title: Late graft failure in FA--case report and review of the literature.
abstract: Hematological disorders in patients with FA can only be cured by allogeneic HSCT. Severe infections in primary and early secondary graft failures pose a particular risk. Whereas most graft failures occur within 100 days, those observed after day +100 are infrequent. Here, we present our analysis of a secondary graft failure more than five yr after a first allogeneic HSCT. In this patient, isolated thrombocytopenia over a period of 12 months resulted in a chimerism subset analysis revealing a considerable decrease in the CD34-positive donor cell fraction. After a second fludarabine-based preparative regimen, the patient received PBSC from the same donor. Chimerism returned to full donor in all subsets. This clinical course demonstrates that isolated thrombocytopenia can precede complete graft failure for several months. Our review of the literature on late graft failures in patients with FA after day +100 reveals the absence of fludarabine in the preparative regimen as a potential risk factor. Further clinical research is necessary to identify more suitable approaches for ensuring safe and stable engraftment.
authors.affiliation: Department of Pediatric Hematology and Oncology, University Children's Hospital Muenster, Muenster, Germany. ehlertk@ukmuenster.de
authors.forename: Karoline
authors.initials: K
authors.lastname: Ehlert
authors.forename: Andreas H
authors.initials: AH
authors.lastname: Groll
authors.forename: Claudia
authors.initials: C
authors.lastname: Rossig
authors.forename: Michael C
authors.initials: MC
authors.lastname: Fruehwald
authors.forename: Heribert
authors.initials: H
authors.lastname: Juergens
mesh_terms: D018952:Antigens, CD34 / Q000096:biosynthesis; D000970:Antineoplastic Agents / Q000494:pharmacology; D002648:Child; D005199:Fanconi Anemia / Q000628:therapy*; D005260:Female; D006084:Graft Rejection; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D009504:Neutrophils / Q000166:cytology; D012307:Risk Factors; D013921:Thrombocytopenia / Q000209:etiology / Q000628:therapy; D019172:Transplantation Conditioning / Q000379:methods; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D014740:Vidarabine / Q000031:analogs & derivatives / Q000494:pharmacology
chemical_list: D018952:Antigens, CD34; D000970:Antineoplastic Agents; D014740:Vidarabine; C024352:fludarabine