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

Introduction Isolated acquired clotting factors deficiency (ACFDs) are rare hemorrhagic disorders mainly caused by antibodies directly neutralizing or accelerating the clearance of factors. Their rarity and heterogeneity may lead to difficulty in diagnosis and management.

Method Clinical features of patients with isolated ACFDs in the Blood Disease Hospital, Chinese Academy of Medical Sciences were collected from July 1997 to December 2021. Clinical information, laboratory test findings, therapy strategies and outcomes were analyzed retrospectively.

Result A total of 219 patients with isolated ACFDs were enrolled in our study, including 132 females and 87 males. The median age of all patients was 50 (4-90) years. The commonest isolated ACFDs was acquired hemophilia A (AHA) affecting 165 (75.3%) patients. Other types (non-AHA) were Factor (F) V deficiency (9.1%), FX deficiency (7.3%), FXII deficiency (2.3%), FXI deficiency (1.8%), FII deficiency (1.8%), FXIII deficiency (0.9%), von Willebrand factor (vWF) deficiency (0.9%), and FIX deficiency (1.5%). The most frequent comorbidities of patients with isolated ACFDs were malignancies (12.3%) and autoimmune diseases (11.0%). Two hundred and nine patients (95.4%) experienced at least one bleeding event, and two patients (one with FIX deficiency and another with FXII deficiency) presented with thrombotic symptoms. All 4 patients with FXII deficiency had no bleeding diathesis. Subcutaneous bleeding was most common with an incidence of 74.0% among all patients. The median bleeding score of all patients was 4 (0-12). Among all patients, 79.5% received replacement treatment and 91.3% underwent immunosuppressive therapy. Of 179 patients with records of survival outcomes, 81.6% achieved complete remission (CR) and 8.9% died. AHA patients achieved a significantly higher CR rate than non-AHA patients (87.6% vs 48.1%, p<0.0001).

Conclusion Clinical characteristics are varied across patients with different kinds of isolated ACFDs. AHA patients are more common and have a better prognosis than non-AHA patients. It is crucial for clinicians to pay more attention to recognizing and managing non-AHA patients to avoid morbidity and mortality.

Keywords: acquired clotting factor deficiency, diagnosis, treatment, prognosis

No relevant conflicts of interest to declare.