Pediatr Blood Cancer
Hematopoietic Stem Cell Transplantation in Infants Adam Gassas, MBChB, MSc, FRCP, DCH,1* Kaleem Ashraf, MD,2 Irina Zaidman, MD,3 Muhammad Ali, Joerg Krueger, MD,1 John Doyle, MD, FRCPC,4 Tal Schechter, MD,1 and Stephan Leucht, MD1 Background. 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. Procedure. 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. Results. 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 nonmetabolic disorders (n ¼ 34); 10 (three neuroblastoma [NBL], three brain tumor, two acute meylogenous leukemia [AML], one rhabdomyosarcoma, and one retinoblastoma) received auto HSCT,
MD,
1
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. Conclusion. The survival rate of infants who receive HSCT is encouraging. Pediatr Blood Cancer # 2014 Wiley Periodicals, Inc.
Key words: hematopoietic stem cell transplantation; infants
INTRODUCTION Hematopoietic stem cell transplantation (HSCT) is a wellknown life-saving procedure for many malignant and nonmalignant diseases. Since the first successful human HSCT in 1968 [1], many children and adults have received HSCT with reasonable results [2]. However, transplant-related mortality (TRM) continues to be a major risk after HSCT, averaging anywhere between 5% and 30% depending on disease status and donor source [3]. Furthermore, for HSCT survivors, significant morbidity could occur due to high-dose chemotherapy or radiation therapy along with specific HSCT complications such as graftversus-host disease (GVHD) and delayed immune reconstitution [4]. Children are at the peak of their growth and development as infants, in the first year of their lives; it is unknown how high-dose chemotherapy or radiotherapy will affect them as their organs continue to mature. And because the number of infants receiving HSCT is low, it is difficult to undertake large studies on this vulnerable patient population. Another interesting phenomenon is that disease biology may be different in infants compared to older children. For example, infants with acute lymphoblastic leukemia (ALL) have inferior outcome than older children with ALL [4,5]. On the other hand, infants with other cancers, such as neuroblastoma, have much better outcome than older children with neuroblastoma [6]. Our objective was to review the indications, overall survival, and long-term toxicities of HSCT in infants in a large pediatric transplant center, namely the Hospital for Sick Children in Toronto, Ontario.
MATERIALS AND METHODS This study was approved by our institutional research ethics board. The medical records of children who received HSCT between April 1992 and March 2010 at The Hospital for Sick Children in Toronto were reviewed to identify those who underwent HSCT during infancy (
Hematopoietic Stem Cell Transplantation in Infants Adam Gassas, MBChB, MSc, FRCP, DCH,1* Kaleem Ashraf, MD,2 Irina Zaidman, MD,3 Muhammad Ali, Joerg Krueger, MD,1 John Doyle, MD, FRCPC,4 Tal Schechter, MD,1 and Stephan Leucht, MD1 Background. 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. Procedure. 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. Results. 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 nonmetabolic disorders (n ¼ 34); 10 (three neuroblastoma [NBL], three brain tumor, two acute meylogenous leukemia [AML], one rhabdomyosarcoma, and one retinoblastoma) received auto HSCT,
MD,
1
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. Conclusion. The survival rate of infants who receive HSCT is encouraging. Pediatr Blood Cancer # 2014 Wiley Periodicals, Inc.
Key words: hematopoietic stem cell transplantation; infants
INTRODUCTION Hematopoietic stem cell transplantation (HSCT) is a wellknown life-saving procedure for many malignant and nonmalignant diseases. Since the first successful human HSCT in 1968 [1], many children and adults have received HSCT with reasonable results [2]. However, transplant-related mortality (TRM) continues to be a major risk after HSCT, averaging anywhere between 5% and 30% depending on disease status and donor source [3]. Furthermore, for HSCT survivors, significant morbidity could occur due to high-dose chemotherapy or radiation therapy along with specific HSCT complications such as graftversus-host disease (GVHD) and delayed immune reconstitution [4]. Children are at the peak of their growth and development as infants, in the first year of their lives; it is unknown how high-dose chemotherapy or radiotherapy will affect them as their organs continue to mature. And because the number of infants receiving HSCT is low, it is difficult to undertake large studies on this vulnerable patient population. Another interesting phenomenon is that disease biology may be different in infants compared to older children. For example, infants with acute lymphoblastic leukemia (ALL) have inferior outcome than older children with ALL [4,5]. On the other hand, infants with other cancers, such as neuroblastoma, have much better outcome than older children with neuroblastoma [6]. Our objective was to review the indications, overall survival, and long-term toxicities of HSCT in infants in a large pediatric transplant center, namely the Hospital for Sick Children in Toronto, Ontario.
MATERIALS AND METHODS This study was approved by our institutional research ethics board. The medical records of children who received HSCT between April 1992 and March 2010 at The Hospital for Sick Children in Toronto were reviewed to identify those who underwent HSCT during infancy (
