Home treatment of haemarthrosis with recombinant activated factor VII in patients with haemophilia A or B and inhibitors: experience from developing countries.
Home therapy for uncomplicated mild/moderate bleeding can decrease healthcare burden, promote self-esteem, reduce complications, and provide near-normal quality of life. To evaluate recombinant activated factor VII (rFVIIa) as home therapy for joint bleeds in Algeria, Morocco, Oman, Saudi Arabia, and United Arab Emirates. Twenty-seven patients aged more than 2 years with congenital haemophilia and inhibitors were monitored for up to 8 months after a first haemarthrosis episode treated with rFVIIa. Assessments were made by patients/caregivers with a standardized diary. The main measures included home-managed bleeds, haemostasis, and pain relief within 9 h after first injection. Additional analyses included convenience, time to pain resolution, and doses given within 48 h. Of 132 bleeds, 84 (63.6%) were managed at home. Of these, successful haemostasis (partial or complete) was achieved at 9 h in 87.8%, with pain relief for 84.0%. For all treatment settings, successful haemostasis at 9 h was achieved for 86.3% of bleeds, with pain relief achieved for 74.8% of bleeds. Higher initial dosing was associated with fewer injections. Median time to complete haemostasis was 48 h (spontaneous bleeds) and 24 h (traumatic bleeds). Median time to complete pain relief was 24 h for both bleed types. Satisfaction with treatment was high. No safety concerns were reported. Results from this observational study agree with previous data on the safety and efficacy of home treatment with rFVIIa and will help to increase awareness and aggregate experience, fostering confidence in home management of haemophilia patients with inhibitors in developing countries.
aService d'Hématologie, Hopital Isaad Hassani, CHU Beni-Messous, Alger, Algeria bCollege of Medicine and King Khalid University Hospital, King Saud University, Riyadh, Saudi Arabia cSheikh Khalifa Medical City, Abu Dhabi, UAE dNovo Nordisk Healthcare AG, Zurich Oerlikon, Switzerland eDepartment of Child Health, Sultan Qaboos University Hospital, Muscat, Oman fService of Medicine, Cheikh Zaid Hospital, Rabat, Morocco gDepartment of Haematology, Public Hospital of Mascara, Mascara hDepartment of Haematology, University Hospital of Annaba, Annaba iDepartment of Hematology, University Hospital of Sétif, Sétif jDepartment of Haematology, University Hospital Tlemcen, Tlemcen, Algeria kKing Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia lNovo Nordisk Canada Inc., Mississauga, ON, Canada mHaematology Department, University Hospital of Constantine, Constantine nUniversity Hospital Oran, Bd. Dr Benzerjeb, Oran, Algeria.
Meriem
M
Bensadok
Abdulkareem
A
Almomen
Azzam
A
Alzoebie
Soraya
S
Benchikh El Fegoun
Yasser
Y
Wali
Fati
F
Hamzy
Nemra Gaid
NG
Mehalhal
Fatiha
F
Grifi
Selma
S
Hamdi
Naima
N
Mesli
Tarek
T
Owaidah
Hossam Ali
HA
Saad
Nouredine Sidi
NS
Mansour
Hadj
H
Touhami
D003906:Developing Countries; D015942:Factor VIIa / Q000627:therapeutic use*; D005240:Feasibility Studies; D006395:Hemarthrosis / Q000628:therapy*; D006467:Hemophilia A / Q000150:complications*; D002836:Hemophilia B / Q000150:complications*; D006801:Humans; D011994:Recombinant Proteins / Q000627:therapeutic use*
D011994:Recombinant Proteins; D015942:Factor VIIa