Müskens Konradin F, Collot-d'Escury Winny N R, Dandis Rana, Haitjema Saskia, Kuball Jürgen, de Witte Moniek A, Bierings Marc, Lindemans Caroline A, Nierkens Stefan, Belderbos Mirjam E
Princess Máxima Center for Pediatric Oncology Utrecht The Netherlands.
Central Diagnostic Laboratory, University Medical Center Utrecht Utrecht University Utrecht The Netherlands.
Hemasphere. 2024 Dec 17;8(12):e70059. doi: 10.1002/hem3.70059. eCollection 2024 Dec.
Despite advances in allogeneic hematopoietic cell transplantation (HCT), poor graft function (PGF) remains an important complication with substantial morbidity and mortality. The investigation of preventive and therapeutic PGF treatments is hindered by inconsistencies in reported incidence and outcomes across studies, which may be explained by heterogeneity in PGF definition. To assess the impact of definition heterogeneity, we conducted a multicenter study, analyzing over 35.000 longitudinal blood counts from 427 pediatric and 405 adult HCT recipients. We compared the incidence, risk factors, and outcome of PGF, based on the three most common definitions. We identified 97 pediatric and 75 adult HCT recipients fulfilling at least one PGF definition. The 2-year cumulative incidence of PGF varied significantly depending on the definition used, ranging from 6.8% to 20% in children and 4.9% to 18% in adults. Two-year mortality for PGF patients ranged from 33% to 40% in children and 46% to 65% in adults. Notably, PGF patients identified solely by lenient definitions had similar mortality to HCT recipients with good graft function. Risk factors for PGF also varied by definition in both cohorts, and included older recipient age and cord blood transplantation. In conclusion, our study demonstrates that differences in PGF definition significantly impact the reported incidence, risk factors, and outcome. This underscores the need to harmonize PGF definitions across scientific studies, clinical practice, and transplant registries. Future studies, using standardized, quantitative thresholds for PGF, are required to determine optimal treatment strategies for both mild and severe forms of PGF.
尽管异基因造血细胞移植（HCT）取得了进展，但移植功能不良（PGF）仍然是一种重要的并发症，具有较高的发病率和死亡率。由于各研究报告的发病率和结果不一致，阻碍了对PGF预防和治疗方法的研究，这可能是由PGF定义的异质性所解释的。为了评估定义异质性的影响，我们进行了一项多中心研究，分析了来自427名儿科和405名成人HCT受者的超过35000份纵向血细胞计数。我们基于三种最常见的定义比较了PGF的发病率、危险因素和结果。我们确定了97名儿科和75名成人HCT受者符合至少一种PGF定义。PGF的2年累积发病率根据所使用的定义有显著差异，儿童中从6.8%到20%不等，成人中从4.9%到18%不等。PGF患者的2年死亡率在儿童中为33%至40%，在成人中为46%至65%。值得注意的是，仅通过宽松定义确定的PGF患者的死亡率与移植功能良好的HCT受者相似。两个队列中PGF的危险因素也因定义而异，包括受者年龄较大和脐血移植。总之，我们的研究表明，PGF定义的差异显著影响报告的发病率、危险因素和结果。这强调了在科学研究、临床实践和移植登记中统一PGF定义的必要性。未来需要使用标准化的、定量的PGF阈值进行研究，以确定轻度和重度PGF形式的最佳治疗策略。
