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Blood. 2015 Aug 20;126(8):1009-16; quiz 1050. doi: 10.1182/blood-2014-12-614743. Epub 2015 May 22.

Long-term efficacy and safety of cladribine (2-CdA) in adult patients with mastocytosis.

Author information

1
Sorbonne Universités, UPMC Univ Paris VI, Assistance Publique-Hôpitaux de Paris, Département de Dermatologie et Allergologie, Hôpital Tenon, Centre de Référence des Mastocytoses, Paris, France;
2
Université Sorbonne-Paris-Cité, Paris V, Assistance Publique-Hôpitaux de Paris, Service de Maladies Infectieuses et Tropicales, Centre de Référence des Mastocytoses, Institut Imagine, Hôpital Necker Enfants Malades, Centre d'Infectiologie Necker-Pasteur, Paris, France;
3
Service d'Hématologie, Centre Hospitalier Universitaire (CHU) et Faculté de Médecine, Caen, France;
4
Centre de Référence des Mastocytoses, Hôpital Necker Enfants Malades, Paris V, IFR Necker, Paris, France;
5
Centre de Référence des Mastocytoses, Hôpital Necker Enfants Malades, Paris V, IFR Necker, Paris, France; Service d'Hématologie, Faculté de Médecine Paris V, Université Sorbonne Paris-Cité et Assistance Publique-Hôpitaux de Paris, Hôpital Necker, Paris, France;
6
Unité de Recherche Clinique, Hôpital Necker Paris, France;
7
Service Médecine Interne, CHU de Nantes, France;
8
Service de Médecine Interne, Centre Hospitalier Lyon Sud, Pierre Bénite, France;
9
Service d'Hématologie, Faculté de Médecine Paris V, Université Sorbonne Paris-Cité et Assistance Publique-Hôpitaux de Paris, Hôpital Necker, Paris, France;
10
Service de Médecine Interne, Hôpital Sud, Rennes, France;
11
Service de Médecine Interne, CHU Henri Mondor, Créteil, France;
12
Service d'Hématologie et Thérapie Cellulaire, UMR CNRS 7292, Centre Hospitalier Universitaire, Tours, France;
13
Service de Médecine Interne, CHU Avicenne, Bobigny, France;
14
Service de Dermatologie, CHU de Poitiers, France;
15
Service de Médecine Interne, Hôpital Foch, Suresnes, France;
16
Service d'Hématologie, Hôpital Avicenne, Bobigny, France;
17
Sorbonne Universités, UPMC Univ Paris VI, Service de Médecine Interne, Hôpital Tenon, Paris France;
18
Centre de Référence des Mastocytoses, Hôpital Necker Enfants Malades, Paris V, IFR Necker, Paris, France; Université Sorbonne-Paris-Cité, Paris V, Assistance Publique-Hôpitaux de Paris, (INSERM) U1151 Laboratoire d'Onco-Hématologie, Hôpital Necker, Paris, France;
19
Centre de Référence des Mastocytoses, Hôpital Necker Enfants Malades, Paris V, IFR Necker, Paris, France; Université Sorbonne-Paris-Cité, Paris V, Assistance Publique-Hôpitaux de Paris, Service d'Anatomie-Pathologie, Hôpital Necker, Paris, France;
20
CRCM, Inserm, U1068, Institut Paoli-Calmettes, Aix-Marseille Université, UM105, CNRS, UMR7258, F-13009, Marseille, France;
21
Centre de Référence des Mastocytoses, Hôpital Necker Enfants Malades, Paris V, IFR Necker, Paris, France; Service d'Hématologie, Faculté de Médecine Paris V, Université Sorbonne Paris-Cité et Assistance Publique-Hôpitaux de Paris, Hôpital Necker, Paris, France; Institut Imagine, Université Sorbonne Paris-Cité et Assistance Publique-Hôpitaux de Paris, Hôpital Necker, Paris, France; and Laboratoire "Cellular and Molecular Basis of Hematological Disorders and Therapeutic Implications," INSERM U1163, CNRS ERL 8254, Paris, France.

Abstract

Mastocytosis (M) is a clonal myeloid-disabling disorder for which no curative therapy is currently available. Cladribine (2-chlorodeoxyadenosine [2-CdA]) is a synthetic purine analog cytoreductive treatment, for which efficacy is mostly reported in advanced M. Here we report, with a long-term follow-up period (>10 years) efficacy and safety in 68 adult patients with M (36 [53%] had indolent M and 32 [47%] had advanced M) treated by 2-CdA (0.14 mg/kg in infusion or subcutaneously, days 1-5; repeated at 4-12 weeks until 1 to 9 courses). Median 2-CdA courses number was 3.7 (1-9). The overall response rate was 72% (complete remission [R]/major/partial R: 0%/47%/25%) and according to indolent/advanced M was 92% (major/partial R: 56%/36%) and 50% (major/partial R: 37.5%/12.5%), respectively. Clinical improvement was observed for 10 of 11 mediator release and 6 of 7 mast cell infiltration-related symptoms including urticaria pigmentosa and organomegaly (P < .02). Serum tryptase levels decreased (P = .01). Median durations of response were 3.71 (0.1-8) and 2.47 (0.5-8.6) years for indolent and aggressive M, respectively. The most frequent grade 3/4 toxicities were lymphopenia (82%), neutropenia (47%), and opportunistic infections (13%). 2-CdA appears to provide a significant efficacy with some toxicity in various M subtypes, mostly in indolent M, refractory to multiple symptomatic therapies.

PMID:
26002962
DOI:
10.1182/blood-2014-12-614743
[Indexed for MEDLINE]
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