Giordano Paola, Lassandro Giuseppe, Valente Michele, Molinari Angelo Claudio, Ieranò Paola, Coppola Antonio
1Department of Biomedical Science and Human Oncology, University of Bari "Aldo Moro", Bari, Italy.
Pediatr Hematol Oncol. 2014 Nov;31(8):687-702. doi: 10.3109/08880018.2014.930768. Epub 2014 Jul 9.
Hemophilias are the most known inherited bleeding disorders. The challenges in the management of hemophilic children are different from those in adults: prophylaxis regimen removed the hallmark of crippling disease with lifelong disabilities; individualized regimens are being implemented in order to overcome venous access problems. Presently, at least in high-income countries, advances in treatment of hemophilia resulted in continuous improvement of the patients' quality of life and life expectancy. Inhibitors remain the most severe complication of hemophilia therapy. The treatment' compliance is the key to achieve a successful management. The patient, his family, the medical and psychological team are the players of a comprehensive care system. The current management of hemophilic children is the example of huge resource investments enabling long-term benefits in particular quality of life as a primary objective of the healthcare process.
血友病是最广为人知的遗传性出血性疾病。血友病患儿的管理挑战与成人不同:预防方案消除了致残性疾病伴随终身残疾的特征;正在实施个体化方案以克服静脉通路问题。目前,至少在高收入国家,血友病治疗的进展使患者的生活质量和预期寿命不断提高。抑制剂仍然是血友病治疗最严重的并发症。治疗依从性是成功管理的关键。患者及其家人、医疗和心理团队是综合护理系统的参与者。目前对血友病患儿的管理是大量资源投入的典范,这些投入带来了长期益处,尤其是将生活质量作为医疗保健过程的首要目标。