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…
ID
Bron
Verkorte titel
Aandoening
Allogeneic Hematopoietic Stem Cell Transplantation
Ondersteuning
Onderzoeksproduct en/of interventie
Uitkomstmaten
Primaire uitkomstmaten
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.
Achtergrond van het onderzoek
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.
Doel van het onderzoek
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.
Onderzoeksopzet
Single collection of peripheral blood >5 years after HSCT
Onderzoeksproduct en/of interventie
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).
Publiek
Wetenschappelijk
Belangrijkste voorwaarden om deel te mogen nemen (Inclusiecriteria)
- 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.
Belangrijkste redenen om niet deel te kunnen nemen (Exclusiecriteria)
- 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).
Opzet
Deelname
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In overige registers
Register | ID |
---|---|
NTR-new | NL9587 |
CCMO | NL77721.041.21 |
OMON | NL-OMON54142 |