10 results on '"Gabriel La Rocca"'
Search Results
2. Características clínicas y epidemiológicas de las neoplasias mieloproliferativas Philadelphia negativas en el sistema público de salud de Chile
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Ximena Valladares, Rafael Benavente, Christine Rojas, Camila Peña, Rodrigo Valenzuela, Virginia Monardes, Hernán López, Marcelo Abarca, Pilar León, Rocío Osorio, Erika Pérez, Pablo Soto, Gabriel La Rocca, Daniela Cardemil, Vivianne Torres, Marvila Intriago, María Elena Cabrera, and María Soledad Undurraga
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General Medicine - Published
- 2021
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3. Survival analysis of transplant-eligible newly-diagnosed multiple myeloma patients harboring t(4;14), t(14;16), and/or del(17p) in the real-world setting
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David Garrido, Irma Slavutsky, Eloisa Riva, Camila Peña, Natalia Schutz, Luz Tarín-Arzaga, Humberto Martínez-Cordero, Virginia Bove, Rocío Osorio, Mauricio Chandía, Cecilia Beltrán, Javier Schulz, Daniela Cardemil, Carolina Contreras, Carmen Gloria Vergara, Javiera Donoso, Marcela Espinoza, Gabriel La Rocca, Hernán López-Vidal, Pilar León, Christine Rojas Hopkins, Pablo Soto, Sandra Aranda, Vivianne Torres, Macarena Roa, Paola Ochoa, Patricio Jose Duarte, Guillermina Remaggi, Sebastián Yantorno, Ariel Corzo, Soledad Zabaljauregui, Claudia Shanley, Sergio Lopresti, Sergio Orlando, Verónica Verri, Luis Quiroga, Carlos García, Vanesa Fernández, Jhoanna Ramirez, Azucena Verduga, Alicia Molina, María Pacheco, William Mantilla, Alex Mite, Inés Reyes, Brenner Sabando, Francisca Ramírez, Claudia Sossa, Virginia Abello, Henry Idrobo, Kenny Mauricio Galvez Cardenas, Domingo Saavedra, Guillermo Quintero, Raimundo Gazitúa, Lina Gaviria, Rigoberto Gomez, Mónica Osuna, Alicia Henao-Uribe, Omar Cantú-Martínez, David Gómez-Almaguer, Yarely Itzayana García-Navarrete, Antonio Cruz-Mora, Yahveth Cantero-Fortiz, Guillermo J Ruiz-Argüelles, and Dorotea Fantl
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Cancer Research ,Oncology - Abstract
Cytogenetic abnormalities (CA) such as t(4;14), t(14;16), and del(17p), are associated with a poor prognosis in Multiple Myeloma (MM) patients. However, there is scarce information regarding the Latin-American population. This study aims to analyze the impact of t(4;14), t(14;16), and del(17p) on the progression-free survival (PFS) and overall survival (OS) of transplant-eligible newly-diagnosed MM (NDMM) patients in Latin America. Retrospective survival analysis based on the Grupo de Estudio Latinoamericano de MM (GELAMM) registry, including all adult patients with NDMM harboring CA t(4;14), t(14;16), and/or del(17p). Fifty-nine patients were included; the median age was 57 years, 55.9% males, 22% ISS-I, 25.4% ISS-II, and 47.5% ISS-III. The majority (89.8%) had 1 alteration, whereas 10.2% had del(17p) and t(4;14). The frequencies of CA were del(17p) in 61.0%, t(4;14) in 25.4%, and t(14;16) in 3,4%. Autologous stem cell transplantation (ASCT) was performed in 61.0% of cases. Five-year OS for the entire cohort was 60.8% and 5-year PFS was 28.1%. Bortezomib-based induction regimen (BBR) (P = 0.029), consolidation with ASCT (P0.001), and maintenance therapy (P = 0.004) were associated with an improved 5-year OS. In the multivariate analysis, ASCT was the only variable with a positive impact on OS (HR 0.11, 95% CI 0.033 to 0.34, P0.001). The median PFS presented a non-statistically significant benefit in BBR, ASCT, and maintenance therapy groups. BBR induction, ASCT, and maintenance therapy were associated with improved OS in high-risk NDMM patients.
