A survey on patient perception of reduced-intensity transplantation in adults with sickle cell disease
A survey on patient perception of reduced-intensity transplantation in adults with sickle cell disease
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DOI：
10.1038/sj.bmt.1705622
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发表时间：
2007-04-01
影响因子：
4.8
通讯作者：
Bareford, D.
中科院分区：
文献类型：
--
作者：
Chakrabarti, S.;Bareford, D.
降低强度条件反射（RIC）的发展和骨髓移植治疗儿童镰状细胞病（SCD）的成功也提高了在成人中重新审视这一前景的可能性。在支持疗法管理的慢性衰弱性疾病中，患者的感知对于任何潜在的治愈性疗法的进展都是至关重要的。为了探索这方面，我们进行了一项基于问卷调查的30名成人SCD。62%的患者愿意接受移植相关死亡率（TRM）> 10%;其中30%的患者TRM > 30%。64%的人认为移植失败（GF）风险> 10%是可接受的，41%的人认为风险> 30%是可接受的。只有50%的人可以接受不育。慢性移植物抗宿主病（GVHD）是不能接受的大多数（80%）。76%的患者有一个完整的兄弟姐妹，60%的患者愿意参加RIC移植的临床试验。这项调查表明，大多数患有SCD的成年人可能愿意考虑治疗性选择，如RIC移植，即使TRM或GF很高。主要关切涉及慢性移植物抗宿主病和不孕症。考虑到患者对治愈和并发症的看法，迫切需要在临床试验的框架内探索SCD患者的RIC移植。
The development of reduced-intensity conditioning (RIC) and the success of BMT for paediatric sickle cell disease (SCD) have raised the possibility of revisiting this prospect in adults as well. In a chronic debilitating disorder managed with supportive therapy, the patients' perception is critical in the advancement of any potential curative therapy. To explore this aspect, we undertook a questionnaire-based survey on 30 adults with SCD. Sixty two per cent of the patients were ready to accept a transplant-related mortality (TRM) > 10%; 30% of them a TRM > 30%. A risk of graft failure (GF) > 10% was acceptable to 64%, with a risk > 30% acceptable to 41%. Infertility was acceptable to only 50%. Chronic graft-versus-host disease (GVHD) was unacceptable to the majority (80%). Seventy six per cent% of patients had a full sibling and 60% were willing to participate in a clinical trial of RIC transplantation. This survey suggests that the majority of adults with SCD might be willing to consider a curative option such as RIC transplantation even with a high TRM or GF. The major concerns relate to chronic GVHD and infertility. There is an urgent need to explore RIC transplants in SCD patients within the framework of a clinical trial, considering patient perception regarding cure and complications.