Key Points• Eltrombopag/dexamethasone is a safe and effective combination for treating newly diagnosed ITP patients.• This treatment may prove useful in achieving lasting responses without additional immunosuppression in some patients.Immune thrombocytopenia (ITP) results from platelet destruction and production suppression. Eltrombopag belongs to a new class of thrombopoietin-mimetic drugs that raise platelet counts in ITP patients. We performed a single-arm study to assess the response to a single course of dexamethasone (40 mg by mouth, days 1-4) in combination with eltrombopag (50 mg, days 5-32) in 12 adults with newly diagnosed ITP in an outpatient setting. Median follow-up was 12.5 months. After therapy (day 33), 100% of patients achieved at least ‡30 3

Immunosuppressive therapy (IST) with anti-thymocyte globulin (ATG) plus cyclosporine A (CsA) is the standard treatment for aplastic anemia (AA) patients not eligible for allogeneic hematopoietic stem cell transplantation (HSCT). In the absence of ATG + CsA, androgens continue to be a treatment option. We documented the clinical evolution of AA patients treated with danazol instead of ATG + CsA. AA patients lacking both, human leukocyte antigen-matched donor and access to IST, were treated with danazol and modern support therapy and compared with those receiving a HSCT. Overall survival (OS), response rates, and death risk odds were calculated. Fifty AA patients were studied. Thirteen received a HSCT and 37 danazol and support therapy. Median daily dose of danazol was 400 mg (300 to 600 mg), administered during a median of 12 months. Five-year OS was higher for patients receiving HSCT (92%) compared to the danazol group (41%) (P = 0.001). Overall response rate was 46% (17/37) in the danazol-treated group and the median time to initial response was 3 months (1-27). Tendency to achieve remission was similar among severity groups (P = 0.094). The only adverse side effect recorded on the danazol group was an episode of gastrointestinal bleeding. No patient treated with danazol suffered clonal evolution of his/her disease. Although ATG plus CsA is the therapy of choice for AA patients without a donor when neither HSCT nor IST is available, danazol remains an acceptable therapeutic option for AA patients.

Treatment of autoimmune cytopenias remains unsatisfactory for patients refractory to first-line management. We evaluated the safety and efficacy of low-dose rituximab plus alemtuzumab in patients with steroidrefractory autoimmune hemolytic anemia and immune thrombocytopenic purpura. Nineteen of 21 included patients were assessable for response (11 with immune thrombocytopenic purpura, 8 with autoimmune hemolytic anemia). Treatment with 10 mg of alemtuzumab subcutaneously on days 1 to 3, plus 100 mg of rituximab intravenously weekly in 4 doses, was administered. The overall response rate was 100%, with complete response in 58%. IntroductionAutoimmune hemolytic anemia (AIHA) and immune thrombocytopenic purpura (ITP) are characterized by antibody-mediated destruction of red blood cells and platelets; as in other autoimmune disorders, B and T lymphocytes have important roles in their pathogenesis. 1 Corticosteroids are the standard initial treatment, with a 60% response rate. Patients unresponsive to corticosteroids usually experience a complicated course and show increased morbidity and mortality rates. Splenectomy and a large number of drugs have been used as second-line therapies with variable success, without evidence that any single agent is more effective than another. 2 Rituximab effectively depletes B cells involved in the production of antibodies and has been studied as a second-line treatment for ITP at full 3-10 and low doses 11,12 ; its use in ITP has led to a median overall response rate of approximately 60%. 7,9 Alemtuzumab, a humanized IgG monoclonal antibody specific for the CD52 antigen, expressed on lymphocytes, has been used in the treatment of lymphoproliferative disorders and recently for autoimmune diseases. 13,14 Preliminary results indicate that alemtuzumab may induce responses in patients with autoimmune cytopenias. [15][16][17][18] We evaluated the safety and efficacy of low-dose rituximab plus alemtuzumab in combination therapy in patients with steroidrefractory AIHA and ITP. The rationale for combining the 2 monoclonal antibodies was their reported single-agent activity, and the possibility of a synergistic effect, based on the fact that T lymphocytes are involved in the control of expansion of immunoglobulinproducing, autoreactive B-lymphocyte clones. MethodsApproval for the study was obtained from the Ethics Committee of the School of Medicine and University Hospital of the Universidad Autónoma de Nuevo León. Eligible patients were adults with active symptomatic ITP or AIHA, who had previously received treatment with at least one line of therapy, or followed a chronic relapsing course, and who provided written informed consent. Patients were excluded if they had active bacterial or viral infection, HIV, hepatitis C virus, cytomegalovirus immunoglobulin M (CMV-IgM)-positive serology, hepatitis B surface antigen positivity, pregnancy, or concomitant malignant disease. Pretreatment assessment included complete blood, reticulocyte, lymphocyte subset counts, serum immunoglobulins, direct an...

The combination of low-dose rituximab and high-dose dexamethasone as frontline therapy for adults with primary immune thrombocytopenia was effective and had a high overall response rate and a low incidence of relapse.