Background Multiple myeloma (MM) is a heterogeneous disease that is most frequently diagnosed in the elderly. Therefore, data on clinical characteristics and outcomes in the young population are scarce and it is recognized that it remains incurable even in this group of patients. We present here the outcomes of patients under 40 years old cohort in Latin-American countries. On behalf of GELAMM (Grupo de Estudio Latino-Americano de Mieloma Múltiple). Methods Retrospective international multicenter cohort study. We analyzed MM patients under 40 years old who received treatment in 6 Latin-American countries, between 2010 and 2018. Demographics and disease features were analyzed using descriptive statics. We examined treatment characteristics and response rates. The overall survival (OS) of the entire cohort was analyzed using Kaplan-Meier curves. Results Eighty-six patients of 6 countries were analyzed (Table1). The mean age was 35.4 years old, and 60% were male. The most frequent monoclonal component type was IgG followed by light chain MM. Risk determined by ISS was distributed in almost equal percentages. The most frequent cytogenetic alteration was the t (4;14) that was found in four patients out of 25 evaluated. The missing data were greater than 70%. Skeleton-related events were the most frequent clinical feature, followed by anemia and renal failure. Plasmacytomas and fractures were present in more than 20 percent of cases. With regard to treatment, VCD / CyBorD was the most used regimen, followed by VTD. The overall response rate (ORR) was 63%. Fifty-three patients received high dose therapy and autologous stem cell transplantation (62%). Only 8% received post-transplant consolidation, and 45% received maintenance therapy. The median OS of the entire cohort was 45 months, and a plateau in the survival curve was not observed, suggesting that patients continue relapsing over the time. Conclusion In this Latin American multicenter study, we found that the young population with MM has similar presentation characteristics to those of elderly patients. A significant amount of information is lost regarding the risk characterization, especially in regard with cytogenetics. With respect to treatment, less than half of the patients achieve very good partial response or better. It is striking that more than a third of this young patients did not access to high doses of chemotherapy and bone marrow transplantation. Maintenance therapy is offered to less than half patients. The median OS is lower than in other series of patients younger than 40 years, even than in the elderly cohorts. Prospective multicentric studies are required to elucidate the behavior of the disease in this group of patients. Disclosures Peña: Pfizer: Membership on an entity's Board of Directors or advisory committees; Janssen: Other: Congress inscription and flights; Biotoscana: Other: Congress inscription and flights; Novartis: Other: Congress inscription and flights; Tecnofarma: Other: Congress inscription and flights; Roche: Other: Congress inscription and flights. Rojas:Novartis: Membership on an entity's Board of Directors or advisory committees; Pfeizer: Membership on an entity's Board of Directors or advisory committees; Abbvie: Membership on an entity's Board of Directors or advisory committees; Roche: Membership on an entity's Board of Directors or advisory committees. Abello:Takeda: Other: Participation in advisory board meeting. Gomez-Almaguer:Takeda: Consultancy, Speakers Bureau; Celgene: Consultancy, Speakers Bureau; Amgen: Consultancy, Speakers Bureau; Janssen: Consultancy, Speakers Bureau; Teva: Consultancy, Speakers Bureau.
Background: Primary immune thrombocytopenia (ITP) is an acquired autoimmune disorder that results from accelerating platelet clearance, destruction, and production. Front-line therapy for newly diagnosed ITP includes corticosteroids, intravenous immune globulin, or anti-D immunoglobulin. However, after these single-agent therapies, relapses will occur in half of patients. We previously reported the safety, feasibility, and efficacy of the combination of dexamethasone, low-dose rituximab, and the thrombopoietin receptor agonist (TPO-Ra) eltrombopag as front-line treatment in newly diagnosed ITP. Romiplostim, another TPO-Ra is approved by the FDA for patients with chronic ITP. However, the safety, tolerability, and efficacy of romiplostim combined with low-dose rituximab, and high-dose dexamethasone in newly diagnosed ITP remain unknown. Objective: Our primary objectives were safety, and tolerability. Secondary objectives were initial response and relapse incidence. Methods: An open-label, single-arm study was performed in Hospital Universitario "Dr. José Eleuterio González" in Monterrey, Mexico (Clinical trials.gov NCT04588194). Eligible patients were newly diagnosed ITP patients, treatment-naïve, ≥18 years, with a platelet count ≤30×10 9/L. Patients were excluded if they had an active infection, pregnancy, or malignant disease. The treatment regimen was romiplostim (2 mcg/kg weekly, four doses), low-dose rituximab (100 mg weekly, four doses) and high-dose dexamethasone (40 mg PO, days 1-4). Dexamethasone was