinical spectrum of disease manifestations. The potentially life-threatening systemic symptoms and tumoral proliferation are poorly controlled despite the use of several cytotoxic chemotherapies and/or symptomatic treatments. Twenty consecutive adult SM patients with histologically confirmed bone marrow (BM) involvement received interferon-alpha subcutaneously (1-5 million units/m2/d, with progressive dose intensification over the first month of treatment) and were evaluated after 6 months of therapy. Seven of them had previously received symptomatic treatments, including steroids, which were ineffective. Among the 13 patients treated for at least 6 months, seven partial and six minor responses, mainly concerning vascular congestion and skin lesions, were obtained, while BM infiltration remained unchanged in 12 patients. The significant reduction of mast-cell mediator levels after 6 months of treatment was not predictive of clinical remission. The rate of depression was unexpectedly high (seven patients; 35%). Two patients died soon after starting therapy (one myocardial infarction, one septic shock). Six months of interferon-alpha may relieve vascular congestion in adults with SM, probably by inhibiting mast-cell degranulation.
authors.affiliation: Service d'Hématologie Clinique, Hôpital Avicenne, Université Paris-Nord, Bobigny, Paris, France. philippe.casassus@avc.ap-hop-paris.fr
authors.forename: Philippe
authors.initials: P
authors.lastname: Casassus
authors.forename: Nadine
authors.initials: N
authors.lastname: Caillat-Vigneron
authors.forename: Antoine
authors.initials: A
authors.lastname: Martin
authors.forename: Jeanne
authors.initials: J
authors.lastname: Simon
authors.forename: Valérie
authors.initials: V
authors.lastname: Gallais
authors.forename: Patrice
authors.initials: P
authors.lastname: Beaudry
authors.forename: Virginie
authors.initials: V
authors.lastname: Eclache
authors.forename: Liliane
authors.initials: L
authors.lastname: Laroche
authors.forename: Pierre
authors.initials: P
authors.lastname: Lortholary
authors.forename: Martine
authors.initials: M
authors.lastname: Raphaël
authors.forename: Loïc
authors.initials: L
authors.lastname: Guillevin
authors.forename: Olivier
authors.initials: O
authors.lastname: Lortholary
mesh_terms: D000328:Adult; D000368:Aged; D001853:Bone Marrow / Q000473:pathology; D004334:Drug Administration Schedule; D005260:Female; D005500:Follow-Up Studies; D006632:Histamine / Q000378:metabolism; D006801:Humans; D007093:Imidazoles / Q000652:urine; D000077190:Interferon alpha-2; D016898:Interferon-alpha / Q000009:adverse effects / Q000627:therapeutic use*; D008297:Male; D008407:Mast Cells / Q000378:metabolism; D034721:Mastocytosis, Systemic / Q000188:drug therapy* / Q000378:metabolism / Q000473:pathology; D008875:Middle Aged; D011446:Prospective Studies; D011994:Recombinant Proteins; D012697:Serine Endopeptidases / Q000097:blood; D016896:Treatment Outcome; D053802:Tryptases
chemical_list: D007093:Imidazoles; D000077190:Interferon alpha-2; D016898:Interferon-alpha; D011994:Recombinant Proteins; C002667:methylimidazoleacetic acid; D006632:Histamine; D012697:Serine Endopeptidases; D053802:Tryptases