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- 2022
4. Different outcomes for transplant-eligible newly diagnosed multiple myeloma patients in Latin America according to the public versus private management: a GELAMM study
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Ines Reyes, Sandra Aranda, Alicia Molina, Pilar León, Claudia Shanley, Christine Rojas Hopkins, Carmen Gloria Vergara, Macarena Roa, Humberto Martinez-Cordero, Carolina Contreras, Alicia Henao-Uribe, Veronica Verri, Guillermo J. Ruiz-Argüelles, Marcela Espinoza, Guillermina Remaggi, Eloisa Riva, Raimundo Gazitua, Jhoanna Ramirez, Vanesa Fernandez, Guillermo Quintero, Gabriel La Rocca, Yahveth Cantero-Fortiz, Soledad Zabaljauregui, Virginia Bove, Sergio Orlando, Claudia Sossa, Carlos Cristóbal Medina García, Omar Cantú-Martínez, Mónica Osuna, Kenny Mauricio Gálvez Cárdenas, Cecilia Beltran, Sergio Lopresti, Henry Idrobo, Pablo Soto, María Pacheco, Virginia Abello, Vivianne Torres, Luis Quiroga, Alex Mite, Patricio Duarte, Domingo Saavedra, Javiera Donoso, Paola Ochoa, Francisca Ramírez, Rigoberto Gomez, Yarely Itzayana García-Navarrete, Dorotea Fantl, Natalia Schutz, Ariel Corzo, Sebastian Yantorno, David Gómez-Almaguer, Rocío Osorio, Luz Tarín-Arzaga, Antonio Cruz-Mora, Daniela Cardemil, Javier Schulz, Camila Peña, Lina Gaviria, Brenner Sabando, Hernán López-Vidal, and Mauricio Chandia
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Cancer Research ,medicine.medical_specialty ,Latin Americans ,Newly diagnosed ,Transplantation, Autologous ,03 medical and health sciences ,0302 clinical medicine ,Autologous stem-cell transplantation ,Internal medicine ,Antineoplastic Combined Chemotherapy Protocols ,Overall survival ,medicine ,Humans ,Multiple myeloma ,business.industry ,Hematopoietic Stem Cell Transplantation ,Hematology ,medicine.disease ,Latin America ,Treatment Outcome ,Oncology ,Late diagnosis ,030220 oncology & carcinogenesis ,Proteasome inhibitor ,Private healthcare ,Multiple Myeloma ,business ,030215 immunology ,medicine.drug - Abstract
The aim of this study was to describe clinical and survival characteristics of transplant-eligible multiple myeloma (MM) patients in Latin America (LA), with a special focus on differences between public and private healthcare facilities. We included 1293 patients diagnosed between 2010 and 2018. A great disparity in outcomes and survival between both groups was observed. Late diagnosis and low access to adequate frontline therapy and ASCT in public institutions probably explain these differences. Patients treated with novel drug induction protocols, followed by autologous stem cell transplantation (ASCT) and maintenance, have similar overall survival compared to that published internationally.
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- 2020
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5. Mieloma múltiple en Chile: Respuesta a tratamiento en pacientes con mieloma múltiple elegibles para trasplante autólogo de progenitores hematopoyéticos
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Rocío Osorio, Mauricio Chandía, Carolina Contreras, Gabriel La Rocca, Jorge Rojas-Vallejos, Marcela Espinoza, Christine Rojas, Camila Peña, Javiera Donoso, Pablo Soto, Carmen Vergara, Daniela Cardemil, Sandra Aranda, and Hernán López-Vidal
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medicine.medical_specialty ,Cyclophosphamide ,business.industry ,Bortezomib ,medicine.medical_treatment ,Hematopoietic Stem Cell Transplantation ,General Medicine ,Hematopoietic stem cell transplantation ,medicine.disease ,Gastroenterology ,Dexamethasone ,Transplantation ,Thalidomide ,Autologous stem-cell transplantation ,Internal medicine ,medicine ,CTD ,Multiple Myeloma ,business ,Multiple myeloma ,medicine.drug - Abstract
Background The treatment of choice of newly diagnosed multiple myeloma (NDMM) is an induction with proteasome inhibitors followed autologous stem cell transplantation (HSCT). Since 2013, the treatment of these patients in the public system is based on CTD (cyclophosphamide, thalidomide, and dexamethasone). Aim To evaluate the response rates achieved with CTD, and the results of HSCT in patients with NDMM in the public setting. Material and Methods Data from patients considered as candidates for HSCT from different centers of the National Adult Antineoplastic Drug Program (PANDA, for its acronym in Spanish), diagnosed between 2013 and 2017, was analyzed. The response to treatment of first and second lines of treatment was evaluated, in addition to the results of HSCT. An optimal Response was defined as the sum of strict complete remission, complete remission and very good partial response (sCR, CR and VGPR). Results One hundred and seventy-seven patients were analyzed, 54% women, and 53% with IgG multiple myeloma. Information about the international staging system was retrieved in 127 patients (71%). Seventeen percent were ISS I, 22% in ISS II and 32% ISS III. CTD was used as first treatment in 106 patients (60%), and cyclophosphamide, bortezomib and dexamethasone (CyBorD) in 13 (7%). As first line, CTD had an overall response of 50.9%, and CyBorD of 76.9%. Thirty patients were treated with bortezomib as second line treatment. Forty patients (22%) underwent HSCT. The 5-year Overall Survival (OS) in transplanted patients and non-transplanted patients was 100 and 62% respectively (p < 0.01). Conclusions The response rate achieved by CTD in these patients is suboptimal. The response to CyBorD was better.