allowed up to 3 cycles. Partial (PR) and complete responses (CR) were defined as an increase in platelet counts ≥30×10 9/L (at least a doubling of the baseline count) and ≥100×10 9/L, respectively. Results: We included ten consecutive patients. The median age was 34.5 years (range, 17-63). Seven patients were men (70%). The median platelet account at diagnosis was 6x10 9/L (range 0-23), and median follow-up was 180 days (range 30-270). The median number of romiplostim doses was 3.5 (range 1-4), and three (30%) patients required dose adjustment due to thrombocytosis. All but one patient achieved response (CR or PR) at a median of 7 days (range 7-28). Five patients (50%) achieved CR at 28 days of treatment, and four patients (40%) PR. No significant adverse effects have occurred during treatment; one patient presented grade 1 myalgia and the other a grade 2 soft tissue infection. Five patients (50%) relapsed during follow-up. Recently, four (40%) patients remain in CR and three (30%) in PR. Conclusion: The combination of romiplostim, low-dose rituximab, and high-dose dexamethasone was safe and effective. This "total therapy" approach was associated with minimum side effects and rapid initial response. Prospective validation in a larger sample is needed. Figure 1 Figure 1. Disclosures Gomez-Almaguer: Janssen: Honoraria, Speakers Bureau; Roche: Honoraria, Speakers Bureau; Bristol-Myers-Squibb: Honoraria, Speakers Bureau; Takeda: Honoraria, Speakers Bureau. OffLabel Disclosure: Romiplostim in firs-line therapy for immune thrombocytopenia
Bone marrow (BM) aspiration plays an important role in hematologic malignancies diagnosis. Access and cost of diagnostic flow cytometry remains a problem in low and middle-income countries. In this context, morphological diagnosis by BM smear often represents the only means to rapidly diagnose our patients. Therefore, in this context obtaining the highest quality sample possible during the procedure is paramount. Despite being a well-known problem, evidence-based recommendations to improve BM aspirate quality are few, with studies evaluating factors associated with poor quality samples lacking. Objectives To determine factors associated with poor quality BM aspirates defined by an aspicular or hemodiluted sample in a hematology referral center. Materials and methods We conducted a retrospective study in our University Hospital and analyzed the BM smear samples stored in our center performed from October 2014 to December 2018. We collected and analyzed data based on diagnosis, age, gender, recent chemotherapy, and the variables of a complete blood count performed just before each BM aspiration. The quality of the BM smear was defined in any of the following: aspicular (without spicules), pauciaspicular (1-3 spicules), spicular (> 3 spicules), defining aspicular BM smear as non-diagnostic samples. Univariate analysis was performed looking for diferences between operators (in a 3-year residency program). In the other hand, in the multivariate analysis we seek to reveal the factors associated with obtaining hemodiluted (aspicular) bone marrow aspirate-smears. Results A total of 1,073 BM aspirates were evaluated. Hematology fellows performed 97% of BM aspirates; the remaining 3% were performed by attending physicians. In our analysis, 301 aspirates were aspicular, constituting 28.1% of the total number of aspirate smears. Most BM aspirates were performed for a diagnostic evaluation (66.3%) with the rest of the procedures for subsequent hematologic malignancy response assessments. In the univariate analysis, no differences were observed between operators. In a multivariate analysis the presence of an age >65 years (OR 3.1, 95% CI 2.3 to 4.1) and hemoglobin <6.0 g/dL (OR 2.7, 95% CI 1.4 to 4.5) at the time of the procedure were significantly associated to obtaining a non-diagnostic sample. Diagnosis, WBC count, platelet count, operator experience or other variables did not show statistical relevance. In our center, 18.81% of diagnostic patient samples that had acute leukemia were diagnosed without flow cytometry and through BM aspirate morphology alone. A second procedure to reach a diagnosis was necessary in 7.97% of the patients due to aspicular samples, obtaining a mean of diagnostic-treatment delay of 18.3 days (±5.7 days). Conclusions We found no differences between operators, emphasizing that there are other factors to consider in addition to a correct BM aspiration technique or operator experience. We believe this is crucial to recognize, especially in developing countries where morphological diagnosis remains the only means for the diagnosis or response evaluation of our patients. BM aspirate sample quality is multifactorial, being age and hemoglobin important factors. In addition, obtaining aspicular or hemodiluted samples represents not only a diagnostic challenge, but also delays the treatment of our patients. Disclosures Gomez-Almaguer: Celgene: Consultancy, Speakers Bureau; Takeda: Consultancy, Speakers Bureau; Teva: Consultancy, Speakers Bureau; Amgen: Consultancy, Speakers Bureau; Janssen: Consultancy, Speakers Bureau.
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