Long-term prophylaxis in patients with severe congenital factor XIII deficiency is not complicated by inhibitor formation.
: Congenital factor XIII (FXIII) deficiency is a rare bleeding disorder accompanied by a variety of bleeding events. Severely deficient patients require regular replacement therapy. With development of FXIII concentrate, the risk of viral infections transmitted by fresh frozen plasma and cryoprecipitate is diminished, but the possibility of inhibitor development remains a challenging issue in the management of these patients. The aim of this study was to assess FXIII inhibitor development in Iranian patients with FXIII deficiency (FXIIID). This study enrolled 50 (30 women and 20 men) patients with severe congenital FXIIID from southeast Iran who underwent long-term (more than 4 years or more than 50 injections) prophylaxis with FXIII concentrate (Fibrogammin P, Dade Behring, Marburg, Germany). We evaluated plasma FXIII activity and FXIII inhibitor on day 28 after the last prophylaxis administration. The method for investigation of FXIII inhibitor was based on Bethesda assay. The mean age of the study population was 13.8 ± 8.3 years. The minimum and maximum FXIII activity levels were less than 1-4.5% (mean ± SD, 2.6 ± 0.7%). Our investigations showed that all patients with severe form of FXIIID were treated without inciting inhibitor development. Despite long-term prophylaxis in the studied patients, none was found to have developed FXIII inhibitors.
aDepartment of Pediatrics Haematology and Oncology, Ali Ebn-e Abitaleb Hospital Research Center for Children; Department of Hematology, Allied Medical School, Iran University of Medical Sciences bBlood Transfusion Research Center, High Institute for Education and Research in Transfusion Medicine cDepartment of Hematology and Blood Transfuion, School of Allied Medical Sciences, Shahid Bheshti University of Medical Sciences, Tehran dEducation Development Center (EDC), Urmia University of Medical Sciences, Urmia eDepartment of Hematology and Blood Transfusion, School of Allied Medical Sciences, Iran University of Medical Sciences, Tehran fSchool of Nursing and Midwifery, Shahroud University of Medical Sciences, Shahroud, Iran *Akbar Dorgalaleh and Minoo Ahmadinejad contributed equally to the writing of this article.
Majid
M
Naderi
Minoo
M
Ahmadinejad
Maryam S
MS
Hosseini
Es'hagh
E
Moradi
Akbar
A
Dorgalaleh
Morteza
M
Shamsizadeh
D000293:Adolescent; D000328:Adult; D001323:Autoantibodies / Q000009:adverse effects / Q000032:analysis* / Q000187:drug effects; D002648:Child; D002675:Child, Preschool; D005176:Factor XIII / Q000009:adverse effects / Q000276:immunology* / Q000627:therapeutic use*; D005177:Factor XIII Deficiency / Q000150:complications / Q000151:congenital / Q000188:drug therapy* / Q000517:prevention & control; D005260:Female; D006470:Hemorrhage / Q000188:drug therapy / Q000517:prevention & control; D006801:Humans; D007492:Iran; D008297:Male; D055815:Young Adult
D001323:Autoantibodies; D005176:Factor XIII