Richards M, Altisent C, Batorova A, Chambost H, Dolan G, de Moerloose P, Fraga M, Hermans C, Karafoulidou A, Klamroth R, Lassila R, Rothschild C
Paediatric Haematology Department, Children's Day Hospital, St James University Hospital, Leeds, UK.
Haemophilia. 2007 Sep;13(5):473-9. doi: 10.1111/j.1365-2516.2007.01478.x.
A survey of 21 haemophilia doctors, throughout Europe, who care for a total of approximately 5000 patients with bleeding disorders addressing practice and opinions regarding prophylaxis in patients aged 16-24 years and adults aged over 50 years, is presented. The outcome of adolescent patients who reduced or stopped prophylaxis was recorded. Eighteen of 19 respondents would consider modification of established prophylaxis in the adolescent age group, principal considerations being avoidance of risks of further concentrate exposure, predicted poor compliance and treatment costs. The preferred age for modification was 16-20 years, but there was very little consensus on the particular prophylactic regime recommended. Approximately, half of a cohort of 218 patients with severe haemophilia successfully reduced or stopped prophylaxis when they reached adolescence. Only 26 of 92 (28%) of the patient cohort who stopped prophylaxis, required reintroduction of a prophylactic regime and 12 of 59 (20%) of those who reduced the intensity of prophylaxis had to reintroduce a more intensive regime. A majority of respondents would consider starting prophylaxis in those over 50 years. There was no consensus as to indications for this practice or the nature of the prophylaxis protocol. We conclude that there is an absence of consensus on the management of patients with severe haemophilia, as they pass through adolescence and young adulthood, and reach the age of 50. Aggregate outcome data suggest a significant proportion of patients in the 18-22 years age range may be able to reduce or stop prophylaxis. A substantial number of older patients are on prophylaxis.
本文介绍了一项针对欧洲21位血友病医生的调查，这些医生总共负责约5000名出血性疾病患者，调查内容涉及16 - 24岁患者以及50岁以上成人预防性治疗的实践和观点。记录了减少或停止预防性治疗的青少年患者的结果。19位受访者中有18位会考虑调整青少年年龄组既定的预防性治疗方案，主要考虑因素是避免进一步接触凝血因子的风险、预计依从性差和治疗成本。调整的首选年龄为16 - 20岁，但对于推荐的具体预防方案几乎没有达成共识。在218名重度血友病患者中，约有一半在进入青春期时成功减少或停止了预防性治疗。在停止预防性治疗的92名患者队列中，只有26名（28%）需要重新采用预防性治疗方案，在降低预防性治疗强度的59名患者中，有12名（20%）不得不重新采用强度更高的方案。大多数受访者会考虑对50岁以上的患者开始预防性治疗。对于这种做法的适应症或预防方案的性质没有达成共识。我们得出结论，对于重度血友病患者在度过青春期和青年期并达到50岁时的管理，缺乏共识。汇总结果数据表明，18 - 22岁年龄范围内的相当一部分患者可能能够减少或停止预防性治疗。大量老年患者正在接受预防性治疗。
