Essential thrombocythemia (ET) and polycythemia vera (PV) are rare in adolescent and young adult (AYA). These conditions, similar to those in older patients, are linked with thrombotic complications and the potential progression to secondary myelofibrosis (sMF). This retrospective study of ET and PV patients diagnosed before age 25 evaluated complication rates and impact of cytoreductive drugs on outcomes. Among 348 patients (278 ET, 70 PV) with a median age of 20 years, the of thrombotic events was 1.9 per 100 patient-years. Risk factors for thrombosis included elevated white blood cell count (>11 × 109/L) (HR: 2.7, p = 0.012) and absence of splenomegaly at diagnosis (HR: 5.7, p = 0.026), while cytoreductive drugs did not reduce this risk. The incidence of sMF was 0.7 per 100 patient-years. CALR mutation (HR: 6.0, p < 0.001) and a history of thrombosis (HR: 3.8, p = 0.015) were associated with sMF risk. Interferon as a first-line treatment significantly improved myelofibrosis-free survival compared to other treatments or the absence of cytoreduction (p = 0.046). Although cytoreduction did not affect thrombotic event, early interferon use reduced sMF risk. These findings support interferon use to mitigate sMF risk in AYA ET and PV patients.

Biology of Cardiovascular Diseases University of Bordeaux INSERM UMR1034 Pessac France

Cellular Biology Department INSERM UMRS 1131 St Louis Hospital APHP Paris France

Centre Hospitalier Universitaire de Brest Brest France

Department of Haematology Norfolk and Norwich University Hospitals National Health Service Trust Norwich United Kingdom

Department of Hematology Amsterdam University Medical Centers Amsterdam Netherlands

Department of Hematology Annecy Genevois Hospital Pringy France

Department of Hematology Collegium Medicum in Bydgoszcz Nicolaus Copernicus University Torun Poland

Department of Hematology Collegium Medicum Jagiellonian University Krakow Poland

Department of Hematology Holy Cross Oncology Center Kielce Poland

Department of Hematology Medical University Lodz Poland

Department of Hematology St Vincent De Paul Hospital Lille France

Department of Hematology State Hospital Opole Poland

Department of Hematology University Hospital Dubrava School of Medicine University of Zagreb Zagreb Croatia

Department of Hematology University Hospitals Leuven and Department of Microbiology and Immunology Laboratory of Molecular Immunology KU Leuven Leuven Belgium

Department of Internal Medicine with Respiratory Medicine Center Academic and Research Medical institute Sumy State University Sumy Ukraine

Department of Medical Oncology and Hematology University Hospital Zurich Zurich Switzerland

Division of Hematology Oncology Chang Gung Memorial Hospital and Chang Gung University Taoyuan Taiwan

Experimental Hematology Department of Biomedicine University Hospital and University Basel Switzerland

Guy's and St Thomas' NHS Foundation Trust London ENG United Kingdom

Haematology Belfast City Hospital Queen's University Belfast Belfast United Kingdom

Haematology Department Guy's and St Thomas' NHS Foundation Trust London United Kingdom

Hematology and Bone Marrow Transplantation Department University of Medical Sciences Poznan Poland

Hematology and Transplantation Department Medical University and Clinical Center Gdansk Poland

Hematology Department AP HP University of Paris Saclay Bicêtre Hospital Paris France

Hematology Department Hospital del Mar Hospital del Mar Research Institute Barcelona Spain

Hematology Department Jagiellonian University Hospital Krakow Poland

Hematology Department Le Mans Hospital Le Mans France

Hematology Department University Hospital CHUAC A Coruña Spain

Hematology Department University Hospital Linkoping Sweden

Hematology Division and Faculty of Medicine Geneva University Hospitals University of Geneva Geneva Switzerland

Hopital Saint Louis Paris France

Institute of Hematology and Blood Transfusion Prague Prague Czech Republic

Institute of Hematology and Transfusion Medicine Warsaw Poland

Iuliu Hatieganu University of Medicine and Pharmacy Department of Hematology Cluj Napoca Romania

Laboratory of Biological Hematology University Hospital Dijon France

Servicio de Hematología Hospital Universitario Lucus Augusti Lugo Spain

The Confluent Private Hospital Nantes France

University Clinic for Hematology Oncology Hemostaseology and Palliative Care Johannes Wesling Medical Center University of Bochum Minden Germany

Wroclaw Medical University Wroclaw Poland

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