We aim to determine the long-term integrity of hematopoiesis in pediatric and young adult HSCT survivors.
ID
Source
Brief title
Condition
- Anaemias nonhaemolytic and marrow depression
- Immune disorders NEC
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The primary aim of this study is to determine the percentage of long-term HSCT
survivors with clonal hematopoiesis (CH), and to identify clinical determinants
that are associated with CH.
Secondary outcome
We also aim to determine the percentage of long-term HSCT survivors with the
following other types of hematopoietic dysfunction: (1) cytopenia; (2) loss of
donor chimerism; (3) bone marrow failure; (4) myelodysplasia; and (5)
donor-cell leukemia. Finally, we will perform in-depth molecular studies to
analyze hematopoietic integrity upon HSCT, including genomic and flow
cytometric analyses.
Background summary
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 increase. In pediatric and young adult HSCT survivors,
the donor stem cells may have to live far beyond the normal human life span. 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 objective
We aim to determine the long-term integrity of hematopoiesis in pediatric and
young adult HSCT survivors.
Study design
This is an observational, explorative study, embedded in the HSCT follow-up
outpatient clinics of the Princess Máxima Center and UMC Utrecht. 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 and buccal swab for
in-depth assessment of hematopoietic integrity after HSCT.
Study burden and risks
This study requires pediatric and young adult recipients, as their
post-transplant survival exceeds that of older adult HSCT recipients by several
decades, posing unique challenges on the integrity and longevity of the
engrafted HSCs. In addition, as clinical HSCT regimens differ between children
and adults (e.g. use of irradiation, chemotherapy dose), results obtained in
adult HSCT recipients cannot be translated directly to children.
This study will provide unique insights into the long-term effects of HSCT on
hematopoiesis, while posing minimal risks to the participants. Results from
this study may contribute to improved, risk-adjusted clinical follow-up.
Moreover, understanding of the clinical determinants of long-term HSCT-related
hematopoietic dysfunction may result in better HSCT protocols.
Heidelberglaan 25
UTRECHT 3584CS
NL
Heidelberglaan 25
UTRECHT 3584CS
NL
Listed location countries
Age
Inclusion criteria
Underwent allogeneic HSCT at age <40 yrs
A minimum survival of 5 years after HSCT.
Exclusion criteria
Failure of the HSCT recipient, donor and/or their legal representatives to
understand the patient information and informed consent form (either due to
intellectual disability or to language problems).
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.
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
In other registers
| Register | ID |
|---|---|
| Other | Netherlands Trial Register NL9587 |
| CCMO | NL77721.041.21 |