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- 2019
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6. [Clinical and epidemiological characteristics of the Philadelphia negative myeloproliferative neoplasms in Chile]
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Ximena, Valladares, Rafael, Benavente, Christine, Rojas, Camila, Peña, Rodrigo, Valenzuela, Virginia, Monardes, Hernán, López, Marcelo, Abarca, Pilar, León, Rocío, Osorio, Erika, Pérez, Pablo, Soto, Gabriel La, Rocca, Daniela, Cardemil, Vivianne, Torres, Marvila, Intriago, María Elena, Cabrera, and María Soledad, Undurraga
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Myeloproliferative Disorders ,Primary Myelofibrosis ,Mutation ,Humans ,Chile ,Janus Kinase 2 ,Polycythemia Vera ,Aged ,Thrombocythemia, Essential - Abstract
Philadelphia-negative myeloproliferative neoplasms (Ph-MPN) are chronic hematological disorders characterized by the overproduction of one or more mature myeloid blood cell lineages. Classical Ph-MPN are polycythemia vera (PV), essential thrombocytopenia (ET) and primary myelofibrosis (PMF).To assess the epidemiological, clinical and diagnostic characteristics of Ph-MPN in Chile.Retrospective review of medical records of all patients referred as MPN from 2012 to 2017. Patients with (9;21) translocation were excluded.Data of 462 cases with a median age of 69 years from 10 public hospitals was reviewed. ET was the most frequently Ph-MNP found. The incidence of Ph-MPN was 1.5 x 100.000 cases. The JAK2 V617F mutation study was performed in 96% of patients and only 30% had a bone marrow biopsy. Thrombotic events were observed in 29% of patients. Bleeding events were observed in 7%. Five-year overall survival was 87%.ET is the most frequent Ph-MPN. The mean incidence was lower than reported in the literature, in part because of a sub diagnosis.
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- 2021
7. [Response rates to first-line treatment in eligible patients to autologous stem transplantation in Chile]
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Camila, Peña, Jorge, Rojas-Vallejos, Marcela, Espinoza, Javiera, Donoso, Pablo, Soto, Daniela, Cardemil, Sandra, Aranda, Carolina, Contreras, Carmen Gloria, Vergara, Gabriel, LA Rocca, Rocío, Osorio, Hernán, López-Vidal, Mauricio, Chandía, and Christine, Rojas
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Adult ,Male ,Time Factors ,Hematopoietic Stem Cell Transplantation ,Kaplan-Meier Estimate ,Middle Aged ,Combined Modality Therapy ,Transplantation, Autologous ,Dexamethasone ,Disease-Free Survival ,Bortezomib ,Antineoplastic Combined Chemotherapy Protocols ,Humans ,Female ,Multiple Myeloma ,Cyclophosphamide ,Retrospective Studies - Abstract
Background The treatment of choice of newly diagnosed multiple myeloma (NDMM) is an induction with proteasome inhibitors followed autologous stem cell transplantation (HSCT). Since 2013, the treatment of these patients in the public system is based on CTD (cyclophosphamide, thalidomide, and dexamethasone). Aim To evaluate the response rates achieved with CTD, and the results of HSCT in patients with NDMM in the public setting. Material and Methods Data from patients considered as candidates for HSCT from different centers of the National Adult Antineoplastic Drug Program (PANDA, for its acronym in Spanish), diagnosed between 2013 and 2017, was analyzed. The response to treatment of first and second lines of treatment was evaluated, in addition to the results of HSCT. An optimal Response was defined as the sum of strict complete remission, complete remission and very good partial response (sCR, CR and VGPR). Results One hundred and seventy-seven patients were analyzed, 54% women, and 53% with IgG multiple myeloma. Information about the international staging system was retrieved in 127 patients (71%). Seventeen percent were ISS I, 22% in ISS II and 32% ISS III. CTD was used as first treatment in 106 patients (60%), and cyclophosphamide, bortezomib and dexamethasone (CyBorD) in 13 (7%). As first line, CTD had an overall response of 50.9%, and CyBorD of 76.9%. Thirty patients were treated with bortezomib as second line treatment. Forty patients (22%) underwent HSCT. The 5-year Overall Survival (OS) in transplanted patients and non-transplanted patients was 100 and 62% respectively (p0.01). Conclusions The response rate achieved by CTD in these patients is suboptimal. The response to CyBorD was better.
