A Single-Center Study of Patients with Isolated Acquired Clotting Factor Deficiency: Clinical Features, Treatments, and Prognosis

Introduction and aim: As a group of rare hemorrhagic disorders, isolatedacquired clotting factor deficiency (ACFD) is mainly caused by the existence of anti-factor antibodies or substances with absorbability of clotting factors. The most widely spread isolated ACFD is the acquired FVIII deficiency known as acquired hemophilia (AHA), and the remaining factors of deficiency are much less prevalent. Isolated ACFD is characterized by bleeding diathesis ranging from mild to life-threatening bleeding events. Their rarity and heterogeneity may lead to difficulty in diagnosis and management. We performed a retrospective study to analyze the characteristics of isolated ACFDs other than AHA, including acquired factor (F) II, FV, FIX, FX, FXI, FXII, FXIII, and VWF deficiencies, to enhance our understanding of these diseases. Methods: All patients with isolated ACFD other than AHA admitted to the Blood Disease Hospital, Chinese Academy of Medical Sciences, from July 1997 to December 2021, were retrospectively enrolled in the present study. The follow-up time started from when the patient was admitted to our center till March 2022. Clinical information, such as demographics, associated diseases, manifestations, laboratory tests, treatments, and prognosis, was collected. Results: A total of 54 patients with isolated ACFD admitted to our center were enrolled in our final analysis, including 28 males and 26 females. Acquired factor V deficiency (AFVD) was the most frequently diagnosed (20/54, 37.0%) isolated ACFD, followed by acquired factor X deficiency (AFXD) (n=16, 29.6%). The median age at diagnosis of all isolated ACFD patients was 55 (4-78) years. However, all four patients with acquired FII deficiency (AFIID) were young women (< 20 years). The median time from presenting bleeding diathesis or being found with an abnormal laboratory test to be diagnosed as isolated ACFD for all patients was 60 (2-2555) days. Underlying conditions were found in 35 (64.8%) patients with isolated ACFD, including malignancies (n=19, 35.2%), autoimmune diseases (n=12, 22.2%), and infections (n=4, 7.4%). Of the 19 patients associated with malignancies, 18 patients were secondary to hematologic malignancies. Plasma cell dyscrasias were the primary etiologies for patients with AFXD, with a proportion of 75.0% (12/16). A total of 44 (81.5%) patients with isolated ACFD experienced at least one bleeding episode. Subcutaneous hemorrhage was the most common bleeding phenotype affecting 61.4% (27