Hypereosinophilic syndrome: A multicenter, retrospective analysis of clinical characteristics and response to therapy - 12/08/11
Reprint requests: Amy D. Klion, MD, Bldg 50, Rm 6351, Laboratory of Parasitic Diseases, NIAID, National Institutes of Health, Bethesda, MD 20892.Abstract |
Background |
Hypereosinophilic syndrome (HES) is a heterogeneous group of rare disorders defined by persistent blood eosinophilia ≥1.5 × 109/L, absence of a secondary cause, and evidence of eosinophil-associated pathology. With the exception of a recent multicenter trial of mepolizumab (anti–IL-5 mAb), published therapeutic experience has been restricted to case reports and small case series.
Objective |
The purpose of the study was to collect and summarize baseline demographic, clinical, and laboratory characteristics in a large, diverse cohort of patients with HES and to review responses to treatment with conventional and novel therapies.
Methods |
Clinical and laboratory data from 188 patients with HES, seen between January 2001 and December 2006 at 11 institutions in the United States and Europe, were collected retrospectively by chart review.
Results |
Eighteen of 161 patients (11%) tested were Fip1-like 1–platelet-derived growth factor receptor ⍺ (FIP1L1-PDGFRA) mutation—positive, and 29 of 168 patients tested (17%) had a demonstrable aberrant or clonal T-cell population. Corticosteroid monotherapy induced complete or partial responses at 1 month in 85% (120/141) of patients with most remaining on maintenance doses (median, 10 mg prednisone equivalent daily for 2 months to 20 years). Hydroxyurea and IFN-⍺ (used in 64 and 46 patients, respectively) were also effective, but their use was limited by toxicity. Imatinib (used in 68 patients) was more effective in patients with the FIP1L1-PDGFRA mutation (88%) than in those without (23%; P < .001).
Conclusion |
This study, the largest clinical analysis of patients with HES to date, not only provides useful information for clinicians but also should stimulate prospective trials to optimize treatment of HES.
El texto completo de este artículo está disponible en PDF.Key words : Eosinophil, hypereosinophilic syndrome, FIP1L1-PDGFRA
Abbreviations used : CEL, FP, GM, HES, L-HES, TARC, TCR
Esquema
| Supported by the Division of Intramural Research of the NIAID/NIH (A.D.K., P.U.O., T.B.N.), grants AI41472 and AI72265 from the NIH (B.S.B.), grant AI061097 from the NIH (G.J.G.), the Human Immunology grant program of the Dana Foundation (B.S.B.), the Swiss National Science Foundation (H.-U.S.), the Belgian National Fund for Scientific Research (F.R.), and the Campaign Urging Research for Eosinophilic Disorders (M.E.R.). B.S.B. is a Cosner Scholar in Translational Research from Johns Hopkins University. The funding organizations had no role in the design and conduct of the study; collection, management, analysis, and interpretation of the data; and preparation or review of the manuscript. The manuscript was approved by the Division of Intramural Research, NIAID/NIH. |
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| Disclosure of potential conflict of interest: G. J. Gleich and K. M. Leiferman have equity in Ception, have received research support from GlaxoSmithKline, and are on the advisory board for APFE. J. E. Kahn has received consulting fees and research support from GlaxoSmithKline. T. B. Nutman is a stockholder in Johnson & Johnson and is employed by the National Institutes of Health. J. Ring has received research support from Novartis, Schering-Plough, Fujisawa, GlaxoSmithKline, Bencard, Stallergenes, ALK-Abelló, Allergopharma, Pharmacia, DPC Biermann, Aventis, Almirall, Leo, Galderma, and Switch Biotech. M. E. Rothenberg is a speaker and consultant for Merck; is a consultant for Ception Therapeutics, Novartis, Nycomed, and Centocor; has received research support from the National Institutes of Health, FAAN, and the Dana Foundation; is on the Medical Advisory Board for APFED; and is on the Executive Council for the International Eosinophil Society. F. Roufosse has received consulting fees from GlaxoSmithKline. J. Sheikh is on the speakers’ bureau for Alcon, Meda, Sanofi-Aventis, and UBC; is a consultant for and is on the Advisory Board for Zeer.com; has received research support from GlaxoSmithKline; has been a legal consultant on the topics of allergy/immunology medical malpractice and latex allergy; is a member of the ACAAI; and is on the Executive Board of the Massachusetts Allergy Society (Secretary) and the New England Society of Allergy (CME Director). H.-U. Simon has received consulting fees from Pfizer, has received honoraria from Merck, and has received research support from the Swiss National Science Foundation, GlaxoSmithKline, and AstraZeneca. A. Wardlaw has received honoraria and research support from GlaxoSmithKline. P. F. Weller has served as a consultant for GlaxoSmithKline and has received research support from Merck. The rest of the authors have declared that they have no conflict of interest. |
Vol 124 - N° 6
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