A novel cell-based therapy for patients with aplastic anemia.
OBJECTIVE
Aplastic anemia (AA) is a rare but potentially life-threatening disease. There is a need for the development of new, more effective and less toxic therapies for treating AA. The safety and efficacy of an immune cell-based therapy for AA was examined.


METHODS
Thirty-one patients with idiopathic AA received intravenous infusions of ex vivo-activated autologous and allogeneic immune cells at least once a week. Response to therapy was assessed by symptoms, transfusion dependency, blood counts, bone marrow biopsy and survival.


RESULTS
Of the 31 patients, 25 (81%) had either complete (11, 35%) or partial (14, 45%) responses, while six (19%) showed no response to the therapy. The overall survival rates at 3 years were 90%.


CONCLUSIONS
The therapy described appears to be safe and effective. The data from this pilot study suggest that a larger, controlled study is warranted.
Chen
Jiayu
J
Department of Hematology, The 12th Municipal Hospital of Guangzhou, Guangzhou, China.
Liu
Weiwei
W
Yu
Wei
W
Chen
Lingzhen
L
Wu
Jinming
J
Zhan
Yu
Y
Wu
Lupo
L
Yang
Demao
D
D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000139:chemically induced / Q000276:immunology* / Q000473:pathology / Q000628:therapy*; D001554:Benzene / Q000009:adverse effects; D000001:Calcimycin / Q000494:pharmacology; D002452:Cell Count; D002648:Child; D018450:Disease Progression; D005260:Female; D005500:Follow-Up Studies; D006410:Hematopoiesis / Q000276:immunology*; D006801:Humans; D016219:Immunotherapy, Adoptive; D007376:Interleukin-2 / Q000276:immunology / Q000494:pharmacology; D007963:Leukocytes, Mononuclear / Q000187:drug effects / Q000276:immunology / Q000378:metabolism* / Q000473:pathology; D008213:Lymphocyte Activation / Q000187:drug effects; D008297:Male; D008875:Middle Aged; D010865:Pilot Projects; D016896:Treatment Outcome
D007376:Interleukin-2; D000001:Calcimycin; D001554:Benzene