Unrelated Umbilical Cord Blood Following HLA-haploidentical Hematopoietic Stem Cell Transplantation in Patients With β-thalassemia Major

Nanfang Hospital, Southern Medical University

Status: Unknown

Conditions: Thalassemia Major

Treatments: Genetic: unrelated CB following haplo-identical hematopoietic stem cells transplantation

Study type: Interventional

Funder types: Other

Identifiers

NCT02126046

NFTM20130101

Details and patient eligibility

About

Allo-hematopoietic stem cell transplantation(HSCT) is the only way to cure β-thalassemia major at present. To expand donor pool，we developed a haplo-identical HSCT (Hi-HSCT) platform. But in prior Hi-HSCT using high dose post-transplant Cyclophosphamide in patients with leukemia, cytopenia post-transplant often developed, which was considered as a symptom of GVHD. Therefore, the investigators add unrelated umbilical cord blood (UCB) to the Hi-HSCT. It has reported that, as third-party cells, UCB will reduce GVHD.The purpose of this study is to determine whether unrelated UCB following Hi-HSCT can improve outcomes of Hi-HSCT in patients with β-thalassemia major.

Enrollment: 30 estimated patients

Sex: All

Ages: 12 months to 18 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• β-thalassemia major
• < 18 year old
• Unrelated umbilical cord blood following Haplo-identical HSCT

Exclusion criteria

• ≥ 18 year old
• HLA- matched related donors
• Unrelated donor transplants
• Unrelated umbilical cord blood transplants
• Severe iron overload in heart by T2*

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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30 participants in 1 patient group

Hi-HSC-CBT

Other group

Treatment:

Genetic: unrelated CB following haplo-identical hematopoietic stem cells transplantation