Cansever Murat, Zietara Natalia, Chiang Samuel C C, Ozcan Alper, Yilmaz Ebru, Karakukcu Musa, Rohlfs Meino, Somekh Ido, Canoz Ozlem, Abdulrezzak Ummuhan, Bryceson Yenan, Klein Christoph, Unal Ekrem, Patiroglu Turkan
Department of Pediatrics, Division of Pediatric Immunology.
Department of Pediatrics, Dr. von Hauner Children's Hospital, Ludwig-Maximilians-Universität München, Munich, Germany.
J Pediatr Hematol Oncol. 2020 Mar;42(2):156-159. doi: 10.1097/MPH.0000000000001487.
Gain of function mutations in the p110δ catalytic subunit of the phosphatidylinositol-3-OH kinase (PIK3CD) classified as activated phosphoinositide 3-kinase delta syndrome (APDS) are the cause of a primary immunodeficiency characterized by recurrent sinopulmonary infections, and lymphoproliferation. Previously, autoimmunity and Epstein-Barr virus-related B-cell lymphoma have been documented for patients with APDS; here, we present a case that extends the picture, as the patient shows the full diagnostic criteria of hemophagocytic lymphohistiocytosis at 6 months of age. He experienced Hodgkin lymphoma as a 2.5-year-old baby. Next-generation sequencing returned a de novo heterozygous missense variant in PIK3CD (LRG_191t1: c.3061G>A; p.Glu1021Lys), confirming the primary immunodeficiency. After 2 courses of ifosfamide, cisplatin, and etoposide combined with brentuximab, the patient successfully underwent allogeneic hematopoietic stem cell transplantation from his HLA full matched sister, and he has been well for 18 months after that. The hematologist treating Hodgkin lymphoma and/or hemophagocytic lymphohistiocytosis should be vigilant about the possible underlying immune deficiency, and they should consider APDS in their differential diagnosis.
磷脂酰肌醇-3-羟基激酶(PIK3CD)的p110δ催化亚基功能获得性突变被归类为活化磷脂酰肌醇3-激酶δ综合征(APDS),是一种以反复鼻窦肺部感染和淋巴细胞增殖为特征的原发性免疫缺陷的病因。此前,已有文献报道APDS患者存在自身免疫和爱泼斯坦-巴尔病毒相关的B细胞淋巴瘤;在此,我们报告一例扩展了该病表现的病例,该患者在6个月大时就出现了符合噬血细胞性淋巴组织细胞增生症的全部诊断标准。他在2.5岁时患了霍奇金淋巴瘤。二代测序发现PIK3CD基因有一个新生的杂合错义变异(LRG_191t1: c.3061G>A;p.Glu1021Lys),证实了原发性免疫缺陷。在接受了2个疗程的异环磷酰胺、顺铂和依托泊苷联合 Brentuximab治疗后,患者成功接受了来自其 HLA 完全匹配姐姐的异基因造血干细胞移植,此后18个月情况良好。治疗霍奇金淋巴瘤和/或噬血细胞性淋巴组织细胞增生症的血液科医生应警惕可能存在的潜在免疫缺陷,并在鉴别诊断时考虑APDS。