Abatacept-based Graft-Versus-Host Disease Prophylaxis in Haplo-identical Hematopoietic Cell Transplant in a High-risk Cohort.

BACKGROUND: There is insufficient guidance in using posttransplant cyclophosphamide in patients with organ dysfunctions. Abatacept (Aba), a T cell costimulation blockade, has recently been shown to prevent severe acute graft-versus-host disease (GVHD). OBSERVATION: We report adding Aba as GVHD prophylaxis in 4 pediatrics patients who received haplo-hematopoietic cell transplantation. Two patients had grade 2 acute GVHD and 2 had mild chronic GVHD. All 4 patients are alive with full donor chimerism, and 3 are off immunosuppressants. CONCLUSION: An Aba-based regimen can result in reliable engraftment and acceptable GVHD when concerns of organ dysfunction prevents the use of posttransplant cyclophosphamide in haplo-hematopoietic cell transplantation.

Central nervous system relapse in a child with anaplastic large cell lymphoma: potential for new therapeutic strategies.

BACKGROUND: Central nervous system (CNS) relapse is rare in childhood anaplastic large cell lymphoma (ALCL) and is associated with a poor prognosis. CASE: We describe an 8-year-old boy with ALCL who developed an early CNS relapse without initial CNS disease. Despite aggressive medical management, the patient's neurological status deteriorated rapidly and he died shortly after. CONCLUSION: Optimal treatment for children with relapsed ALCL involving the CNS remains unclear. Novel agents, including ALK inhibitors, that have CNS-penetration might be helpful and pediatric studies are warranted.