Premium
Essential thrombocythemia A retrospective case series
Author(s) -
Barg Assaf Arie,
Toren Amos,
Tamary Hannah,
Yacobovich Joanne,
SteinbergShemer Orna,
Gilad Oded,
Goldstein Gal,
Miskin Hagit,
RevelVilk Shoshana,
Rosenbeg Nurit,
Kenet Gili,
Zemer Vered Shkalim
Publication year - 2020
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.28183
Subject(s) - medicine , essential thrombocythemia , anagrelide , thrombocytosis , cohort , myelofibrosis , pediatrics , retrospective cohort study , thrombosis , deep vein , polycythemia vera , platelet , bone marrow
Background Essential thrombocythemia (ET) is rare in children, and pediatric guidelines are lacking. Therefore, we aimed to evaluate ET diagnosis and treatment in a pediatric cohort. Procedure Data of patients with ET from three hospitals were reviewed. Molecular diagnosis included JAK2 V617F, CALR , and MPL mutations. Patients were evaluated for acquired von Willebrand syndrome (AVWS). Follow‐up included clinical symptoms, adverse events, and treatment. Results Twelve children (median age: 8 years, range 1‐14.5) were included. Mean lag period between the first documentation of thrombocytosis until ET diagnosis was 36 months. Six patients were positive for JAK2 V617F and two for CALR mutations. In six of nine patients, AVWS was diagnosed. At diagnosis, only 33% of patients started therapy with aspirin ( n = 4) and hydroxyurea ( n = 2). In three of eight untreated patients, therapy was added during follow‐up. The cohort was followed for a median of 32.5 months (range: 4‐108 months). Clinical follow‐up disclosed vascular complications in 4 of 12 patients (deep vein thrombosis, n = 1; transient ischemic attack, n = 3). Two females experienced excessive bleeding; both were diagnosed with AVWS. Neither leukemia nor myelofibrosis evolved in our cohort. Conclusion Increased awareness to pediatric ET is warranted, as delayed diagnosis is common. Compared to adults, AVWS may be more prevalent among children with ET.