Childhood myeloproliferative neoplasm registry
The Myeloproliferative Neoplasms (MPN) Childhood Registry and Biobank
This registry will collect medical and genetic information from children and teens with MPNs to see if patterns emerge that can help doctors choose better care.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 500 (estimated) |
| Ages | 0 Years to 17 Years |
| Sex | All |
| Sponsor | University of Erlangen-Nürnberg Medical School Academic / other |
| Drugs / interventions | mepolizumab, benralizumab, ruxolitinib, imatinib, Tofacitinib, chemotherapy |
| Locations | 1 site (Erlangen, Bavaria) |
| Trial ID | NCT07410247 on ClinicalTrials.gov |
What this trial studies
This observational registry enrolls children and adolescents (under 18) newly diagnosed with BCR::ABL1-negative MPNs — polycythemia vera, essential thrombocythemia, primary myelofibrosis — or pediatric hypereosinophilic syndrome and collects clinical, laboratory, genetic, and treatment data. Patients are treated at participating centers and data are collected prospectively from diagnosis with planned long-term follow-up to document complications, transformation, and outcomes. The registry aims to describe disease features, treatment approaches, and long-term outcomes to support pediatric-specific diagnostic and therapeutic guidance. Participation involves no experimental interventions and relies on standard clinical care and data sharing.
Who should consider this trial
Good fit: Children and adolescents under 18 years who are newly diagnosed with PV, ET, PMF, or pediatric HES, treated at a participating center, and whose guardians provide written consent are eligible.
Not a fit: Patients with secondary causes of polycythemia, thrombocytosis, myelofibrosis, or HES, as well as adults, are excluded and unlikely to benefit from this registry.
Why it matters
Potential benefit: If successful, the registry could improve understanding of pediatric MPNs and lead to better diagnosis, risk stratification, and treatment guidance for children.
How similar studies have performed: Adult MPN registries have yielded useful clinical and genetic insights, but pediatric-focused registries are much rarer and data on children remain limited.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Newly diagnosed PV, ET, PMF or pHES * Age \< 18 years (up to 17 years and 365 days) at the day of diagnosis * Patient treated in a participating center * Written informed consent to registry participation Exclusion Criteria: o Secondary polycythemia, thrombocytosis, myelofibrosis or HES with underlying reasons other than PV, ET, PMF or pHES
Where this trial is running
Erlangen, Bavaria
- Department of Pediatrics and Adolescent Medicine, University Hospital Erlangen, Friedrich-Alexander-Universität (FAU) Erlangen-Nürnberg, Erlangen, Germany — Erlangen, Bavaria, Germany (Recruiting)
Study contacts
- Principal investigator: Axel Karow, MD — Department of Pediatrics and Adolescent Medicine, University Hospital Erlangen, Friedrich-Alexander-Universität (FAU) Erlangen-Nürnberg, Erlangen, Germany
- Study coordinator: Axel Karow, PD Dr. med.
- Email: axel.karow@uk-erlangen.de
- Phone: +4991318541376
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.