Efficacy and safety of anagrelide in childhood essential thrombocythemia

Kanai, Ayako; Morokawa, Hirokazu; Matsuda, Kazuyuki; Shimazaki, Ei; Nakazawa, Yozo

doi:10.1111/ped.13330

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“…Notwithstanding, in a global analysis, the percentages of positivity were 31.7% for JAK2 V617F , 5.6% for CALR and 1% for MPL mutations Analyzing the eight cohorts of ET patients in whom all driver mutations were tested, the proportions of positivity became 31% for JAK2 V617F , 10% for CALR , and 2% for MPL and so 57% of the cases were triple-negative (Figure 1B). 12,14,16,17,19,20,22,33 These percentages are quite different from those in adults, among whom there is a much higher frequency of triple-negative cases and lower percentages of JAK2 - or CALR -positive cases. 64 As for PV patients, the number of studies reporting JAK2 V617F allele burden was small and the mean value was 24.1%.…”

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confidence: 88%

Characteristics and outcomes of patients with essential thrombocythemia or polycythemia vera diagnosed before 20 years of age: a systematic review

et al. 2019

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Although it is well known that myeloproliferative neoplasms occur in younger patients, few large cohorts of such patients have been reported. Thus, our knowledge about circumstances of diagnosis, outcome and treatment is limited, especially for children and young adults. We therefore performed a systematic review of cases, published since 2005, concerning patients aged below 20 years at the time of diagnosis of essential thrombocythemia or polycythemia vera. We identified 396 cases of essential thrombocythemia and 75 of polycythemia vera. The median age at diagnosis was 9.3 and 12 years, respectively, and females constituted 57.6% and 45% of the groups, respectively. Half of the patients were asymptomatic at diagnosis. The proportion of so-called triple negativity was high: 57% in essential thrombocythemia and 73% in polycythemia vera. The incidence of thrombosis during the follow-up was 9.3% in patients with polycythemia vera and less, 3.8%, in those with essential thrombocythemia. Venous events were predominant (84.2%), with hemorrhagic episodes being rarer (<5%). The risk of evolution also seemed low (2% to myelofibrosis and no reports of acute leukemia), but the median follow-up was only 50 months. Survival curves were not available. Half of the patients received an antithrombotic drug and 40.5% received a cytoreductive drug. All data should be analyzed with care because of the proportion of missing data (10.7% to 74.7%). This review highlights interesting points concerning this population of young patients with myeloproliferative neoplasms, including that such patients were identified as negative for all common driver mutations, but also shows the need for larger contemporary cohorts with longer follow-up to assess the true prognosis of these patients.

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