Medical Oncology Department, Pediatric Oncology Center, Beijing Children's Hospital, National Center for Children's Health, Beijing Key Laboratory of Pediatric Hematology Oncology, Key Laboratory of Major Diseases in Children, Ministry of Education, National Key Discipline of Pediatrics, Capital Medical University, Beijing, 100045, People's Republic of China.
Pathology Department, National Center for Children's Health, Beijing Children's Hospital, Capital Medical University, Beijing, 100045, People's Republic of China.
Orphanet J Rare Dis. 2022 Dec 12;17(1):432. doi: 10.1186/s13023-022-02575-4.
Subcutaneous panniculitis T-cell lymphoma (SPTCL) is a rare, cytotoxic T-cell lymphoma with which some patients have accompanying hemophagocytic syndrome (HPS). There is currently no standard treatment regimen. In the past, the most commonly used treatment was multidrug chemotherapy. In contrast, numerous case reports or small series suggest that immunosuppressive drugs could also be effective for some patients. Since this NHL subtype is extremely rare in children and adolescents, to improve the understanding of this disease and standardize its rational treatment, we retrospectively summarized the treatment regimens of 18 pathologically diagnosed children with SPTCL to compare the clinical efficacy of multidrug chemotherapy and immunomodulatory therapy.
The median age of onset was 11.1 years. Painless subcutaneous nodules or skin patchy lesions were found in all patients, most commonly involving the lower extremities and/or trunk. Before January 1, 2019, the treatment was mainly chemotherapy, and 10 patients were initially treated with chemotherapy, among whom was one patient who progressed during initial treatment, was voluntarily discharged and was subsequently lost to follow-up, one patient who died of disease progression, and the remaining 8 patients who all achieved sustained remission, with a complete remission (CR) rate of 80% (8/10). Corticosteroids combined with cyclosporine A or ruxolitinib were the most common initial immunosuppressive agents at our center after January 1, 2019 and had a CR rate of 71.4% (5/7). In addition, 1 patient achieved partial remission (PR) during follow-up, and one had autologous hematopoietic stem cell transplantation (AHSCT) after 4 months of drug withdrawal. There were 7 patients (38.9%, one case in chemotherapy group and six cases in immunotherapy group) with HPS and 4/5 screened patients (80%) with positive HAVCR2 gene mutations. The median follow-up was 17 months.
The prognosis of SPTCL is relatively good. Previous multi-drug and long-term chemotherapy treatment has clear efficacy, and recent immunomodulatory therapy as pre-chemotherapy therapy can also benefit patients.
皮下脂膜炎 T 细胞淋巴瘤（SPTCL）是一种罕见的细胞毒性 T 细胞淋巴瘤，部分患者伴有噬血细胞综合征（HPS）。目前尚无标准的治疗方案。过去，最常用的治疗方法是多药化疗。相比之下，许多病例报告或小系列研究表明，免疫抑制剂也可能对一些患者有效。由于这种 NHL 亚型在儿童和青少年中极为罕见，为了提高对这种疾病的认识并规范其合理治疗，我们回顾性总结了 18 例经病理诊断的 SPTCL 患儿的治疗方案，比较了多药化疗和免疫调节治疗的临床疗效。
发病中位年龄为 11.1 岁。所有患者均有无痛性皮下结节或皮肤斑片状病变，最常见于下肢和/或躯干。2019 年 1 月 1 日前，治疗主要为化疗，10 例患者初始接受化疗，其中 1 例初始治疗时进展，自愿出院后失访，1 例死于疾病进展，其余 8 例均获得持续缓解，完全缓解（CR）率为 80%（8/10）。2019 年 1 月 1 日后，我院中心最常用的初始免疫抑制剂是糖皮质激素联合环孢素 A 或芦可替尼，CR 率为 71.4%（5/7）。此外，1 例患者在随访期间获得部分缓解（PR），1 例患者在停药 4 个月后进行了自体造血干细胞移植（AHSCT）。有 7 例（38.9%，化疗组 1 例，免疫治疗组 6 例）患者发生噬血细胞综合征（HPS），5 例筛查患者中有 4 例（80%）HAVCR2 基因突变阳性。中位随访时间为 17 个月。
SPTCL 的预后相对较好。既往多药、长期化疗治疗效果明确，近期免疫调节治疗作为化疗前治疗也可使患者受益。
