Article - June 2009 Pediatric Mastocytosis study

Pediatric-onset mastocytosis: A long term clinical follow-up and correlation with bone marrow histopathology
Link to Journal Article

Contributors:  Ashraf Uzzaman, MD 1, Irina Maric, MD 2, Pierre Noel, MD 2, Brett V. Kettelhut, MD 3, Dean D. Metcalfe, MD 1, Melody C. Carter, MD 1 * 1Laboratory of Allergic Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland 2Hematology Section, Department of Laboratory Medicine, Warren G. Magnuson Clinical Center, National Institutes of Health, Bethesda, Maryland 3Department of Pediatrics, University of Nebraska Medical Center, Omaha, Nebraska

email: Melody C. Carter (mcarter@niaid.nih.gov)


 * Correspondence to Melody C. Carter, Bldg. 10, Room 11C213, 10 Center Drive MSC1881, Bethesda, MD 20892-1881.

Ashraf Uzzaman-performed research, collected data, and wrote the manuscript. Irina Maric-performed research, analyzed and interpreted data, and wrote the manuscript. Pierre Noel-analyzed and interpreted data, Brett V. Kettelhut-performed research and collected data, Dean D. Metcalfe-designed research, analyzed and interpreted data, and wrote the manuscript, Melody C. Carter-designed research, performed research, collected data, analyzed and interpreted data, and wrote the manuscript.

Ashraf Uzzaman and Irina Maric contributed equally to this work.

Funded by: NIH

Keywords bone marrow • cutaneous mastocytosis • KIT • pediatric mastocytosis • serum tryptase • urticaria pigmentosa

Abstract

Background Pediatric onset mastocytosis usually presents as urticaria pigmentosa; and less often as diffuse cutaneous mastocytosis. While the literature indicates that disease often resolves, there has been a move to more aggressive therapy for mastocytosis early in life. We addressed the long term prognosis of pediatric-onset disease by examining 17 children with mastocytosis which we had reported on in 1989 [1].

Procedure We successfully contacted 15 of these patients and data was collected regarding their clinical status. Original bone marrow specimens were re-stained, re-examined, and correlated with disease outcome using consensus criteria. Three of five patients with persistent disease underwent repeat bone marrow biopsies.

Results There was complete regression of disease as defined by cutaneous findings and symptoms (clinical disease severity) in 10 of 15 patients (67%). Three patients had major (20%) and two had partial regression of disease (13%). Repeat marrow examinations on three patients with persistent disease documented systemic mastocytosis based on marrow findings in one patient who had partial regression of disease and was the only patient with initial morphologic evidence of systemic disease. Of the remaining two patients, one demonstrated partial regression and the other major regression of disease; and neither had evidence of systemic mastocytosis.

Conclusion This study demonstrates that initial bone marrow biopsies were prognostic in that those without evidence of systemic disease experienced disease regression; and that the long term prognosis for children managed symptomatically with mastocytosis is highly encouraging. Pediatr Blood Cancer.

© 2009 Wiley-Liss, Inc.

Citation:

AU: Ashraf Uzzaman, Irina Maric, Pierre Noel, Brett V. Kettelhut, Dean D. Metcalfe, Melody C. Carter TI: Pediatric-onset mastocytosis: A long term clinical follow-up and correlation with bone marrow histopathology SO: Pediatric Blood & Cancer VL: 9999 NO: 9999 PG: n/a YR: 2009 CP: Copyright © 2009 Wiley-Liss, Inc. ON: 1545-5017 PN: 1545-5009 AD: Laboratory of Allergic Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland; Hematology Section, Department of Laboratory Medicine, Warren G. Magnuson Clinical Center, National Institutes of Health, Bethesda, Maryland; Department of Pediatrics, University of Nebraska Medical Center, Omaha, Nebraska DOI: 10.1002/pbc.22125 US: http://dx.doi.org/10.1002/pbc.22125