Lifelong prophylaxis in a large cohort of adult patients with severe haemophilia: a beneficial effect on orthopaedic outcome and quality of life.
BACKGROUND
In the 1950s, Sweden initiated prophylaxis as a lifelong treatment for haemophilia. It was the first country to do so.


OBJECTIVE
To describe and evaluate dosing and outcome of prophylactic treatment in a large cohort of adult people with severe haemophilia who have been using prophylaxis most of their lives.


METHODS
Eighty-one patients born between 1932 and1992 were divided into two groups (Group A started prophylaxis at the age of ≤ 3 yr; Group B at three or more years of age) and evaluated retrospectively. Outcome was evaluated using the Hemophilia Joint Health Score (HJHS) and SF-36, a measure of quality of life.


RESULTS
The median number of joint bleeds per year was 0 in both study groups; however, the annual number of joint bleeds during the final 3 yr of observation was higher in group B than in group A (P < 0.006). Twenty-five of 30 patients in group A and 27/51 patients in group B had no joint bleeds in that period. Group A had significantly better joint outcomes than group B. Patients in group A experienced better physical and social health than those in group B.


CONCLUSIONS
This follow-up has provided for the first time more extensive and detailed information regarding the practice of prophylactic treatment in a large cohort of adults with severe haemophilia. The present study confirms that early start of prophylaxis continuing throughout the lifespan has been successful in virtually eliminating joint bleeds, preserving a close to normal joint status, and keeping patients healthy and able to live normal lives.
Malmö Centre for Thrombosis and Haemostasis, Skåne University Hospital, Malmö Department of Clinical Sciences, Lund University, Malmö, Sweden. mohammed.khawaji@med.lu.se
Mohammed
M
Khawaji
Jan
J
Astermark
Erik
E
Berntorp
D000293:Adolescent; D000328:Adult; D000368:Aged; D015331:Cohort Studies; D005164:Factor IX / Q000627:therapeutic use; D005169:Factor VIII / Q000627:therapeutic use; D005500:Follow-Up Studies; D006395:Hemarthrosis / Q000517:prevention & control; D006467:Hemophilia A / Q000150:complications* / Q000523:psychology / Q000628:therapy*; D006470:Hemorrhage; D006801:Humans; D008875:Middle Aged; D009985:Orthopedics / Q000379:methods*; D011788:Quality of Life; D013548:Sweden; D016896:Treatment Outcome
D005169:Factor VIII; D005164:Factor IX