Search

Introduction: Haploidentical peripheral blood stem cell transplantation ( Haplo-PBSCT) is a curative option in patient with hematology malignancy. In Mexico is very difficult to find an identical HLA donor , for this reason Haplo PBSCT it is an option in our public Health institution. Materials and methods: we aimed a retrospective study since (2017 -2021) in our Hospital Unidad Médica de alta Especialidad 25, Instituto Mexicano del Seguro Social (IMSS UMAE 25) Monterrey, Mexico. We include patients over 15 years of age who were undergo Haplo PBSCT with hematolgy malignancies . We performed Analysis of Overall and Relapse-free survival with kaplan meier curves and incidence of acute and chronic graft versus host disease (GVHD) and percentage of Transplant-Related Mortality (TRM). Results: we analized a 38 patients, the median age was 35 years (range 15-64). Of the total , 25 were male (65%) and 13 famale (34%). The rest of baseline data is described in table 1. The median time of neutrophil engrafment was days 17 ( 11-26 ) and platelets was 19.5 days ( 12-38 ). The Incidence of Primary Graft failure was 18 % ( seven patients). Patients with graft had 99-100% chimerism at day 100. The Incidence of aGVHD was 26 % and the cGVHD was 16%. DLI was used in four patients to treat passenger lymphocyte syndrome (2 patients) and ( 2 patients) with severe graft rejection. The principal cause of death was sepsis in eleven patients (29%). Two patients with acute leukemia (AML and ALL) died after development of post transplant lymphoproliferative disease. The TRM was 28.2%. There were no relapse-related deaths during follow-up after Haplo-PBSCT. The median of survival was 4.5 months ( range 1-44) with Overall Survival (OS) of 42 % at 3 years ( figure 1). Conclusion: Our analysis shows results that were comparable with those published in first world international transplant centers . Relapse remains the major cause of transplant failure, in our population the antigenic disparity between donor and recipient can strengthen the immune response versus disease. Haplo-PBSCT is a feasible transplantation method in patients with hematological malignancies in developing countries with limited resources. Figure 1 Figure 1. Disclosures No relevant conflicts of interest to declare.

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.

Introduction: Myelodysplastic syndromes (MDS) are a heterogeneous group of clonal hematological disorders characterized by ineffective hematopoiesis and variable degrees of peripheral cytopenias. Neither curative nor standard therapy has been available yet for the majority of patients with MDS. Patients with available matched donor may undergo an allogeneic bone marrow transplant with a potential cure rate 30-50%. Danazol is a synthetic anabolic steroid with properties similar to corticosteroids, such as inhibition of interleukin-1 and TNF-α production. Also, has demonstrated activity in immune cytopenias and aplastic anemia but its efficacy in MDS has shown contradictory results. Recently there has been increasing interest in telomere dysfunction in hematological diseases. Short telomeres are the major prognostic risk factor for clonal evolution to myelodysplasia and acute leukemia. Danazol not only prevents telomere loss, but a mean increase occurs with improvement observed early during the course of hormone therapy. In our center with poor access to new strategies and therapies the use of danazol remains as an attractive option for patients with MDS because of its low cost. In this study we aim to document the clinical evolution of patients diagnosed with MDS treated with danazol as first-line therapy in our institution. Methods: We include patients diagnosed with MDS according to WHO criteria treated with danazol between 2005 and 2015. Response was defined as one or more of the following criteria: a rise in the platelet count >25 x 109/I, an increase in hemoglobin >2 g/dl or disappearance of a previous requirement for red blood cell transfusion, or an increase in neutrophil count >0.5 x 109/L. All patients received packed red blood cell and platelet transfusions according to physician criteria. Patients were given intravenous or oral antibiotics as needed as prophylaxis. Criteria for stop danazol included: no response to treatment, toxicity or patient's refusal to continue treatment. Blood counts were preformed at least 1-month intervals for the first 4 months. We compared those who responded versus no responders using the Student's t test or Mann-Whitney U test as corresponded. Results: Forty-two patients with MDS were treated with danazol. Median follow-up was 12 months (range 3-75). Median dose used was 400 mg (range 100-600) orally in two divided doses. Mean duration of treatment for all patients was 6 months (range 3-72 months). The distribution by WHO subtypes included 26 refractory cytopenia with multilineage dysplasia (RCMD) (62%), 6 patients with refractory cytopenia with unilineage dysplasia (RCUD), 6 refractory anemia with excess blasts (RAEB), 2 refractory anemia with ring sideroblasts (RARS) and 1 MDS associated with isolated del(5q). Twenty-four (60%) patients presented clinical response. Response for patients with anemia was 23.8% (10/24), increase in absolute neutrophil count occurred in 36.8% (7/19), and 60% (24/40) presented an increase in platelet count. Time to initial response was 2 months (range, 1-8) while time to better response of 3 month (range, 1-8). Response was not associated to any MDS classification or administrated dose. Toxicity was mild and danazol was not discontinued in any patient. Side effects included three patients with gastrointestinal symptoms, and 4 patients reported weight gain. Median overall survival was 24 months (CI95% 5.1-42). Fifteen patients died (35.7%). Five patients progressed to AML. Conclusion: Different agents have been used in MDS. However, for the majority of patients with MDS no curative treatment exists. In conclusion our data suggest that Danazol may be effective in MDS with minimal toxicity, especially in patients with thrombocytopenia. Response was independent of severity, WHO classification and administrated dose. Table Clinical characteristics of responders versus non-responders Table. Clinical characteristics of responders versus non-responders Disclosures Gomez-Almaguer: 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; Celgene: Consultancy, Membership on an entity's Board of Directors or advisory committees; Amgen: Consultancy, Membership on an entity's Board of Directors or advisory committees.