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Treatment sequencing for anemic myelofibrosis
with Jean-Jacques Kiladjian & Angela Fleischman
Monday, February 26, 2024 | 16:30 CET
This independent educational activity is supported by Bristol Myers Squibb. All content is developed independently by the faculty. The funder is allowed no influence on the content of this activity.
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During the 2023 Global MPN Scientific Foundation (GMPNSF) Virtual Symposium entitled ‘Shining the light on pediatric, young adult, and pregnant patients living with MPNs’ held March 23–24, 2023, Kucine, Ianotto, and Randi discussed the special considerations in treating pediatric patients with myeloproliferative neoplasms (MPN).
The MPN Hub has previously reported on disease characteristics and outcomes in pediatric patients with MPN, highlighting the unique clinical presentations and outcomes in these patients compared with older adult patients. Here, we summarize the key considerations in diagnosing and treating pediatric patients with MPN.
A suggested diagnostic work-up for a child being evaluated for MPN is outlined in Figure 1.
Figure 1. Suggested diagnostic work-up for a child being evaluated for MPN*
CBC, complete blood count; LDH, lactate dehydrogenase; MPN, myeloproliferative neoplasm; PMF, primary myelofibrosis; TIBC, total iron binding capacity.
*Adapted from Kucine, et al.2
†Acquired von Willebrand’s disease generally occurs with extreme thrombocytosis, generally this test is performed in patients with platelet counts >1000 × 109/L or with bleeding symptoms.
‡If JAK2 mutation is identified, testing for CALR and MPL mutations in ET, PMF, or pre-PMF patients is not indicated.
It is particularly important to consider why a child diagnosed with MPN might need treatment.1 The impacts of long term cytoreductive medications, abnormal blood counts on vasculature, increased cancer risk, cardiovascular risk, and fertility and pregnancy complications must be taken into account.2
While consensus guidelines have been published for the management of MPN in adults, literature on the treatment of pediatric patients is limited, with no data available indicating that the same risk stratification and scoring systems are applicable to both adults and children. From the available literature, the consensus is that fully asymptomatic children with MPN should not receive cytoreductive treatments, whilst children who have experienced thrombosis or severe hemorrhage should receive cytoreductive treatment.
No cytoreductive agents are approved for use in children with MPN; however, off-label use of the following are reported in clinical practice.1,2
Each treatment presents specific risks and benefits; these should be discussed with the child’s family before a treatment plan is agreed.
The symposium offered a thorough and insightful discussion of special considerations in the diagnosis and treatment of pediatric patients with MPN. The speakers highlighted that available data are based primarily on experience in older patients and that it is paramount to adopt a flexible, adaptive approach in the pediatric setting. Whilst a high platelet count generally indicates treatment in adults, this is not necessarily the case in children. The risk/benefit profiles of cytoreductive treatments are very different between adults and children, these must be assessed carefully in the context of the full clinical picture; ensuring that children with MPN receive an appropriate diagnosis and treatment plan.
Disease characteristics and outcomes in pediatric and young adult patients with MPN
Summary of a study investigating the disease characteristics and outcomes for pediatric and young adult patients diagnosed with myeloproliferative neoplasms.
Special populations in MPN: pediatric, adolescent, and young adults with MPN
Triple negative mutations i.e., the absence of driver mutations in JAK2, MPL and CALR are reported in many young patients with myeloproliferative neoplasms (MPN).
Treatment outcomes with pegylated interferon in young patients with PV/ET
Myeloproliferative neoplasms (MPN) are very rare in young patients compared with adults. Not surprisingly, diagnostic criteria, risk classification, or treatment guidelines in...
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