Hypereosinophilic syndrome: A multicenter, retrospective analysis of clinical characteristics and response to therapy

• Published in: Journal of allergy and clinical immunology Vol. 124; no. 6; pp. 1319 - 1325.e3
• Main Authors: Ogbogu, Princess U., MD, Bochner, Bruce S., MD, Butterfield, Joseph H., MD, Gleich, Gerald J., MD, Huss-Marp, Johannes, MD, Kahn, Jean Emmanuel, MD, Leiferman, Kristin M., MD, Nutman, Thomas B., MD, Pfab, Florian, MD, Ring, Johannes, MD, Rothenberg, Marc E., MD, PhD, Roufosse, Florence, MD, Sajous, Marie-Helene, MD, Sheikh, Javed, MD, Simon, Dagmar, MD, Simon, Hans-Uwe, MD, PhD, Stein, Miguel L., MD, Wardlaw, Andrew, MD, Weller, Peter F., MD, Klion, Amy D., MD
• Format: Journal Article
• Language: English
• Published: New York, NY Mosby, Inc 01.12.2009; Elsevier; Elsevier Limited
• Subjects: Adolescent; Adrenal Cortex Hormones - administration & dosage; Adrenal Cortex Hormones - therapeutic use; Adult; Aged; Aged, 80 and over; Allergy and Immunology; Antibodies, Monoclonal - administration & dosage; Antibodies, Monoclonal - therapeutic use; Antibodies, Monoclonal, Humanized; Benzamides; Biological and medical sciences; Chemokine CCL17 - blood; Chemokines; Child; Colleges & universities; Cyclosporine - administration & dosage; Cyclosporine - therapeutic use; Drug therapy; Drug Therapy, Combination; Eosinophil; Eosinophils - drug effects; Eosinophils - immunology; Eosinophils - metabolism; Female; FIP1L1-PDGFRA; Flow cytometry; Fundamental and applied biological sciences. Psychology; Fundamental immunology; Hematologic and hematopoietic diseases; Humans; Hydroxyurea - administration & dosage; Hydroxyurea - therapeutic use; hypereosinophilic syndrome; Hypereosinophilic Syndrome - drug therapy; Hypereosinophilic Syndrome - immunology; Hypereosinophilic Syndrome - metabolism; Imatinib Mesylate; Immunopathology; Interferon-alpha - administration & dosage; Interferon-alpha - therapeutic use; Interleukin-5 - blood; Ligands; Light therapy; Male; Medical sciences; Middle Aged; mRNA Cleavage and Polyadenylation Factors - immunology; mRNA Cleavage and Polyadenylation Factors - metabolism; Oncogene Proteins, Fusion - immunology; Oncogene Proteins, Fusion - metabolism; Other diseases. Hematologic involvement in other diseases; Parasitic diseases; Piperazines - administration & dosage; Piperazines - therapeutic use; Pyrimidines - administration & dosage; Pyrimidines - therapeutic use; Receptor, Platelet-Derived Growth Factor alpha - immunology; Receptor, Platelet-Derived Growth Factor alpha - metabolism; Retrospective Studies; Sarcoidosis. Granulomatous diseases of unproved etiology. Connective tissue diseases. Elastic tissue diseases. Vasculitis; Tryptases - blood; Young Adult; TCR; Fip1-like 1–platelet-derived growth factor receptor α; GM; FP; CEL; Eosinophil; FIP1L1-PDGFRA; T-cell receptor rearrangement PCR; L-HES; Chronic eosinophilic leukemia; Lymphocytic variant hypereosinophilic syndrome; hypereosinophilic syndrome; Geometric mean; Thymus and activation-related chemokine; HES; TARC; Immunopathology; Symptomatology; Platelet derived growth factor receptor A; Immunology; Treatment; Multicenter study; Granulocyte; Hemopathy; Retrospective; Hypereosinophilic syndrome
• ISSN: 0091-6749; 1097-6825
• DOI: 10.1016/j.jaci.2009.09.022

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 α ( 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-α (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.