title: Sirolimus as maintenance treatment in an infant with life-threatening multiresistant pure red cell anemia/autoimmune hemolytic anemia.
abstract: A 9-month-old boy with life-threatening multiresistant pure red cell anemia/autoimmune hemolytic anemia within the frame of a possible, undiagnosed immune-mediated disease was initially treated with prednisone. Further-line therapies of the following 7 relapses included immunoglobulins, rituximab, cyclophosphamide, and alentuzumab followed by other maintenance treatments as cyclosporine, methotrexate, and mycophenolate. After all the administered therapies failed, the patient was successfully treated by splenectomy followed by fludarabine and then sirolimus as maintenance treatment. Relapses might have been caused by the lack of a complete debulking of triggering cells and/or ineffective maintenance therapy. Splenectomy and sirolimus may have played a complementary role in the management of both situations.
authors.affiliation: *Haematology Unit ‡Intensive Care Unit, G. Gaslini Children's Hospital, Genoa †Haematology Unit, Pausilipon Hospital §Department of Pediatrics, Second University of Naples, Naples, Italy ¶Immunology and Pediatric Hematology Department, Necker Children's Hospital, Paris, France.
authors.forename: Maurizio
authors.initials: M
authors.lastname: Miano
authors.forename: Vincenzo
authors.initials: V
authors.lastname: Poggi
authors.forename: Laura
authors.initials: L
authors.lastname: Banov
authors.forename: Francesca
authors.initials: F
authors.lastname: Fioredda
authors.forename: Concetta
authors.initials: C
authors.lastname: Micalizzi
authors.forename: Johanna
authors.initials: J
authors.lastname: Svahn
authors.forename: Giovanni
authors.initials: G
authors.lastname: Montobbio
authors.forename: Federica
authors.initials: F
authors.lastname: Gallicola
authors.forename: Angelo C
authors.initials: AC
authors.lastname: Molinari
authors.forename: Rosanna
authors.initials: R
authors.lastname: Parasole
authors.forename: Fara
authors.initials: F
authors.lastname: Petruzziello
authors.forename: Alain
authors.initials: A
authors.lastname: Fischer
authors.forename: Michaela
authors.initials: M
authors.lastname: Calvillo
authors.forename: Carlo
authors.initials: C
authors.lastname: Dufour
mesh_terms: D000744:Anemia, Hemolytic, Autoimmune / Q000175:diagnosis / Q000628:therapy*; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D003131:Combined Modality Therapy; D004351:Drug Resistance; D006801:Humans; D007223:Infant; D008297:Male; D012010:Red-Cell Aplasia, Pure / Q000175:diagnosis / Q000628:therapy*; D020123:Sirolimus / Q000008:administration & dosage; D013156:Splenectomy; D016896:Treatment Outcome; D014740:Vidarabine / Q000008:administration & dosage / Q000031:analogs & derivatives
chemical_list: D014740:Vidarabine; C024352:fludarabine; D020123:Sirolimus