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A toolkit for healthcare transition in adolescents with MPN

By Sheetal Bhurke

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May 23, 2025

Learning objective: After reading this article, learners will be able to cite a new clinical development in myeloproliferative neoplasms.


 

Adolescents and young patients with MPN experience the same complications of MPN as older adults, including thrombosis, bleeding, and disease transformation. Most young patients do not receive healthcare transition (HCT) support, leading to adverse outcomes such as medical complications, increased emergency department use, and poorer medication adherence. Kucine et al. developed an HCT toolkit comprising an educational checklist for physicians, a transition readiness assessment tool for patients, and consensus statements for clinical practice, published in Pediatric Blood & Cancer.1 

The toolkit was developed by an international panel of MPN physicians from France, Germany, Italy, and the USA. Pediatric and adult hematologists with expertise in caring for AYAs with MPNs also participated. Seven virtual panel meetings were held over 14 months.      

 

Key learnings1

The panel recommended incorporating a checklist of key points for physicians to discuss with AYAs with MPN during multiple visits. The checklist sections covered disease knowledge, disease management, and appointment management. 

The readiness assessment tool assesses patients’ disease knowledge, HCP disease monitoring, and treatment. The tool is recommended for administration every 6 months.

The panel recommended checking VAF of known mutations and a broader myeloid gene panel when feasible, ultrasound of the liver and spleen at diagnosis, and routine reimaging every 1–3 years.

Routine bone marrow follow-up evaluations for AYAs with stable clinical and laboratory findings were not recommended. 

Platelet counts alone should not be the basis for starting treatment. Cytoreduction agents were recommended for QoL-affecting symptoms or bleeding risks. 

For improving long-term outcomes, IFN was recommended as a first-line cytoreductive agent, particularly in young patients with JAK2V617F- or CALR-mutated MPN.

The toolkit represents an initial effort to improve the healthcare transition process for AYAs with MPN. Further research is needed to refine, implement, and evaluate the impact of the toolkit in real-world settings.

Abbreviations: AYA, adolescent and young adult; CALR, calreticulin; ED, emergency department; HCT, healthcare transition; IFN, interferon; MPN, myeloproliferative neoplasms; QoL, quality of life; VAF, variant allele frequency.

References

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