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- 2019
8. Real World Outcomes in Latin-American Patients with Multiple Myeloma Under 40 Years Old
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Paola Ochoa, Ariel Corzo, Sebastian Yantorno, Yarely Itzayana García-Navarrete, Hernán López-Vidal, Alicia Molina, Ines Reyes, Henry Idrobo, Cecilia Beltran, Pilar Papilco, Pilar León, Vanesa Fernandez, Marcela Espinoza, Luz del Carmen Tarín Arzaga, Claudia Shanley, Camila Peña, Lina Gaviria, Pablo Soto, Antonio Cruz-Mora, Gabriel La Rocca, Veronica Verri, Alicia Henao-Uribe, Rocío Osorio, Patricio Duarte, Daniela Cardemil, Humberto Martinez-Cordero, Sergio Lopresti, Guillermina Remaggi, Virginia Abello, Guillermo Quintero, Mauricio Chandia, Yahveth Cantero-Fortiz, Vivianne Torres, Omar Cantú-Martínez, Virginia Bove, Javier Schulz, Rigoberto Gomez, David Gómez-Almaguer, Sergio Orlando, Juan José García García, Macarena Alejandra Roa Salinas, Dorotea Fantl, Guillermo J. Ruiz-Argüelles, Brenner Sabando, Jhoanna Ramirez, Soledad Zabaljauregui, Natalia Schutz, Fiorella Villano, Sandra Aranda, Carolina Contreras, Domingo Saavedra, Javiera Donoso, Monica Osuna Pérez, Claudia Sossa, Carmen Gloria Vergara, Christine Rojas, Francisca M. Ramirez Aspiazu, Eloisa Riva, Luis Quiroga, and Alex Mite
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Oncology ,medicine.medical_specialty ,biology ,business.industry ,Anemia ,education ,Immunology ,Cell Biology ,Hematology ,medicine.disease ,Biochemistry ,Chemotherapy regimen ,Immunoglobulin G ,Transplantation ,Internal medicine ,biology.protein ,medicine ,Vocal cord dysfunction ,Plasmacytoma ,business ,health care economics and organizations ,Survival analysis ,Multiple myeloma - Abstract
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.
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- 2019
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9. Unequal Outcomes in Transplant Eligible Patients with Multiple Myeloma in Latin America: Differences between Public and Private Centers
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Guillermo J. Ruiz-Argüelles, Christine Rojas, Henry Idrobo, Vivianne Torres, Jhoanna Ramirez, Soledad Zabaljauregui, Carlos Cristóbal Medina García, Brener Sabando, Raimundo Gazitua, Francisca M. Ramirez Aspiazu, Vanesa Fernandez, Carolina Contreras, Patricio Duarte, Virginia Bove, Sergio Orlando, Rigoberto Gomez, Sergio Lopresti, Guillermo Quintero, Omar Cantú-Martínez, Pilar Papilco, Antonio Cruz-Mora, Camila Peña, Lina Gaviria, Paola Ochoa, Claudia Sossa, Virginia Abello, Alicia Molina, Ines Reyes, Claudia Shanley, Kenny Galvez, Luis Quiroga, Alex Mite, Pilar León, Daniela Cardemil, Javier Schulz, Marcela Espinoza, Luz del Carmen Tarín Arzaga, Sandra Aranda, Pablo Soto, Alicia Henao-Uribe, Gabriel La Rocca, Mauricio Chandia, Dorotea Fantl, Yarely Itzayana García-Navarrete, Ariel Corzo, Sebastian Yantorno, Natalia Schutz, Fiorella Villano, Domingo Saavedra, Javiera Donoso, Monica Osuna Pérez, Guillermina Remaggi, Yahveth Cantero-Fortiz, Eloisa Riva, Hernán López-Vidal, Cecilia Beltran, Carmen Gloria Vergara, Macarena Roa, Veronica Verri, Rocío Osorio, and David Gómez-Almaguer
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Pediatrics ,medicine.medical_specialty ,business.industry ,Proportional hazards model ,Immunology ,Gold standard ,Cell Biology ,Hematology ,medicine.disease ,Biochemistry ,Transplantation ,Median follow-up ,medicine ,Progression-free survival ,business ,Survival analysis ,Multiple myeloma ,Cohort study - Abstract
Background Multiple myeloma (MM) is a frequent hematologic malignancy. The current gold standard frontline strategy includes a proteasome inhibitor (PI)-based induction, followed by autologous stem cell transplant (ASCT). Access to novel drugs in Latin America (LA) is limited. ASCT is available in most countries, but real access to it is highly heterogeneous. Data regarding patients´ outcomes in candidates to ASCT in the region is scarce. The aim of this study was to describe clinical characteristics and outcomes of MM transplant eligible patients in LA countries. Material and Methods Retrospective international multicenter cohort study. Consecutive MM transplant- eligible patients diagnosed between 2010 and 2018 from participating centers in Chile, Argentina, Ecuador, Mexico, Colombia, and Uruguay were included. Data were collected from clinical records in a standardized report form. We analyzed clinical characteristics at diagnosis and frontline therapy outcomes, including ASCT. Transplant-eligible patients were defined as fit patients younger than 66 years old. Active MM and response to treatment were defined according to current IMWG criteria. Inclusion criteria: 1.