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在 Connect®髓系疾病登记处的学术和社区站点中,新发高危骨髓增生异常综合征和急性髓系白血病患者的移植转诊模式:潜在的护理障碍。

Transplantation Referral Patterns for Patients with Newly Diagnosed Higher-Risk Myelodysplastic Syndromes and Acute Myeloid Leukemia at Academic and Community Sites in the Connect® Myeloid Disease Registry: Potential Barriers to Care.

机构信息

Seidman Cancer Center, University Hospitals Cleveland Medical Center, Case Western Reserve University, Cleveland, Ohio.

Department of Hematology, Ohio State University, Columbus, Ohio.

出版信息

Transplant Cell Ther. 2023 Jul;29(7):460.e1-460.e9. doi: 10.1016/j.jtct.2023.04.011. Epub 2023 Apr 21.

Abstract

Hematopoietic stem cell transplantation (HCT) is indicated for patients with higher-risk (HR) myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). Age, performance status, patient frailty, comorbidities, and nonclinical factors (eg, cost, distance to site) are all recognized as important clinical factors that can influence HCT referral patterns and patient outcomes; however, the proportion of eligible patients referred for HCT in routine clinical practice is largely unknown. This study aimed to assess patterns of consideration for HCT among patients with HR-MDS and AML enrolled in the Connect® Myeloid Disease Registry at community/government (CO/GOV)- or academic (AC)-based sites, as well as to identify factors associated with rates of transplantation referral. We assessed patterns of consideration for and completion of HCT in patients with HR-MDS and AML enrolled between December 12, 2013, and March 6, 2020, in the Connect Myeloid Disease Registry at 164 CO/GOV and AC sites. Registry sites recorded whether patients were considered for transplantation at baseline and at each follow-up visit. The following answers were possible: "considered potentially eligible," "not considered potentially eligible," or "not assessed." Sites also recorded whether patients subsequently underwent HCT at each follow-up visit. Rates of consideration for HCT between CO/GOV and AC sites were compared using multivariable logistic regression analysis with covariates for age and comorbidity. Among the 778 patients with HR-MDS or AML enrolled in the Connect Myeloid Disease Registry, patients at CO/GOV sites were less likely to be considered potentially eligible for HCT than patients at AC sites (27.9% versus 43.9%; P < .0001). Multivariable logistic regression analysis with factors for age (<65 versus ≥65 years) and ACE-27 comorbidity grade (<2 versus ≥2) showed that patients at CO/GOV sites were significantly less likely than those at AC sites to be considered potentially eligible for HCT (odds ratio, 1.6, 95% confidence interval, 1.1 to 2.4; P = .0155). Among patients considered eligible for HCT, 45.1% (65 of 144) of those at CO/GOV sites and 35.7% (41 of 115) of those at AC sites underwent transplantation (P = .12). Approximately one-half of all patients at CO/GOV (50.1%) and AC (45.4%) sites were not considered potentially eligible for HCT; the most common reasons were age at CO/GOV sites (71.5%) and comorbidities at AC sites (52.1%). Across all sites, 17.4% of patients were reported as not assessed (and thus not considered) for HCT by their treating physician (20.7% at CO/GOV sites and 10.7% at AC sites; P = .0005). These findings suggest that many patients with HR-MDS and AML who may be candidates for HCT are not receiving assessment or consideration for transplantation in clinical practice. In addition, treatment at CO/GOV sites and age remain significant barriers to ensuring that all potentially eligible patients are assessed for HCT.

摘要

造血干细胞移植(HCT)适用于高危(HR)骨髓增生异常综合征(MDS)和急性髓系白血病(AML)患者。年龄、体能状态、患者脆弱性、合并症和非临床因素(如费用、与治疗地点的距离)均被认为是可影响 HCT 转诊模式和患者结局的重要临床因素;然而,在常规临床实践中,有多少符合条件的患者被转诊接受 HCT 治疗尚不清楚。本研究旨在评估在社区/政府(CO/GOV)或学术(AC)基地的 Connect®髓系疾病登记处登记的 HR-MDS 和 AML 患者中考虑 HCT 的模式,并确定与移植转诊率相关的因素。我们评估了 2013 年 12 月 12 日至 2020 年 3 月 6 日期间在 164 个 CO/GOV 和 AC 基地的 Connect 髓系疾病登记处登记的 HR-MDS 和 AML 患者中考虑和完成 HCT 的模式。登记处记录了基线和每次随访时患者是否被考虑接受移植。以下是可能的答案:“潜在有资格接受考虑”、“潜在无资格接受考虑”或“未评估”。各基地还记录了患者在每次随访时是否随后接受了 HCT。使用多变量逻辑回归分析,对年龄和合并症进行协变量调整,比较了 CO/GOV 和 AC 基地之间考虑 HCT 的比例。在 Connect 髓系疾病登记处登记的 778 例 HR-MDS 或 AML 患者中,CO/GOV 基地的患者接受 HCT 潜在有资格评估的可能性低于 AC 基地的患者(27.9%比 43.9%;P<.0001)。对年龄(<65 岁与≥65 岁)和 ACE-27 合并症分级(<2 与≥2)进行多变量逻辑回归分析,结果显示 CO/GOV 基地的患者与 AC 基地的患者相比,接受 HCT 潜在有资格评估的可能性显著降低(比值比,1.6;95%置信区间,1.1 至 2.4;P=0.0155)。在被认为有资格接受 HCT 的患者中,CO/GOV 基地的 45.1%(65/144)和 AC 基地的 35.7%(41/115)的患者接受了移植(P=0.12)。大约一半的 CO/GOV(50.1%)和 AC(45.4%)基地的患者被认为没有资格接受 HCT;最常见的原因是 CO/GOV 基地的年龄(71.5%)和 AC 基地的合并症(52.1%)。在所有基地中,有 17.4%的患者未被其治疗医生报告(因此未被考虑)接受 HCT(CO/GOV 基地为 20.7%,AC 基地为 10.7%;P=0.0005)。这些发现表明,许多可能适合接受 HCT 的 HR-MDS 和 AML 患者在临床实践中并未接受评估或考虑接受移植。此外,在 CO/GOV 基地接受治疗和年龄仍是确保所有潜在合格患者都接受 HCT 评估的重要障碍。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/8adb/11104018/c07a06601f71/nihms-1962639-f0001.jpg

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