This is a PubMed case report so unable to post a link. For those not able to get PubMed here are the basic details and if you want the two case reports let me know and I will copy and paste them for you.
Refractory Immune Thrombocytopenia Successfully Treated With High-dose Vitamin D Supplementation and Hydroxychloroquine
Two Case Reports
Barry Bockow, Tamara Bockow KaplanDisclosures
J Med Case Reports. 2013;7(91)
Abstract and Introduction
Introduction: Immune thrombocytopenic purpura is thought to be characterized by an immune response against the host's own platelets. If the thrombocytopenia is severe, patients are initially treated with high-dose steroids. Other more toxic second line treatments are considered if steroids fail. Here, we report the case of two patients in whom conventional treatment was unsuccessful but who responded to hydroxychloroquine and high-dose vitamin D replacement therapy. To the best of our knowledge, this is the first description of successful treatment for immune thrombocytopenia with high-dose vitamin D and hydroxychloroquine.
Case presentation: Case 1: We report the case of a 79-year-old Caucasian man who presented with high titer antinuclear antibodies, positive anti-SSA/Ro autoantibodies and clinically was felt to have an overlap of systemic lupus erythematosus and/or Sjögren's syndrome with profound life-threatening thrombocytopenia. There was no evidence of underlying malignancy. The patient's platelet count significantly increased with vitamin D and hydroxychloroquine treatment, but upon vitamin D discontinuation his platelet levels plummeted. Hydroxychloroquine therapy was maintained throughout treatment. With reinstitution of high-dose vitamin D therapy, platelet counts were restored to normal levels.
Case 2: We also report the case of an 87-year-old Caucasian woman who presented with high titer antinuclear antibodies, positive anti-SSA/Ro autoantibodies and was felt to have an overlap of systemic lupus erythematosus and/or Sjögren's syndrome with immune thrombocytopenia; she also had severely low levels of 25-hydroxy vitamin D (17ng/mL). There was no evidence of underlying malignancy. She responded to high-dose vitamin D replacement and hydroxychloroquine treatment, thereby alleviating the need for high-dose steroid treatment. She remains in remission while taking vitamin D, hydroxychloroquine and very low-dose prednisone. No untoward side effects were observed in either patient.
Conclusions: In our two case reports, we found an association between vitamin D deficiency and immune thrombocytopenia where platelet levels responded to vitamin D treatment and hydroxychloroquine but not to prednisone. We believe there may be synergism between vitamin D supplementation and hydroxychloroquine. The mechanism by which high-dose vitamin D results in increased platelet counts in immune thrombocytopenia patients is unknown. However, vitamin D has long been thought to play an immunomodulatory role, which may include a dampened immune response in patients with immune thrombocytopenia or other autoimmune diseases.
Immune thrombocytopenic purpura (ITP) is thought to be an autoimmune disorder mediated by autoantibodies with no known etiology, and the incidence is approximately 2.5 per 100,000 persons per year. The goal of treatment is to keep the platelet count above 3×104/mm3 to prevent major internal organ bleeding.
Current treatment involves intravenous corticosteroids, immunosuppressants such as mycophenolate mofetil and azathioprine, Cytoxan® (cyclophosphamide), and intravenous immunoglobulin (IVIg). Anti-D immunoglobulin can only be given to RhD-positive individuals, thus limiting treatment options for RhD-negative persons. Rituximab, a chimeric monoclonal antibody, has also been tried as an experimental treatment, and combinations of therapies have been used with some success. Each of these therapies may be associated with significant side effects and efficacy is often temporary, requiring either additional and/or alternative treatment. Many of the side effects of steroids are well known and include hypertension, diabetes, osteoporosis and adrenal insufficiency. Several of these drugs are carcinogenic and there have been reports of malignancy formation after treatment, for example a high risk of acute myelogenous leukemia after Cytoxan® treatment. Response rates vary, and there is currently no consensus regarding the appropriate treatment protocol for this condition. Corticosteroids show efficacy in 50% to 80% of cases, but if treatment is stopped, the remission rate is only 10% to 30%. If patients are refractory to drug treatments, splenectomy is a second line option; two-thirds of patients who undergo splenectomy for ITP respond to the treatment. However, complications may arise from this surgical procedure including hemorrhage, abscess, sepsis, thrombosis and death and relapse of ITP occurs in a median of 15% of patients. These patients are at lifelong risk for infection from Streptococcus pneumoniae, Neisseria meningitides and Haemophilus influenza.
Therefore, caution must be used in the treatment of ITP. Aggressive treatment should be reserved for patients with severe and symptomatic thrombocytopenia, and more treatment options are needed to further guide disease management. A successful treatment for any disease should be non-toxic, inexpensive and easily administered. The need for novel treatments for ITP is therefore imperative.
Here, we report two cases of patients with severe ITP who responded to high-dose vitamin D and Plaquenil® (hydroxychloroquine) but who failed conventional therapies.