Paediatric Haematology and Oncology Department, University Hospital La Paz, Madrid, Spain.
Translational Research in Paediatric Oncology, Haematopoietic Transplantation and Cell Therapy, University Hospital La Paz Institute for Health Research - IdiPAZ, Madrid, Spain.
Pediatr Blood Cancer. 2024 Aug;71(8):e31053. doi: 10.1002/pbc.31053. Epub 2024 May 16.
Monomorphic post-transplant lymphoproliferative disorder (mPTLD) is a major cause of morbidity/mortality following solid organ transplant (SOT), with infection, mPTLD progression and organ rejection presenting equal risks. Balancing these risks is challenging, and the intensity of therapy required by individual patients is not defined. Although an increasing body of evidence supports the use of a stepwise escalation of therapy through reduction in immunosuppression (RIS) to rituximab monotherapy and low-dose chemo-immunotherapy, many centres still use B-cell non-Hodgkin lymphoma (B-NHL) protocols, especially when managing Burkitt/Burkitt-like (BL) PTLD. This study sought to define outcomes for children managed in the UK or Spanish centres using low-intensity first-line treatments.
Retrospective data were anonymously collected on patients younger than 18 years of age, with post-SOT mPTLD diagnosed between 2000 and 2020. Only patients given low-intensity treatment at initial diagnosis were included.
Fifty-six patients were identified. Age range was 0.9-18 years (median 10.7). Most (62.5%) had early-onset PTLD. Haematopathological analysis showed 75% were diffuse large B-cell like, 14.3% were BL and nine of 33 (27%) harboured a MYC-rearrangement. Stage III-IV disease was present in 78.6%. All but one had RIS, 26 received rituximab monotherapy and 24 low-dose chemo-immunotherapy, mostly R-COP. Intensified B-NHL chemotherapy was required in 10/56 (17.9%). There were a total of 13 deaths in this cohort, three related to PTLD progression. The 1-year overall survival (OS), event-free survival (EFS) and progression-free survival (PFS) were 92.8%, 78.6% and 80.2%, respectively.
R-COP provides an effective low-dose chemotherapy option. Escalation to more intensive therapies in the minority of inadequately controlled patients is an effective strategy.
同种异体移植后淋巴组织增生性疾病（mPTLD）是实体器官移植（SOT）后发病率/死亡率的主要原因，感染、mPTLD 进展和器官排斥的风险相等。平衡这些风险具有挑战性，并且个体患者所需治疗的强度尚未确定。尽管越来越多的证据支持通过减少免疫抑制（RIS）逐步升级治疗，直至使用利妥昔单抗单药和低剂量化疗免疫治疗，但许多中心仍在使用 B 细胞非霍奇金淋巴瘤（B-NHL）方案，特别是在治疗伯基特/伯基特样（BL）PTLD 时。本研究旨在确定在英国或西班牙中心使用低强度一线治疗的儿童患者的结局。
匿名收集了 2000 年至 2020 年间诊断为 SOT 后 mPTLD 的年龄小于 18 岁的患者的回顾性数据。仅包括在初始诊断时接受低强度治疗的患者。
共确定了 56 名患者。年龄范围为 0.9-18 岁（中位数为 10.7 岁）。大多数（62.5%）为早发性 PTLD。血液病理学分析显示 75%为弥漫性大 B 细胞样，14.3%为 BL，33 例中有 9 例（27%）存在 MYC 重排。疾病分期 III-IV 期为 78.6%。除 1 例患者外，其余患者均接受 RIS，26 例接受利妥昔单抗单药治疗，24 例接受低剂量化疗免疫治疗，主要为 R-COP。10/56（17.9%）名患者需要强化 B-NHL 化疗。该队列中共有 13 例死亡，3 例与 PTLD 进展相关。1 年总生存率（OS）、无事件生存率（EFS）和无进展生存率（PFS）分别为 92.8%、78.6%和 80.2%。
R-COP 提供了一种有效的低剂量化疗选择。在少数未得到充分控制的患者中，升级为更强化的治疗是一种有效的策略。
