MPN Childhood Registry

NCT07410247 | Recruiting

• 1 other identifier: EK FAU: 22-365-B
• Study Type: observational
• Target: 500
• Locations: 1 country
• Sites: 1

Brief Summary

Myeloproliferative neoplasms (MPNs) are caused by defects in blood cell production. This leads to an overproduction of mature cells, such as red blood cells or platelets. As a result, clinical symptoms and complications can develop. Circulatory disorders and resulting pain symptoms, such as headaches, are the most prominent. Furthermore, blood clotting disorders can occur, leading to vascular occlusions or bleeding. Over a longer period, bone marrow transformation into fibrous tissue or the development of acute leukemia can occur, resulting in even more serious complications. In the vast majority of cases, MPNs are diagnosed in older adults. These diagnoses are extremely rare in children and adolescents. Therefore, clinical and genetic characteristics, treatment options, and outcomes in pediatric patients with MPNs are insufficiently described, and only limited data are available for standardizing diagnostic and therapeutic approaches for MPNs in childhood and adolescence. Recommendations and guidelines from the adult field, which have largely been adopted and applied to children and adolescents, do not adequately address the specific needs of patients in childhood and adolescence. To gain more insights into MPN during this early, vulnerable phase of life, we have established a nationwide registry in Germany that systematically collects data from children and adolescents with MPN. The registry focuses particularly on the scientific analysis of the biological characteristics and differences of MPN in children and adolescents compared to adults.

Conditions

Childhood Myeloproliferative Neoplasms

Keywords

Childhood Myeloproliferative Neoplasms

Outcome Measures

Primary Outcomes (4)

• Incidence and epidemiology

  Incidence and epidemiology of PV, ET, PMF and pHES in children and adolescents through systematic inclusion of these patients in a national population-based registry

  15 years

• Characterization

  Characterization of clinical, hematologic, genetic and cytogenetic features of children and adolescents with PV, ET, PMF and pHES

  15 years

• Risk identification

  Identification of pediatric patients with high risk of complications and disease progression and transformation into acute myeloid leukemia (AML)

  15 years

• Improvement of therapy

  Improvement of the therapy for children and adolescents with MPN by discussing treatment options with the treating centers and tailor therapy to the specific and individual needs of pediatric patients

  15 years

Secondary Outcomes (2)

• Preparation of international studies

  15 years

• Development of a biobank

  15 years

Study Arms (1)

Childhood MPN

Pediatric Patients below the age of 18 years diagnosed with Myeloproliferative Neoplasms

Other: Observation

Interventions

Observation OTHER

Observation

Childhood MPN

Eligibility Criteria

• Age: 0 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

Children and adolescents below the age of 18 years diagnosed with Myeloproliferative Neoplasms

You may qualify if:

• 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

You may not qualify if:

• o Secondary polycythemia, thrombocytosis, myelofibrosis or HES with underlying reasons other than PV, ET, PMF or pHES

Sponsors & Collaborators

• University of Erlangen-Nürnberg Medical School: lead
• German Society for Pediatric Oncology and Hematology (GPOH): collaborator

Study Sites (1)

Department of Pediatrics and Adolescent Medicine, University Hospital Erlangen, Friedrich-Alexander-Universität (FAU) Erlangen-Nürnberg, Erlangen, Germany

Erlangen, Bavaria, D-91054, Germany

RECRUITING

Related Links

• Webpage of the German MPN Childhood Registry

Biospecimen

Retention: SAMPLES WITH DNA

Peripheral Blood, Bone Marrow, Hair Follicles, Buccal Swabs

MeSH Terms

Interventions

Observation

Intervention Hierarchy (Ancestors)

Methods; Investigative Techniques

Study Officials

• Axel Karow, MD

  Department of Pediatrics and Adolescent Medicine, University Hospital Erlangen, Friedrich-Alexander-Universität (FAU) Erlangen-Nürnberg, Erlangen, Germany

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Axel Karow, PD Dr. med.

CONTACT

+4991318541376; axel.karow@uk-erlangen.de

Franziska Fahrmeier, MD

CONTACT

+4991318533118; franziska.fahrmeier@uk-erlangen.de

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Target Duration: 15 Years
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: PD Dr. med. Axel Karow

Study Record Dates

• First Submitted: February 8, 2026
• First Posted: February 13, 2026
• Study Start: January 1, 2023
• Primary Completion (Estimated): December 31, 2032
• Study Completion (Estimated): December 31, 2037
• Last Updated: February 13, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR, ANALYTIC CODE
• Time Frame: IPD after scientific publication of data Supporting information with start of registry
• Access Criteria: IPD medical professionals Supporting information public

Locations

DE (1)