TY - JOUR
T1 - Pediatric severe factor XI deficiency
T2 - A multicenter study
AU - Barg, Assaf Arie
AU - Levy-Mendelovich, Sarina
AU - Budnik, Ivan
AU - Mandel-Shorer, Noa
AU - Dardik, Rima
AU - Avishai, Einat
AU - Brutman-Barazani, Tami
AU - Ifrah, Aviya Dvir
AU - Oren-Malek, Liat
AU - Yacobovich, Joanne
AU - Gilad, Oded
AU - Nakav, Sigal
AU - Fruchtman, Yariv
AU - Revel-Vilk, Shoshana
AU - Miskin, Hagit
AU - Kenet, Gili
N1 - Publisher Copyright:
© 2021 Wiley Periodicals LLC
PY - 2022/3
Y1 - 2022/3
N2 - Background: Factor XI (FXI) deficiency is a rare autosomal recessive bleeding disorder. Only scarce publications address its clinical features in children. The increased prevalence of FXI deficiency in Israel enabled data collection for this large multicenter cohort study. Objective: Some hemostatic challenges may be unique or more common in children, such as bleeding in the neonatal period or trauma-related injury. The current study was designed to explore the potential impact of these differences in children with severe FXI deficiency. Methods: Medical files of all children with FXI level under 15% followed at five tertiary centers were evaluated. The retrieved data comprised demographic and clinical characteristics, including bleeding episodes, surgical interventions, treatment strategies, as well as laboratory features. Results: Sixty children, whose median age at diagnosis was 4.2 years and their median FXI level was 4%, were included. Three children experienced triggered intracranial hemorrhage (ICH) and two children had major bleeds. No bleeding complications occurred in surgeries in which hemostatic treatment consisting mostly of tranexamic acid or fresh frozen plasma was applied (n = 45). In contrast, excessive bleeding was noted in 25% of surgical procedures performed without hemostatic preparation (p =.002). Conclusion: This study's findings confirm the generally favorable outcome of this rare bleeding disorder, with no spontaneous bleeds or cases of perinatal ICH. Nonetheless, proper diagnosis and adequate hemostasis in the surgical setting are imperative. Unlike previous studies in adults, our pediatric study suggests an association between the severity of FXI deficiency and bleeding tendency.
AB - Background: Factor XI (FXI) deficiency is a rare autosomal recessive bleeding disorder. Only scarce publications address its clinical features in children. The increased prevalence of FXI deficiency in Israel enabled data collection for this large multicenter cohort study. Objective: Some hemostatic challenges may be unique or more common in children, such as bleeding in the neonatal period or trauma-related injury. The current study was designed to explore the potential impact of these differences in children with severe FXI deficiency. Methods: Medical files of all children with FXI level under 15% followed at five tertiary centers were evaluated. The retrieved data comprised demographic and clinical characteristics, including bleeding episodes, surgical interventions, treatment strategies, as well as laboratory features. Results: Sixty children, whose median age at diagnosis was 4.2 years and their median FXI level was 4%, were included. Three children experienced triggered intracranial hemorrhage (ICH) and two children had major bleeds. No bleeding complications occurred in surgeries in which hemostatic treatment consisting mostly of tranexamic acid or fresh frozen plasma was applied (n = 45). In contrast, excessive bleeding was noted in 25% of surgical procedures performed without hemostatic preparation (p =.002). Conclusion: This study's findings confirm the generally favorable outcome of this rare bleeding disorder, with no spontaneous bleeds or cases of perinatal ICH. Nonetheless, proper diagnosis and adequate hemostasis in the surgical setting are imperative. Unlike previous studies in adults, our pediatric study suggests an association between the severity of FXI deficiency and bleeding tendency.
UR - http://www.scopus.com/inward/record.url?scp=85121810568&partnerID=8YFLogxK
U2 - 10.1002/pbc.29545
DO - 10.1002/pbc.29545
M3 - ???researchoutput.researchoutputtypes.contributiontojournal.article???
C2 - 34957695
AN - SCOPUS:85121810568
SN - 1545-5009
VL - 69
JO - Pediatric Blood and Cancer
JF - Pediatric Blood and Cancer
IS - 3
M1 - e29545
ER -