Clinical trial NCT06075563: Total Lymphoid Irradiation as Conditioning for Pediatric Haploidentical Hematopoietic Stem Cell Transplantation
Official title: Total Lymphoid Irradiation as Conditioning for Pediatric Haploidentical Hematopoietic Stem Cell Transplantation
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Hematopoietic Organs; Disorder
Lead sponsor: Hong Kong Children's Hospital
Sponsor class: OTHER
Collaborators: name: The University of Hong Kong
name: Hong Kong Sanatorium & Hospital
First posted: 2023-10-10
Enrollment: 23
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: In this comparative study, the investigators aim to compare prospective pediatric patients who receive total lymphoid irradiation (TLI) using tomotherapy with age- and disease-matched controls who receive conventional total body irradiation (TBI) as part of conditioning for haploidentical hematopoietic stem cell transplantation (HSCT) for both malignant and non-malignant diseases. The investigators shall evaluate graft failure-free, graft-versus-host disease (GVHD)-free survival, overall survival, frequency of rejection, GVHD, relapse of malignancy, adverse effects and post-transplant immunoreconstitution.

Detailed description: Radiotherapy at a dose of 6Gy divided into 3 equal fractions (2Gy per fraction) to all lymphoid organs (TLI) over 2 days using tomotherapy will be given to participants as part of the conditioning treatment before haploidentical hematopoietic stem cell transplantation (HSCT). Retrospective patients who are age- and disease-matched with the recruited patients and had received radiotherapy to the whole body (TBI) with or without lung shielding at a total dose of 2-12 Gy delivered as 1-6 equal fractions by conventional opposed fields radiotherapy as part of conditioning will be compared for the outcomes.

Eligibility criteria: Inclusion Criteria:

* Aged 0-18 years who requires allogeneic HSCT, has no human leukocyte antigen (HLA)-matched sibling donor but has an HLA-haploidentical donor.
* Adequate organ function to tolerate the conditioning chemotherapy and radiotherapy
* Karnofsky or Lansky performance status score ≥50

Exclusion Criteria:

* Pregnant or lactating woman
* HIV infection
* Patients for whom alternative treatment is deemed more appropriate by treating physician
* Patients who are unlikely to benefit from haploidentical hematopoietic stem cell transplantation, e.g., terminal malignancy with multiorgan failure

Primary outcomes:
measure: Graft failure-free, GVHD-free survival
timeFrame: up to 1 year
Secondary outcomes:
measure: Overall survival
timeFrame: up to 1 year
measure: Proportion of patients who develop relapse among those with malignant diseases
timeFrame: up to 1 year
measure: Proportion of patients who develop graft failure
timeFrame: up to 1 year
measure: Proportion of patients who develop acute graft-versus-host disease
timeFrame: up to 1 year
measure: Proportion of patients who develop chronic graft-versus-host disease
timeFrame: up to 1 year
measure: Proportion of patients who develop adverse events not mentioned in outcomes 4-6
timeFrame: up to 1 year
measure: Blood T lymphocyte count at 3 months
timeFrame: at 3 months
measure: Blood T lymphocyte count at 1 year
timeFrame: at 1 year
Other outcomes:
Locations:
city: Hong Kong
country: Hong Kong
facility: Hong Kong Children's Hospital
Study officials:
affiliation: Hong Kong Children's Hospital
name: Daniel Cheuk
role: PRINCIPAL_INVESTIGATOR
References: