Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation in Children and Adolescents

Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation in Children and Adolescents

Purpose of study: This phase I/II trial is to evaluate the safety and feasibility of TCRαβ-depleted graft from haploidentical family donors in treating children and adolescents with malignant or non-malignant diseases.

Inclusion Criteria:

A. Disease inclusions

1. Hematologic malignancy:

   • Acute lymphoblastic leukemia including induction failure, CR1 (Ph+, t(4:11), hypodiploid and other very high risk features), ≥ CR2, infant ALL with MLL or other unfavorable features
   • Acute myeloid leukemia excluding CR1 with t(8:21), inv(16), t(15:17), and Down syndrome
   • Myelodysplastic syndrome: RCC with -7 or RCC in need of transfusion
   • Chronic myeloid leukemia in AP
   • Juvenile myelomonocytic leukemia
   • Malignant lymphoma, NHL or HD, after failed autologous HSCT
   • Other

2. Non-hematologic malignancy

   • Relapsed or refractory solid tumors including neuroblastoma, rhabdomyosarcoma and so on

3. Non-malignant hematologic disease

   • Acquired severe and very severe aplastic anemia
   • Fanconi anemia
   • Paroxysmal nocturnal hemoglobinuria
   • Congenital dyserythropoietic anemia
   • Others

4. Inherited or metabolic disease

   • Hemophagocytic lymphohistiocytosis
   • Malignant osteopetrosis
   • Storage diseases
   • Others B. Recipient inclusions

1. Age < 21 years 2. No HLA-identical stem cell donor available 3. Lansky-Play performance score >60 4. No active infection at the time of transplantation

Exclusion Criteria:

1. HIV-infection
2. Presence of active and serious infection
3. Cardiac ejection fraction <35% on echocardiography
4. Severe pulmonary dysfunction (DLCO <30%)
5. Liver function abnormalities with bilirubin >4mg/dL and elevation of transaminases > 400U/L
6. Concurrent severe or uncontrolled medical disease
7. Patients who are pregnant
8. Patients unwilling or unable to comply with the protocol or unable to give informed consent