- Patients with newly diagnosed active MM between 2010 and 2018. 2.- Older than 18 years, and younger than 66 years. 3- Candidates for ASCT according to the evaluation of the attending physician Exclusion criteria: 1- Lack of minimum data in the clinical history 2- Plasma cell leukemia, AL amyloidosis or solitary plasmacytoma. 3- HIV infection 4-No consent and/or Ethics Committee approvals. Statistical analysis A descriptive statistic has been done. Comparisons of characteristics between groups was made usingT-student, Chi2 or ANOVA, as appropriate. Survival analysis was performed using Kaplan-Meier curves. Comparisons of survival between groups were made by the logarithmic recording method and the calculations of the risk relationships by Cox regression. Statistical analysis was performed by using STATA 13. Results We included 1293 patients in the study, 363 from Chile, 395 from Argentina, 209 from Colombia, 45 from Ecuador, 151 from Mexico, and 130 from Uruguay. The main characteristics at diagnosis and therapeutic strategies are shown in Table 1. Optimal response (sCR, CR and VGPR) was achieved in 38% of the patients in the cyclophosphamide, bortezomib, and dexamethasone (CyBorD) group, in 46% in the bortezomib, thalidomide, and dexamethasone (VTD) group, and in 36% in the cyclophosphamide, thalidomide, and dexamethasone (CTD) group, the 3 main induction regimens used. Only 53% of patients finally received ASCT. Significant differences were found between both groups, private and public institutions, regarding burden of symptoms, ISS staging, access to PI based induction, ASCT completion and adequate maintenance, with patients from the latter being more symptomatic, and receiving suboptimal therapy. FISH analysis was performed in less than 50% of patients, both in the public and private setting. With a median follow up of 34 months (range 1-113), median overall survival (OS) was 86 months. The 5-year progression free survival (PFS) was 38% and 5- year overall survival (OS) was 64%. When comparing public vs private settings, 5 year OS was 45% vs 80%, with a median OS of 56 months vs not reached, respectively (P In the multivariable analysis renal failure (p=0.03), achieving less than VGPR response (p Conclusion This is the largest report on transplant eligible patients with MM in LA. Great inequities are shown between public and private health systems. Survival in transplant-eligible patients is lower than that described in other regions. Only one third of patients had FISH performed. This means that very few patients are treated with a risk-based induction in LA. Patients in the public setting are diagnosed with a more symptomatic disease, probably due to a late diagnosis. OS is significantly worse in the public setting. This might be explained by the significant differences in access to PI-based induction, ASCT and maintenance between private and public institutions, with patients from the latter receiving suboptimal frontline therapy and maintenance. Reasons for 47% of potential candidates not being transplanted merit further analysis. Table 1 Disclosures Peña: Novartis: Other: Congress inscription and flights; Tecnofarma: Other: Congress inscription and flights; Roche: Other: Congress inscription and flights; Biotoscana: Other: Congress inscription and flights; Janssen: Other: Congress inscription and flights; Pfizer: Membership on an entity's Board of Directors or advisory committees. Rojas:Roche: Membership on an entity's Board of Directors or advisory committees; 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. Abello:Takeda: Other: Participation in advisory board meeting. Gomez-Almaguer:Amgen: Consultancy, Speakers Bureau; Janssen: Consultancy, Speakers Bureau; Teva: Consultancy, Speakers Bureau; Takeda: Consultancy, Speakers Bureau; Celgene: Consultancy, Speakers Bureau.
