Hematopoietic stem cell transplantation in infants

It is rare for infants, who are less than 365 days old, to receive hematopoietic stem cell transplantation (HSCT). Our objective was to review the indications, survival, and late effects of infants who received HSCT.

Between April 1992 and March 2010, a total of 1,363 children underwent HSCT (775 allogeneic [allo]; 588 autologous [auto]) in the Hospital for Sick Children, Toronto. Of these, 51 (3.7%) were infants.

Seventeen infants received allo HSCT for a genetic metabolic disorder. The median age at HSCT was 211 days (29-334 days). After median follow-up of 8.9 years (2.9-20.2 years), 12 patients remained alive, representing an overall survival rate of 70%. Infants with non-metabolic disorders (n = 34); 10 (three neuroblastoma [NBL], three brain tumor, two acute meylogenous leukemia [AML], one rhabdomyosarcoma, and one retinoblastoma) received auto HSCT, and 24 (eight hemophagocytic lymphohistiocytosis [HLH], four juvenile meylomonocytic leukemia [JMML], four Wiscott-Aldrych Syndrome [WAS], three acute lymphoblastic leukemia [ALL], two AML, one severe aplastic anemia [SAA], one chronic granulomatous disease [CGD], and one amegakaryocytic thrombocytopenia) received allo HSCT. Their median age at HSCT was 255 days (142-365 days). At median follow-up of 8.7 years (2.5-17.6 years), 26 infants remained alive, representing an overall survival rate of 76%. In the auto HSCT category, eight of 10 infants are long-term survivors. Late effects such as organ dysfunction, endocrinopathy, and secondary tumors were within accepted range.

The survival rate of infants who receive HSCT is encouraging.