ate Effects and Follow up after pediatric hematological stem cell transplantation and cell therapy (LEEF)

Recruiting

Conditions: <p>Inborn errors of immunity, hemoglobinopathies, and bone marrow failure.</p>

Registration Number: NL-OMON20380

Lead Sponsor: one

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: Not specified

Target Recruitment: 250

Inclusion Criteria

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

Exclusion Criteria

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.

Study & Design

Study Type: Observational non invasive

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>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.</p>

Secondary Outcome Measures