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RAS相关自身免疫性白细胞增殖性疾病(RALD)的临床谱

Clinical Spectrum of Ras-Associated Autoimmune Leukoproliferative Disorder (RALD).

作者信息

Neven Quentin, Boulanger Cécile, Bruwier Annelyse, de Ville de Goyet Maëlle, Meyts Isabelle, Moens Leen, Van Damme An, Brichard Bénédicte

机构信息

Department of Pediatric Hematology and Oncology, Cliniques Universitaires Saint-Luc, Université catholique de Louvain, Avenue Hippocrate 10, 1200, Brussels, Belgium.

Department of Pediatrics, Grand Hôpital de Charleroi, Charleroi, Belgium.

出版信息

J Clin Immunol. 2021 Jan;41(1):51-58. doi: 10.1007/s10875-020-00883-7. Epub 2020 Oct 4.

Abstract

Ras-associated autoimmune leukoproliferative disorder (RALD) is a clinical entity initially identified in patients evaluated for an autoimmune lymphoproliferative syndrome (ALPS)-like phenotype. It remains a matter of debate whether RALD is a chronic and benign lymphoproliferative disorder or a pre-malignant condition. We report the case of a 7-year-old girl diagnosed with RALD due to somatic KRAS mutation who progressed to a juvenile myelomonocytic leukemia phenotype and finally evolved into acute myeloid leukemia. The case report prompted a literature review by a search for all RALD cases published in PubMed and Embase. We identified 27 patients with RALD. The male-to-female ratio was 1:1 and median age at disease onset was 2 years (range 3 months-36 years). Sixteen patients (59%) harbored somatic mutations in KRAS and 11 patients (41%) somatic mutations in NRAS. The most common features were splenomegaly (26/27 patients), autoimmune cytopenia (15/16 patients), monocytosis (18/24 patients), pericarditis (6 patients), and skin involvement (4 patients). Two patients went on to develop a hematopoietic malignancy. In summary, the current case documents an additional warning about the long-term risk of malignancy in RALD.

摘要

Ras相关的自身免疫性白细胞增殖性疾病(RALD)是一种临床实体,最初在因自身免疫性淋巴细胞增殖综合征(ALPS)样表型接受评估的患者中被发现。RALD究竟是一种慢性良性淋巴细胞增殖性疾病还是一种癌前病变,仍存在争议。我们报告了一例7岁女孩的病例,该女孩因体细胞KRAS突变被诊断为RALD,随后进展为青少年骨髓单核细胞白血病表型,最终演变为急性髓系白血病。该病例报告促使我们通过检索PubMed和Embase上发表的所有RALD病例进行文献综述。我们共识别出27例RALD患者。男女比例为1:1,发病时的中位年龄为2岁(范围3个月至36岁)。16例患者(59%)存在KRAS体细胞突变,11例患者(41%)存在NRAS体细胞突变。最常见的特征是脾肿大(26/27例患者)、自身免疫性血细胞减少(15/16例患者)、单核细胞增多(18/24例患者)、心包炎(6例患者)和皮肤受累(4例患者)。两名患者继而发展为血液系统恶性肿瘤。总之,当前病例记录了关于RALD长期恶性肿瘤风险的又一警示。

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