In a 50‑year‑old woman with systemic lupus erythematosus and Lambert‑Eaton myasthenic syndrome who has low factor VIII activity, markedly reduced von Willebrand factor antigen (VWF:Ag) and activity (VWF:RCo), very low ristocetin cofactor, normal multimer distribution, low immunoglobulin A (IgA) and immunoglobulin M (IgM), and elevated polyclonal immunoglobulin G (IgG), what is the most likely diagnosis and how should it be managed?

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Acquired von Willebrand Syndrome Secondary to Autoantibodies in SLE

This patient has acquired von Willebrand syndrome (AVWS) caused by autoantibodies against von Willebrand factor, a known complication of systemic lupus erythematosus, and should be treated with corticosteroids as first-line therapy, with rituximab reserved for refractory cases.

Diagnostic Reasoning

The laboratory pattern is pathognomonic for AVWS rather than congenital von Willebrand disease:

  • Markedly reduced VWF:Ag (19%), VWF activity (17%), and ristocetin cofactor (12%) with normal multimer distribution indicates rapid clearance of VWF-antibody complexes from circulation rather than a structural VWF defect 1, 2.

  • Low factor VIII activity (47%) occurs because factor VIII circulates bound to VWF; when VWF is cleared rapidly by autoantibodies, factor VIII is also depleted 2, 3.

  • Low IgA (118) and IgM (79) with elevated polyclonal IgG in the context of SLE suggests immune dysregulation and supports an autoantibody-mediated process 3.

  • The presence of SLE and Lambert-Eaton myasthenic syndrome (both autoimmune conditions) strongly supports an autoimmune etiology for the VWF deficiency 1, 2, 4.

Key Distinguishing Features from Congenital VWD

  • Normal multimer distribution rules out type 2A VWD (which shows loss of high-molecular-weight multimers) 2.

  • The severity of VWF reduction with normal multimers in an adult with autoimmune disease is characteristic of AVWS, not congenital disease 1, 4.

  • Congenital VWD would have presented with lifelong bleeding symptoms, not new-onset bleeding in a 50-year-old 1.

Confirmatory Testing

Perform mixing studies to detect VWF inhibitor:

  • Mix patient plasma 1:1 with normal pooled plasma and measure VWF:Ag levels 1, 5.

  • Presence of an inhibitor will cause decreased VWF:Ag in the mixture compared to expected levels 5, 3.

  • ELISA for anti-VWF antibodies (IgG, IgA, and IgM) can quantify the autoantibody 3, 4.

Management Algorithm

Acute Bleeding Management

  • Antifibrinolytics (tranexamic acid or aminocaproic acid) for mucosal bleeding 1.

  • Recombinant factor VIIa or activated prothrombin complex concentrate for severe bleeding unresponsive to other measures 1.

  • DDAVP is typically ineffective because autoantibodies rapidly clear any released VWF 2.

  • Factor VIII/VWF concentrates have limited efficacy due to rapid clearance by autoantibodies, though may provide transient benefit 2.

  • High-dose intravenous immunoglobulin (IVIG) can provide transient improvement by blocking antibody-mediated clearance 1, 2.

Definitive Treatment

First-line: Corticosteroids

  • High-dose prednisone (1 mg/kg/day) is the initial treatment of choice 2, 5, 4.

  • Treatment targets the underlying SLE and suppresses autoantibody production 2, 4.

  • Monitor VWF parameters (VWF:Ag, VWF:RCo, factor VIII) to assess response 4.

  • Expect normalization of VWF levels and multimer patterns with successful treatment 5, 4.

Second-line: Rituximab

  • Reserved for patients who fail corticosteroid therapy or have refractory disease 1.

  • Rituximab achieved complete remission in a severe type 3-like AVWS case that failed steroids and IVIG 1.

  • Standard dosing: 375 mg/m² weekly for 4 weeks 1.

Additional immunosuppression:

  • Consider adding azathioprine, mycophenolate, or cyclophosphamide for long-term control of SLE and prevention of AVWS relapse 1, 2.

Critical Pitfalls to Avoid

  • Do not assume congenital VWD in an adult with new-onset bleeding and autoimmune disease—always consider AVWS 1, 4.

  • Do not rely solely on DDAVP or factor VIII/VWF concentrates for bleeding management, as they are typically ineffective in autoimmune AVWS 2.

  • Ensure aggressive treatment of underlying SLE to prevent AVWS relapse—non-compliance with immunosuppression led to fatal relapse in one reported case 1.

  • Monitor VWF parameters regularly during and after treatment to detect early relapse 1, 4.

  • Do not discontinue immunosuppression prematurely—long-term maintenance therapy is essential to prevent recurrence 1.

References

Research

A case of autoimmune severe acquired von Willebrand syndrome (type 3-like).

Transfusion and apheresis science : official journal of the World Apheresis Association : official journal of the European Society for Haemapheresis, 2017

Research

Acquired von Willebrand syndrome in systemic lupus erythematodes.

Clinical and applied thrombosis/hemostasis : official journal of the International Academy of Clinical and Applied Thrombosis/Hemostasis, 2001

Research

Autoantibody to von Willebrand factor in systemic lupus erythematosus.

The Journal of laboratory and clinical medicine, 1993

Research

Acquired von Willebrand's syndrome with lupus-like serology.

The American journal of pediatric hematology/oncology, 1989

Professional Medical Disclaimer

This information is intended for healthcare professionals. Any medical decision-making should rely on clinical judgment and independently verified information. The content provided herein does not replace professional discretion and should be considered supplementary to established clinical guidelines. Healthcare providers should verify all information against primary literature and current practice standards before application in patient care. Dr.Oracle assumes no liability for clinical decisions based on this content.

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