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- 2019
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10. Characterization of Philadelphia-Negative Myeloproliferative Neoplasms in the Chilean Public Health System: Multicentric Study
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Claudia Gajardo, Virginia Monardes, Rodrigo Valenzuela, Marcelo Abarca, Hernán López-Vidal, Ximena Valladares, Maria Elena Cabrera, Pilar León, Marvila Intriago, Daniela Cardemil, Gabriel La Rocca, Camila Peña, Erika Pérez, Rocío Osorio, Vivianne Torres, Christine Rojas, and Pablo Soto
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Philadelphia negative ,Pediatrics ,medicine.medical_specialty ,Ruxolitinib ,Second line treatment ,business.industry ,Public health ,Immunology ,Lactate dehydrogenase measurement ,Myeloproliferative disease ,Cell Biology ,Hematology ,medicine.disease ,Biochemistry ,Platelet Count measurement ,Medicine ,business ,Myelofibrosis ,medicine.drug - Abstract
Background: Philadelphia-negative Myeloproliferative Neoplasms (Ph-MPN) are chronic hematological disorders characterized by the overproduction of one or more mature myeloid blood cell lineages. Classical Ph-MPN are Polycythemia Vera (PV), Essential Thrombocytopenia (ET) and Myelofibrosis (MF). The diagnosis includes clinical, histological and molecular features. There are not data from Chile. The aim of this study is to determinate epidemiological, clinical, diagnostic and therapeutic characteristics of Ph-MPN in our country. Methods: Descriptive and retrospective study. We reviewed the database of the Molecular Biology Laboratory at the Hospital del Salvador, a national reference laboratory, from 2012 to 2017. All patients referred as Ph-MPN were included. We reviewed the clinical records to obtain clinical information. Results: Clinical data was obtained from 468 cases from 12 public hospitals in Chile. Median age at diagnosis was 70 years. Female to Male ratio= 1,15:1, without significant differences between Ph-MPNs. ET was the most frequently Ph-MNP found, accounting for 49,4% of all Ph-MPN, followed by PV (37%) and MF (10,4%). A 66,2% of ET was JAK2 V617F+. Bone marrow biopsy was performed in 35% of ET cases. Only 7,8% had cytogenetic study. Splenomegaly was found in 8%. Thrombosis was observed in 23,8%. The median platelet count was 842x109/L. All patients received hydrea +/- aspirin or oral anticoagulation. Of the total of PV, 86,6% was JAK2+. Bone marrow biopsy was performed in a quarter of the cases. Thrombosis frequency was 14,5%. A 29% had splenomegaly. Median hemoglobin level was 18 gr/dl. All patients were treated with aspirin +/- phlebotomy and about half of them required cytoreduction. Two patients were refractory to hydrea and used ruxolitinib as second line treatment. A 63,3% of the MF were JAK-2+. Bone marrow biopsy was performed in 59% and 20% had a cytogenetic study. Only one fifth of patients had LDH measurement at diagnosis. Splenomegaly was observed in 75,5% of cases. Thrombosis frequency was 13%. Anemia was the most frequent finding in complete blood count. The treatments were heterogeneous, including hydrea, EPO, thalidomide/prednisone, danazol and ruxolitinib. Discussion: TE was the most common Ph-MPN. The epidemiological and blood count findings were similar to the data reported in the literature. It is important to note that with the 2016 WHO classification new criteria, some of patients diagnosed with ET, now will be in PV cathegory (21 patients in our serie). The distribution of JAK2V617F+ in Ph-MPN was similar to the published data, except for PV, in which we found a lower percentage of JAK2+. Thrombosis were lower than the data reported for PV. It is worrisome that bone marrow biopsy and cytogenetic study were performed only in a low percentage of the patients. The treatment strategies were heterogeneous and not standardized among the participating centers. These findings reveal a lack in the use of the diagnostic tools for Ph-MPN. It is important to improve clinical and molecular characterization of these patients in order to guide available therapeutic alternatives in our country. Disclosures No relevant conflicts of interest to declare.
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- 2018
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