Tag Archives: adolescent

Clinical Characteristics Defined in Adolescents and Young Adults With MPNs in Japan

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Vicki Moore, PhD

An analysis based in Japan identified differences in clinical characteristics between adolescents and young adults (AYA) and the non-AYA population among those with polycythemia vera (PV) or essential thrombocytopenia (ET). Findings were reported in the International Journal of Hematology.

The study was a large-scale retrospective analysis of AYA patients (aged 20 to 39 years) in the Japanese JSH-MPN-R18 registry who had been seen for an initial visit for PV or ET between April 2005 and March 2018. The study investigators evaluated clinical characteristics associated with PV or ET in this AYA cohort to increase understanding of myeloproliferative neoplasms (MPNS) in this population, in comparison with non-AYA patients from the registry.

In the registry, a total of 596 patients with PV and 1152 patients with ET were identified. There were 31 AYA patients among those with PV and 141 AYA patients among those with ET, corresponding to 5.2% of the total patients with PV and 12.2% of those with ET. In the AYA cohort, the median age at diagnosis was 33 years with PV and 32 years with ET.

AYA patients with PV had a lower median neutrophil ratio (71%) than non-AYA patients with PV did (78%; P <.01). Their median neutrophil count (6.5 x 109/L) was also lower than in the non-AYA population with PV (9.238 x 109/L; P =.03).

Among patients with ET, the AYA cohort showed lower values than non-AYA patients did for numerous parameters, such as median leukocyte count, neutrophil ratio, neutrophil count, lactate dehydrogenase level, and D-dimer level (P <.01 each). Palpable splenomegaly was also more common among AYA than non-AYA patients in the ET group (6.3% vs 2.3%, respectively; P =.02).

In patients with ET, the 5-year rate of hemorrhage-free survival (HFS) was higher for AYA patients (100%) than for non-AYA patients (93.9%; P <.01). For patients with PV, HFS did not significantly differ between AYA and non-AYA patients.

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Ph- MPN in Adolescent and Young Adult Patients

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June 26, 2024

Elizabeth L. Courville, MD

England JT, Szuber N, Sirhan S, et al. Clinical features and long-term outcomes of a pan-Canadian cohort of adolescents and young adults with myeloproliferative neoplasms: a Canadian MPN group study. Leukemia. 2024;38(3):570-578.

The classical BCR:: ABL1-negative myeloproliferative neoplasms (Ph- MPNs) polycythemia vera (PV), essential thrombocythemia (ET), and primary myelofibrosis (PMF) are typically diseases of older adults, with a median age at diagnosis within the sixth decade of life. In two large case series from cancer centers in the United States, adolescent and young adult (AYA) patients were reported to account for 11 to 12% of the Ph- MPNs evaluated.1,2  The data on AYA patients with Ph- MPNs is less robust than that available for their older counterparts, and this patient population may not be represented in cohorts used to develop prognostic scoring systems.

Recently, James T. England, MD, MSc, and colleagues investigated the clinical features and long-term outcomes of a cohort of 609 patients (17 pediatric patients aged <18 years and 592 patients aged 18-45 years) with Ph- MPNs from across eight participating centers in Canada. Initial diagnoses are shown in Figure 1. Clinical features from the current study cohort are compared with those of a 2018 Mayo Clinic AYA cohort1  (Table). The patients were diagnosed between 2000 and 2022, with MPN driver mutation analysis performed in 89% and next-generation sequencing (NGS) of clinically relevant myeloid genes performed in 48%. More than one-third of patients (211) had NGS testing first performed during initial disease phase, with a median time from diagnosis of 3.9 years (range, 0-29 years). Sixty-four patients had NGS first performed during the post-ET/post-PV secondary myelofibrosis (SMF) phase, while 19 had NGS first performed during the accelerated phase (AP)/blast phase (BP) of disease. Non-MPN driver mutations were detected in a higher proportion of patients evaluated during disease progression (secondary myelofibrosis or elevated blasts) than during initial disease phase, including more frequent high molecular risk (HMR) mutations (Figure 2). Mutations defined as HMR included pathogenic and likely pathogenic variants in ASXL1, EZH2, IDH1/2, SRSF2, TP53, and U2AF1Q157. Among those patients with NGS testing performed during the initial disease phase, additional mutations were most frequently detected in those with overt PMF (26%).

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