Unrelated donor cord blood transplantation for non-malignant disorders in children and adolescents
Authors
Meerim Park ; Young Ho Lee ; Hae-Ryong Kang ; Ji Won Lee ; Hyoung Jin Kang ; Kyung Duk Park ; Hee Young Shin ; Hyo Seop Ahn ; Hee Jo Baek ; Hoon Kook ; Tai Ju Hwang ; Jae Wook Lee ; Nack-Gyun Chung ; Bin Cho ; Hack-Ki Kim ; Soo Hyun Lee ; Keon Hee Yoo ; Ki Woong Sung ; Hong Hoe Koo ; Kyung Nam Koh ; Ho Joon Im ; Jong Jin Seo ; Jun Eun Park ; Yeon Jung Lim ; Chuhl Joo Lyu ; Jae Min Lee ; Jeong Ok Hah
This study analyzes the data reported to the Korean Cord Blood Registry between 1994 and 2008, involving children and adolescents with non-malignant diseases. Sixty-five patients were evaluated in this study: SAA (n = 24), iBMFS, (n = 16), and primary immune deficiency/inherited metabolic disorder (n = 25). The CI of neutrophil recovery was 73.3% on day 42. By day 100, the CI of acute grade II–IV graft-versus-host disease was 32.3%. At a median follow-up of 71 months, five-yr OS was 50.7%. The survival rate (37.5%) and CI of neutrophil engraftment (37.5%) were lowest in patients with iBMFS. Deaths were mainly due to infection, pulmonary complications, and hemorrhage. In a multivariate analysis, the presence of >3.91 × 105/kg of infused CD34 + cells was the only factor consistently identified as significantly associated with neutrophil engraftment (p = 0.04) and OS (p = 0.03). UCBT using optimal cell doses appears to be a feasible therapy for non-malignant diseases in children and adolescents for whom there is no appropriate HLA-matched related donor. Strategies to reduce transplant-related toxicities would improve the outcomes of UCBT in non-malignant diseases.