Assessment of Risk Factors, which Affect the Presence of Inhibitors in Children with Hemophilia A: The Albanian Case-control Study

Arlind Deveja,Anila Godo,Mirela Xhafa,Donjeta Bali,Mirzana Kapllanaj,Etleva Refatllari

doi:10.15344/2455-2364/2019/152

Abstract

Our study was designed to describe the correlation between patient and therapy characteristics with inhibitors presence in Albanian pediatric patients who suffer from hemophilia A (previously treated more than 50 days with FVIII). We investigated all the pediatric (0-14 years old) patients of Albania with hemophilia A (45 patients were initially enrolled). The main outcome was the inhibitor presence, confirmed by our laboratory with qualitative test. The test was performed only in 32 of our patients (71% of population). Thirteen of them (40.6%) are positive for inhibitors presence; this finding is higher than the percentage, which is referred in other countries. We found an association between intensity of replacement therapy (time between first treatment with FVIII and the 50th treatment) (t=2.267; df=30; p=0.031), frequency of treatment until the 50th exposure day (t=3.270; df=30; p=0.003) and the average dosage of FVIII (χ2=12,799; df=2; p=0.002) with inhibitor presence. Also the length of exposure episode (more than 5 days) was correlated with the inhibitor presence (t=6.131; df=1; p=0.015). Breastfeeding was associated with lower risk for inhibitors presence (t=7.166; df=; p=0.011). In our study the severity of the disease, family history for hemophilia, age, reason of the first exposure and surgical treatment during the first 50 days of exposure were not significant statistical correlated. Our findings suggest that the aggressivity (intensity, frequency, dosage) of the replacement therapy with exogenous FVIII have a primary role in inhibitors presence or development.

Highlights

Hemophilia A is a rare bleeding disorder, mostly inherited, which is characterized by the deficit of factor VIII of coagulation
The main focus of our study is to identify variables of replacement therapy for hemophilia A that are related with the formation of inhibitors
The sample (46 patients) included in the study consists of all pediatric patients (0-14 years old) who have been diagnosed with Hemophilia type A

Summary

Introduction

Hemophilia A is a rare bleeding disorder, mostly inherited, which is characterized by the deficit of factor VIII of coagulation. The only treatment for hemophilia A is the replacement therapy with exogenous factor VIII. After the improvement in viral inactivation techniques for eradication of transfusion disease transmission, the main complication of therapy nowadays is the inhibitor development [12]. The development of inhibitors directly affects the future therapy of these patients, exposing them more to complications of the disease [8]. A lot of risk factors are accused for inhibitor development as genetic risk factor [12], ethnicity [1], HLA genotype [6], family history [1], age of first treatment [9] and intensive treatment due to surgery or irregular therapy approach [4]

Methods

Results

Discussion

Conclusion