Allogeneic stem cell transplantation in children with acute lymphoblastic leukemia after isolated central nervous system relapse: our experiences and review of the literature

Abstract

The prognosis of patients with acute lymphoblastic leukemia (ALL) and central nervous system (CNS) relapse has historically been very poor. Although chemoradiotherapy has improved outcomes, some patients still have a poor prognosis after CNS relapse. Therefore, allogeneic hematopoietic stem cell transplantation (allo-SCT) has recently become an option for treatment of CNS leukemia; however, information, particularly on the long-term outcome of transplant recipients, is limited. We performed allo-SCT in eight pediatric patients with ALL (n ¼ 7) or T-cell type non-Hodgkin's lymphoma (n ¼ 1), who had isolated CNS relapse. All patients survived for a median of 70.5 (range, 13-153) months after SCT. Sequelae developed late in some patients: mental retardation (IQ ¼ 47) in one patient, severe alopecia in two patients, limited chronic graft-versus-host-disease in three patients, and amenorrhea and/or hypothyroidism in three patients. Except for a pre-school child with post transplant CNS relapse, six out of seven patients show normal school/social performance. Our results clearly indicate a high cure rate of isolated CNS relapse by allo-SCT in pediatric lymphoid malignancies; however, there needs to be further studies to determine which are the appropriate candidates for transplantation and what is the best transplant regimen to achieve high cure rate and maintain good quality of life.

Submicroscopic bone marrow involvement in isolated extramedullary relapses in childhood acute lymphoblastic leukemia: a more precise definition of “isolated” and its possible clinical implications, a collaborative study of the Resistant Disease Committee of the International BFM study group

“…A more intensified therapy may be necessary with high-dose polychemotherapy and/or autologous/allogeneic SCT for patients with submicroscopic BM involvement. 37 …”

Intrathecal chemoprophylaxis after HSCT in children

“…A recent study from 2006 which focused solely on patients with CNS relapse prior to HSCT achieved a relapse‐free survival of 70 months (median) for seven of their eight patients without post‐HSCT i.t. therapy (8).…”

First Report of Autologous Cord Blood Transplantation in the Treatment of a Child With Leukemia

“…For these reasons, high-dose therapy followed by HSC transplantation may be indicated for some patients with early isolated CNS relapse. 2 Bordigoni et al 3 reported 77% disease-free survival after HSC transplantation in children who had CNS relapse while receiving chemotherapy. This is significantly better than reported survival rates after conventional chemotherapy and radiotherapy in this patient population.…”