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

Princess U. Ogbogu, Bruce S. Bochner, Joseph H. Butterfield, Gerald J. Gleich, Johannes Huss-Marp, Jean Emmanuel Kahn, Kristin M. Leiferman, Thomas B. Nutman, Florian Pfab, Johannes Ring, Marc E. Rothenberg, Florence Roufosse, Marie Helene Sajous, Javed Sheikh, Dagmar Simon, Hans Uwe Simon, Miguel L. Stein, Andrew Wardlaw, Peter F. Weller, Amy D. Klion

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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 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.

Original languageEnglish (US)
JournalJournal of Allergy and Clinical Immunology
Volume124
Issue number6
DOIs
StatePublished - Dec 2009

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Hypereosinophilic Syndrome
Therapeutics
Platelet-Derived Growth Factor Receptors
Mutation
Hydroxyurea
Interleukin-5
Eosinophilia
Prednisone
Eosinophils
Multicenter Studies
Adrenal Cortex Hormones
Demography
Pathology
T-Lymphocytes

ASJC Scopus subject areas

  • Immunology and Allergy
  • Immunology

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Hypereosinophilic syndrome : A multicenter, retrospective analysis of clinical characteristics and response to therapy. / Ogbogu, Princess U.; Bochner, Bruce S.; Butterfield, Joseph H.; Gleich, Gerald J.; Huss-Marp, Johannes; Kahn, Jean Emmanuel; Leiferman, Kristin M.; Nutman, Thomas B.; Pfab, Florian; Ring, Johannes; Rothenberg, Marc E.; Roufosse, Florence; Sajous, Marie Helene; Sheikh, Javed; Simon, Dagmar; Simon, Hans Uwe; Stein, Miguel L.; Wardlaw, Andrew; Weller, Peter F.; Klion, Amy D.

In: Journal of Allergy and Clinical Immunology, Vol. 124, No. 6, 12.2009.

Research output: Contribution to journalArticle

Ogbogu, PU, Bochner, BS, Butterfield, JH, Gleich, GJ, Huss-Marp, J, Kahn, JE, Leiferman, KM, Nutman, TB, Pfab, F, Ring, J, Rothenberg, ME, Roufosse, F, Sajous, MH, Sheikh, J, Simon, D, Simon, HU, Stein, ML, Wardlaw, A, Weller, PF & Klion, AD 2009, 'Hypereosinophilic syndrome: A multicenter, retrospective analysis of clinical characteristics and response to therapy', Journal of Allergy and Clinical Immunology, vol. 124, no. 6. https://doi.org/10.1016/j.jaci.2009.09.022
Ogbogu, Princess U. ; Bochner, Bruce S. ; Butterfield, Joseph H. ; Gleich, Gerald J. ; Huss-Marp, Johannes ; Kahn, Jean Emmanuel ; Leiferman, Kristin M. ; Nutman, Thomas B. ; Pfab, Florian ; Ring, Johannes ; Rothenberg, Marc E. ; Roufosse, Florence ; Sajous, Marie Helene ; Sheikh, Javed ; Simon, Dagmar ; Simon, Hans Uwe ; Stein, Miguel L. ; Wardlaw, Andrew ; Weller, Peter F. ; Klion, Amy D. / Hypereosinophilic syndrome : A multicenter, retrospective analysis of clinical characteristics and response to therapy. In: Journal of Allergy and Clinical Immunology. 2009 ; Vol. 124, No. 6.
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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 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.",
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T2 - A multicenter, retrospective analysis of clinical characteristics and response to therapy

AU - Ogbogu, Princess U.

AU - Bochner, Bruce S.

AU - Butterfield, Joseph H.

AU - Gleich, Gerald J.

AU - Huss-Marp, Johannes

AU - Kahn, Jean Emmanuel

AU - Leiferman, Kristin M.

AU - Nutman, Thomas B.

AU - Pfab, Florian

AU - Ring, Johannes

AU - Rothenberg, Marc E.

AU - Roufosse, Florence

AU - Sajous, Marie Helene

AU - Sheikh, Javed

AU - Simon, Dagmar

AU - Simon, Hans Uwe

AU - Stein, Miguel L.

AU - Wardlaw, Andrew

AU - Weller, Peter F.

AU - Klion, Amy D.

PY - 2009/12

Y1 - 2009/12

N2 - 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.

AB - 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.

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