Abstract | BACKGROUND: ALPS is a disorder of apoptosis resulting in accumulation of autoreactive lymphocytes, leading to marked lymphadenopathy, hepatosplenomegaly, and multilineage cytopenias due to splenic sequestration and/or autoimmune destruction often presenting in childhood. We summarize our experience of rituximab use during the last 8 years in 12 patients, 9 children, and 3 adults, out of 259 individuals with ALPS, belonging to 166 families currently enrolled in studies at the National Institutes of Health. METHODS: RESULTS: CONCLUSION: Toxicities including hypogammaglobulinemia and neutropenia constitute an additional infection risk burden, especially in asplenic individuals, and may warrant avoidance of rituximab until other immunosuppressive medication options are exhausted. Long-term follow-up of ALPS patients with cytopenias after any treatment is necessary to determine relative risks and benefits.
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Authors | V Koneti Rao, Susan Price, Katie Perkins, Patricia Aldridge, Jean Tretler, Joie Davis, Janet K Dale, Fred Gill, Kip R Hartman, Linda C Stork, David J Gnarra, Lakshmanan Krishnamurti, Peter E Newburger, Jennifer Puck, Thomas Fleisher |
Journal | Pediatric blood & cancer
(Pediatr Blood Cancer)
Vol. 52
Issue 7
Pg. 847-52
(Jul 2009)
ISSN: 1545-5017 [Electronic] United States |
PMID | 19214977
(Publication Type: Journal Article, Research Support, N.I.H., Extramural, Research Support, N.I.H., Intramural)
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Copyright | (c) 2009 Wiley-Liss, Inc. |
Chemical References |
- Antibodies, Monoclonal
- Antibodies, Monoclonal, Murine-Derived
- Immunosuppressive Agents
- Rituximab
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Topics |
- Adolescent
- Adult
- Anemia, Hemolytic, Autoimmune
(drug therapy, immunology)
- Antibodies, Monoclonal
(therapeutic use)
- Antibodies, Monoclonal, Murine-Derived
- Autoimmune Diseases
(drug therapy, immunology)
- Child
- Child, Preschool
- Drug Resistance, Neoplasm
- Humans
- Immunosuppressive Agents
(therapeutic use)
- Infant
- Lymphoproliferative Disorders
(drug therapy, immunology)
- Middle Aged
- Rituximab
- Thrombocytopenia
(drug therapy)
- Young Adult
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