Introduction: Immune thrombocytopenia (ITP) is an autoimmune disorder that results from platelet destruction and production suppression. Frontline-therapy includes corticosteroids, intravenous immune globulin or anti-D immunoglobulin. Single-agent treatments have not been successful in inducing prolonged remission, as relapse will occur in approximately 50% of patients. Low-dose rituximab (100 mg) has been used for the treatment of ITP, showing an activity almost similar to the 375 mg/m2 standard dose. We and others have reported sustained response rates ranging from 58% to 76% using rituximab plus dexamethasone as a frontline therapy. Eltrombopag is a thrombopoietin nonpeptide mimetic that has been shown to raise platelet count in chronic ITP, and we have previously reported eltrombopag/dexamethasone as a feasible frontline therapy for ITP reaching 100% response rates.The lack of sustained response in many adult patients with newly diagnosed acute ITP has stimulated the search for a treatment that modifies the natural course of the disease. Objetive: We aim to evaluate efficacy, safety, and response duration of low-dose weekly rituximab (100 mg weekly, four doses) plus high-dose dexamethasone (40 mg PO, days 1-4) in combination with eltrombopag (50 mg, days 1-28) as frontline therapy in newly diagnosed primary ITP in an ambulatory setting. Methods: This is an ongoing open-label, single-arm study performed in patients with newly diagnosed ITP from the Hospital Universitario Dr. Jose Eleuterio Gonzalez in Monterrey, Mexico (Clinical trials.gov NCT02834286). Eligible patients are 16 years or older, with bleeding manifestations and/or a platelet count ²30×109/L, without previous treatment. Patients are excluded if they had active infection, pregnancy, or a malignant disease. A complete blood count is performed at baseline, on days 3, 5, 7 and then weekly for 28 days, monthly until month 6, and every 3 months thereafter. Partial and complete responses are defined as an increase in platelet counts ³30×109/L and ³100×109/L, respectively. Results: Ten consecutive patients have been enrolled from March 2015 until July 2016. Median age was 37 years (16-61). Six patients were women (60%) and four were men (40%). Median platelet account at diagnosis was 7 « 109/L (range 1.2-28). Median follow-up has been 7 months (range 1-13). All patients achieved at least a partial response (PR) at a median of 4 days (range 3-14). Complete response (CR) was achieved in 9 patients in a median of 7 days (7-22); all of them were still in CR at the end of treatment (Day 28). One patient lost response at 28 days and received a second high-dexamethasone course maintaining CR. No significant adverse effects have occurred during treatment, only 1 patient reported mild myalgia. No relapses have been documented until now.Currently, 8 patients remain in CR and 2 in PR. Conclusion:This is the first trial evaluating the response of low-dose rituximab in combination with eltrombopag and high-dose dexamethasone in newly diagnosed patients with ITP.Low-dose rituximab in combination with eltrombopag and high dose dexamethasone is a feasible frontline therapy for ITP. This drug combination showed high response rates achieved very rapidly, with a low incidence of side effectsand might represent an attractive option in patients with ITP and substantial bleeding. Table Characteristics and follow-up of patients M: Male, F: Female, CR: Complete Response, PR: Partial Response Table. Characteristics and follow-up of patients. / M: Male, F: Female, CR: Complete Response, PR: Partial Response Disclosures Gomez-Almaguer: Amgen: Consultancy, Membership on an entity's Board of Directors or advisory committees; Celgene: Consultancy, Membership on an entity's Board of Directors or advisory committees; Janssen: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Bristol: Consultancy, Membership on an entity's Board of Directors or advisory committees.
plete blood counts 10 months after the procedure. To the best of our knowledge this is the first report of a pair of monocygotic twins with concordant AML treated sequentially with an HLA-identical sibling donor graft procured in a single apheresis procedure and performed in an outpatient setting.
Background and Objectives: Although the therapy of advanced-stage Hodgkin Lymphoma (HL) has improved, up to 10% of patients with advanced-stage HL will not achieve complete remission (CR) with standard therapy, and 20%-30% of responders will relapse after treatment. The disease status before autologous stem cell transplantation remains the most important factor predicting outcome for patients with relapsed or refractory HL. The IGEV regimen (Ifosfamide, gemcitabine, vinorelbine and prednisolone) is a good option for salvage induction therapy before autologous stem cell transplantation, with a reported overall response rate of 81%. Due to the high cost of the IGEV regimen, and the financial constrains of our population, we decided to modify the original regimen using cyclophosphamide instead of ifosfamide. Design and methods: From January 2011 to January 2015, 19 patients with relapsed or refractory HL received the protocol consisting of four cycles of cyclophosphamide, gemcitabine, vinorelbine and prednisone (CGEV). It was administrated in outpatient setting. The CGEV regimen consists of cyclophosphamide 600 mg/m2 on days 1 to 3, gemcitabine 800 mg/m2 on days 1 and 4, vinorelbine 20 mg/m2 on day 4, prednisone 100 mg on days 1 to 4 and granulocyte colony-stimulating factor (G-CSF) from day 7 to 12 of each course. Four courses of chemotherapy were planned before autologous stem cell transplantation, after the third course, autologous peripheral blood stem cells (PBSC) were collected. Results: Nineteen patients with primary refractory or relapsed HL received the CGEV regimen. There were 10 males and 9 females. Nodular sclerosis was the most frequent histological subtype in 73%. At diagnosis, there were 7 (36%) patients in clinical stage IV and 12 (63%) in early stage I-II. The lungs were the most frequent extranodal organ involved. B symptoms were present in 9 (47%) patients. All our patients received as first line chemotherapy the regimen ABVD, the disease status before salvage induction chemotherapy with CGEV was: 9 (47%) patients with refractory disease and 10 (53%) patients were in relapse. The median number of cycles administrated was 4 (1-4). Only 14 patients received the total planned courses. The overall response to the CGEV regimen was 78%, of these, 6 (42.8%) patients achieved complete response and 5 (35.7%) partial response. Ten patients underwent autologous stem cell mobilization, obtaining an adequate CD34+ cell collection in all the patients. The median number of CD34+ cell was 9.08 x 106/kg (2.1-5.60 x 106/kg) with a median of 1 apheresis procedure. Hematologic grade 3 and 4 toxicity developed in 11 (59%) patients, no grade 4 extra-hematologic toxicity was observed. There were 3 treatment-related infections. Four patients died, 2 due to septic shock, and 2 with disease progression. Interpretation and conclusions: We describe a similar overall response rate of 78% with CGEV regimen compared with the 81% reported with IGEV regimen, it is less expensive and can be used in relapsed or refractory patients with HL, as salvage regimen before autologous stem cell transplantation. Disclosures No relevant conflicts of interest to declare.
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