title: Pentostatin for treatment of refractory autoimmune lymphoproliferative syndrome.
abstract: Autoimmune lymphoproliferative syndrome (ALPS), a disorder of programmed cell death, could be due to a congenital defect in the Fas signaling pathway or other pathways for apoptosis. Most cases present with lymphoproliferation and certain autoimmune features such as thrombocytopenia, neutropenia, and anemia are due to excessive production of antibodies by B lymphocytes. Majority of cases present within the first few years of life. We report a case of ALPS presenting at birth which was refractory to splenectomy and immunosuppressive therapy, but responded to pentostatin followed by hematopoietic stem cell transplantation (HSCT).
authors.affiliation: Department of Hematology/Oncology/BMT, Nationwide Children's Hospital, Columbus, Ohio 43205, USA. rajinder.bajwa@NationwideChildrens.org
authors.forename: Rajinder
authors.initials: R
authors.lastname: Bajwa
authors.forename: Stephanie
authors.initials: S
authors.lastname: Savelli
authors.forename: Thomas
authors.initials: T
authors.lastname: Gross
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use*; D056735:Autoimmune Lymphoproliferative Syndrome / Q000188:drug therapy*; D003131:Combined Modality Therapy; D004200:Diseases in Twins / Q000188:drug therapy*; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007231:Infant, Newborn; D008297:Male; D015649:Pentostatin / Q000627:therapeutic use*
chemical_list: D000970:Antineoplastic Agents; D015649:Pentostatin