Abstract | PURPOSE OF REVIEW: RECENT FINDINGS: The study enrolled 103 patients (48 men and 55 women), including 47 currently adult patients. All of these patients were treated with G-CSF, starting at a median age of 3.8 years (range 0.04-33.9 years) with a median dose of 3.0 mcg/kg/day (range 0.01-93.1 mcg/kg/day) for a median of 10.3 years (range 0.01-29.3 years). Neutrophils increased in response to G-CSF in all patients (median values before G-CSF 0.2 × 10/l, on G-CSF 1.20 x 10/l). Treatment increased spleen size (before G-CSF, 47%, on treatment on G-CSF 76%), and splenomegaly was the dose-limiting adverse effect of treatment ( pain and early satiety). Clinical observations and records attest to reduce frequency of infectious events and the severity of inflammatory bowel symptoms, but fever and recurrent infections remain a significant problem. In the cohort of patients followed carefully through the Severe Chronic Neutropenia International Registry, four patients have developed myelodysplasia or acute myeloid leukemia and we are aware of four other cases, (altogether seven on G-CSF, one never treated with G-CSF). Liver transplantation in five patients did not correct neutropenia. Four patients had hematopoietic stem cell transplantation; two adults and two children were transplanted; one adult and one child survived. SUMMARY:
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Authors | David C Dale, Audrey Anna Bolyard, Tracy Marrero, Merideth L Kelley, Vahagn Makaryan, Emily Tran, Jamie Leung, Laurence A Boxer, Priya S Kishnani, Stephanie Austin, Corbinian Wanner, Iris A Ferrecchia, Dina Khalaf, Dawn Maze, Joanne Kurtzberg, Cornelia Zeidler, Karl Welte, David A Weinstein |
Journal | Current opinion in hematology
(Curr Opin Hematol)
Vol. 26
Issue 1
Pg. 16-21
(01 2019)
ISSN: 1531-7048 [Electronic] United States |
PMID | 30451720
(Publication Type: Clinical Trial, Journal Article, Multicenter Study, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't)
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Chemical References |
- Granulocyte Colony-Stimulating Factor
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Topics |
- Adolescent
- Adult
- Child
- Child, Preschool
- Disease-Free Survival
- Female
- Glycogen Storage Disease Type I
(blood, drug therapy, mortality, pathology)
- Granulocyte Colony-Stimulating Factor
(administration & dosage)
- Humans
- Male
- Middle Aged
- Neutropenia
(blood, drug therapy, mortality, pathology)
- Neutrophils
(metabolism, pathology)
- Registries
- Survival Rate
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