Better knowledge and awareness of Late Effects after pediatric HSCT for non-malignant diseases will lead to optimal screening procedures after HSCT that may eventually contribute to reduce transplant-related long term morbidity and mortality and…
ID
Bron
Verkorte titel
Aandoening
Inborn errors of immunity, hemoglobinopathies, and bone marrow failure.
Ondersteuning
Onderzoeksproduct en/of interventie
Uitkomstmaten
Primaire uitkomstmaten
1. To investigate the late effects e.g. organ function, graft function, growth, psychosocial and neurocognitive development, dental abnormalities, involved health care providers, socioeconomic and demographic characteristics of children and adults after pediatric HSCT for non-malignant diseases.
2. To identify biological, demographic and psychological and therapeutic determinants for morbidity, mortality and treatment outcome in children and adults with a pediatric HSCT for non-malignant diseases.
3. To evaluate and improve aspects of value-based healthcare organization and other (perceived) care aspects in children and adults with a pediatric HSCT for non-malignant diseases by measurement of patient and treatment characteristics, patient-reported outcomes, patient and healthcare providers reported experiences, perceived patient-centeredness of care, health care use, costs, and to perform analyses of associations between these factors and health care outcome.
Achtergrond van het onderzoek
This retrospective and prospective observational cohort study aims to determine the Late Effects of allogeneic stem cell transplantation (HSCT) at pediatric age in the Netherlands for non-malignant diseases and to identify factors that contribute to these Late Effects. These factors include: biological, sociodemographic, psychological and clinical determinants. Clinical data will be combined with patient/caregivers reported outcome measures and patient/caregivers/healthcare providers reported experience measures. Data will be collected retrospectively and prospectively at participants' yearly regular clinic visits for routine follow-up.
Doel van het onderzoek
Better knowledge and awareness of Late Effects after pediatric HSCT for non-malignant diseases will lead to optimal screening procedures after HSCT that may eventually contribute to reduce transplant-related long term morbidity and mortality and improve quality of life. Next, with emerging therapies (e.g. gene therapy) in the near future, structured insight into the (late) effects of these stem cell therapies is essential for evaluation of its effects.
Onderzoeksopzet
Healthcare outcome measures will be collected retrospectively and prospectively at participants' annual regular clinic visits as part of standard care.
Onderzoeksproduct en/of interventie
NA
Publiek
Wetenschappelijk
Belangrijkste voorwaarden om deel te mogen nemen (Inclusiecriteria)
In order to be eligible to participate in this study, a subject must meet all of the following criteria:
• >2 years after pediatric HSCT for non-malignant diseases
• Written informed consent by the patient or legal guardians, and pediatric consent when indicated
Belangrijkste redenen om niet deel te kunnen nemen (Exclusiecriteria)
A potential subject who meets any of the following criteria will be excluded from participation in this study:
• Any medical or social reasons, which obstruct or inhibit study participation according to the treating physician;
• Patient or legal guardians unable or unwilling to give consent, or lack of pediatric consent when indicated.
Opzet
Deelname
Voornemen beschikbaar stellen Individuele Patiënten Data (IPD)
Opgevolgd door onderstaande (mogelijk meer actuele) registratie
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Andere (mogelijk minder actuele) registraties in dit register
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In overige registers
| Register | ID |
|---|---|
| NTR-new | NL9112 |
| CCMO | NL20.181 |