Prophylaxis versus on demand treatment in adult patients with severe haemophilia A – FN Brno experience

Česká verzia

Authors: G. Chlupová ^1,2; P. Smejkal ^1,3; P. Ovesná ⁴; M. Penka ^1,2
Authors place of work: Oddělení klinické hematologie, Fakultní nemocnice Brno ¹; Interní hematologická a onkologická klinika, Fakultní nemocnice Brno a Lékařská fakulta Masarykova univerzita, Brno ²; Katedra laboratorních metod, Lékařská fakulta, Masarykova univerzita, Brno ³; Institut biostatiky a analýz Lékařské fakulty, Masarykova univerzita, Brno ⁴
Published in the journal: Transfuze Hematol. dnes,22, 2016, No. 2, p. 97-104.
Category: Souhrnné práce, původní práce, kazuistiky

Summary

Prophylactic treatment is standard care for most children with severe haemophilia A and maintaining this treatment in adulthood is recommended. However, the situation differs in adult patients with severe haemophilia A previously treated on demand. Between 2008–2015, we performed the following comparison of selected patients with severe haemophilia A. Over one year, we compared the frequency of bleeding episodes in a group of 8 patients aged 20–27, median (average) 22 (22.5) on continued prophylactic treatment from childhood with a group of 8 patients aged 26–65, median (range) 36 (37.5) treated on demand. Meanwhile, this same group was compared after performance of prophylactic treatment. In this group of patients, an individual period of 12 months was chosen and during which a specific patient did not undergo any invasive treatment, or had any serious bleeding episode after injury. Consequently, these patients were observed for another year (12 months), which ideally followed after their switch to prophylactic treatment. A group of patients with severe haemophilia A on prophylactic treatment was included as soon as they had been referred from the Department of Paediatric Haematology University Hospital Brno if they had not undergone in that particular period any invasive treatment or suffered any serious bleeding episode after injury. Patients were selected at a time when not subjected to any invasive procedure or suffering severe bleeding requiring hospitalization. Our results showed that by switching adult patients with frequent bleeding episodes from on demand treatment to prophylactic treatment, bleeding episodes were reduced by 82% (joint bleeds by 79%), without significantly increasing treatment cost. The median (range) of bleeding episodes in the young patient group with ongoing prophylaxis from childhood was 0 (average 0.375) times per year. The median (range) of bleeding joint episodes in older adult patients with on demand treatment was 27.5 (26). The median (range) 3.5 (4.8) number of bleeds decreased significantly with prophylaxis. Secondary prophylaxis in adult patients with severe haemophilia A significantly reduces the frequency of joint bleeds compared with on demand treatment. Prophylactic treatment of adults is certainly advantageous in certain complicated cases of haemophilia. In the future, costly surgery such as orthopaedic interventions could be postponed in these patients.

Key words:
haemophilia A – prophylactic treatment – treatment on demand – bleeding episodes – joint bleeds – haemophilic arthropathy

Zdroje

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