Alkylator-free conditioning regimen for patients with acquired aplastic anemia, transplanted from genetically identical twins.
Allogeneic stem cell transplantation remains the best option for young patients with SAA. With genetically identical twin as an ideal donor, the majority of SAA patients require appropriate immunosuppression before and after stem cell transplantation to obtain long-term hematopoietic reconstitution. Alkylating agents, used during conditioning, are associated with short- and long-term toxic effects that lead to poor compliance of treatment and could compromise the quality of future life. Three SAA patients, transplanted from genetically identical twins without using alkylating agents during conditioning, showed rapid and sustained hematological reconstitution without any evidence of conditioning-related toxicity.
Trakhtman
P
P
Bone Marrow Transplantation Unit, Russian Institute for Pediatric Hematology, Oncology and Immunology, 117513 Moscow, Russia. trakhtman@mail.ru
Balashov
D
D
Shipicina
I
I
Skvortsova
Y
Y
Shelikhova
L
L
Filimonov
A
A
Novichkova
G
G
Skorobogatova
E
E
Maschan
M
M
Maschan
A
A
D000477:Alkylating Agents; D000741:Anemia, Aplastic / Q000601:surgery*; D000970:Antineoplastic Agents / Q000494:pharmacology*; D000075202:Contraindications; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D019653:Myeloablative Agonists; D012189:Retrospective Studies; D033581:Stem Cell Transplantation; D019172:Transplantation Conditioning / Q000379:methods*; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D014430:Twins, Monozygotic / Q000235:genetics*; D014740:Vidarabine / Q000031:analogs & derivatives* / Q000494:pharmacology
D000477:Alkylating Agents; D000970:Antineoplastic Agents; D019653:Myeloablative Agonists; D014740:Vidarabine; C024352:fludarabine