Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, 81 Irwon-ro Gangnam-gu, Seoul, 06351, South Korea.
Division of Rheumatology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea.
Ann Hematol. 2020 Sep;99(9):2095-2104. doi: 10.1007/s00277-020-04083-6. Epub 2020 May 21.
Secondary hemophagocytic lymphohistiocytosis (HLH) is a rare but fatal condition with various underlying disorders in adult patients and is diagnosed based on the HLH-2004 criteria, which were established based on experience in pediatric patients. However, few studies have prospectively evaluated the treatment outcomes and diagnostic performance of HLH criteria in adult patients with secondary HLH. Thus, we performed a single-center, prospective cohort study of adult patients with suspected HLH, and we analyzed treatment outcomes of patients enrolled between 2017 and 2019 as an interim analysis ( ClinicalTrials.gov Identifier: NCT03117010). Of the 73 patients with suspected HLH, 70 patients completed the evaluation for ≥ 7 of the HLH-2004 criteria, and 55 patients were diagnosed with HLH (55/73, 75%). Although serum ferritin and fever had a sensitivity of more than 90%, both had exceptionally low specificity, whereas soluble CD25 had a sensitivity of more than 90% and specificity of 80%. Forty patients with malignancy-associated HLH had B cell (n = 19) or T- or NK-cell (n = 21) lymphoid malignancy, whereas 15 patients had non-malignant disorders. Non-malignancy-associated HLH had greater than 90% 1-year overall survival (OS) after diagnosis of HLH, whereas that for malignancy-associated HLH was less than 40%. In conclusion, our study showed promising treatment outcomes for patients enrolled in our prospective cohort study, and prospectively demonstrated the diagnostic performance of the HLH-2004 criteria in adult patients with suspected HLH. Given that lymphoma was the most common cause of HLH in adults, thorough evaluation for lymphoma should be performed in adults with suspected HLH.
继发性噬血细胞性淋巴组织细胞增生症（HLH）是一种罕见但致命的疾病，在成年患者中有多种潜在疾病，并根据 HLH-2004 标准进行诊断，该标准是基于儿科患者的经验制定的。然而，很少有研究前瞻性评估 HLH 标准在继发性 HLH 成年患者中的治疗效果和诊断性能。因此，我们对疑似 HLH 的成年患者进行了单中心前瞻性队列研究，并对 2017 年至 2019 年期间入组的患者的治疗结果进行了中期分析（ClinicalTrials.gov 标识符：NCT03117010）。在 73 例疑似 HLH 的患者中，有 70 例患者完成了 ≥7 项 HLH-2004 标准的评估，有 55 例患者被诊断为 HLH（55/73，75%）。尽管血清铁蛋白和发热的敏感性均超过 90%，但特异性均非常低，而可溶性 CD25 的敏感性超过 90%，特异性为 80%。40 例恶性肿瘤相关性 HLH 患者中，B 细胞（n=19）或 T/NK 细胞（n=21）淋巴瘤，15 例患者有非恶性疾病。非恶性肿瘤相关性 HLH 在诊断 HLH 后 1 年的总生存率（OS）超过 90%，而恶性肿瘤相关性 HLH 的 OS 不足 40%。总之，我们的研究显示了前瞻性队列研究中入组患者有较好的治疗效果，并前瞻性地证明了 HLH-2004 标准在疑似 HLH 的成年患者中的诊断性能。鉴于淋巴瘤是成人 HLH 最常见的病因，疑似 HLH 的成人应进行彻底的淋巴瘤评估。
