Hematopoietic cell transplantation for DOCK8 deficiency: Results from a prospective clinical trial.

ABSTRACT:

Background

DOCK8 deficiency is a primary immunodeficiency in which allogeneic hematopoietic cell transplantation (HCT) represents the only known cure. We tested the ability of a busulfan-based regimen to achieve reliable engraftment and high levels of donor chimerism with acceptable toxicity in a prospective clinical trial in DOCK8 deficiency.

Objectives

To both evaluate the ability of HCT to reverse the clinical phenotype and to correct the immunologic abnormalities by 1 year post HCT.

Methods

We conducted a prospective HCT trial for recipients with DOCK8 deficiency. Subjects were recruited from October 5, 2010, to December 30, 2022. Donor sources included fully matched related and unrelated donors and haploidentical donors. The reduced toxicity, myeloablative condit

SUBMITTER: Freeman AF 

PROVIDER: S-EPMC11700784 | biostudies-literature | 2025 Jan

REPOSITORIES: biostudies-literature