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ID
Source
Brief title
Health condition
Allogeneic Hematopoietic Stem Cell Transplantation
Sponsors and support
Intervention
Outcome measures
Primary outcome
Determine the percentage of HSCT survivors with hematopoietic dysfunction, defined as: (1) cytopenia; (2) clonal hematopoiesis; (3) loss of donor chimerism; (4) bone marrow failure; (5) myelodysplasia; and (6) donor-cell leukemia.
Secondary outcome
Identify clinical determinants of hematopoietic dysfunction in long-term pediatric HSCT survivors compared to those without hematopoietic dysfunction.
Analyze the integrity of hematopoiesis at a molecular level (pending funding).
Background summary
This is an observational, explorative study, embedded in the HSCT LATER outpatient clinic of the Princess Máxima Center. The study visit will be combined with a clinical visit, as part of regular post-HSCT follow-up. All participants will undergo clinical assessment of HSCT-related long-term effects by a trained physician, including measurement of differential blood counts, as part of routine clinical care. For this study, we will use these clinical data, and collect an additional blood sample for in-depth assessment of hematopoietic integrity after HSCT.
Study objective
Hematopoietic stem cell transplantation (HSCT) is a last-resort, curative therapy for patients suffering from various, otherwise lethal, diseases. Due to improved treatment strategies, the number of HSCT survivors and their life expectancy continue to increase1. In pediatric HSCT survivors, the donor stem cells may have to live far beyond the normal human life span2. It remains unknown whether transplanted HSCs can sustain life-long healthy blood production in these recipients. In the current project, we hypothesize that HSCT compromises HSC longevity and predisposes to (age-related) hematopoietic dysfunction in the recipient.
Study design
Single collection of peripheral blood >5 years after HSCT
Intervention
From each HSCT survivor, we will collect up to 50 mL peripheral blood. For participants <25 kg, the volume of blood will be adjusted to 2 mL per kg bodyweight (2.5% of total blood volume).
Inclusion criteria
- Underwent allogeneic HSCT at age ≤18 yrs
- A minimum survival of 5 years after HSCT. In case a patient has received multiple HSCTs, the last HSCT will be used to determine this 5-year minimum.
Exclusion criteria
- Recipients of a “NiCord” HSCT. NiCord is a clinical trial on the safety and efficacy of transplantation of ex vivo expanded cord blood HSCs. As outcome measures of our study overlap with the outcome of this trial, NiCord recipients will be excluded.
- Failure of the HSCT recipient and/or their legal representatives to understand the patient information and informed consent form (either due to intellectual disability or to language problems).
Design
Recruitment
IPD sharing statement
Followed up by the following (possibly more current) registration
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
Register | ID |
---|---|
NTR-new | NL9587 |
CCMO | NL77721.041.21 |
OMON | NL-OMON54142 |