Gentium SpA (Nasdaq: GENT) announced that the abstract entitled "defibrotide prevents liver VOD and significantly reduces the complications associated with VOD-at-risk children: final results of a prospective phase II / III multicentre, "was presented on Sunday, March 21, 2010, at the opening session of the Blood and Marrow Transplantation European (EBMT) 36th Annual Meeting in Vienna . The abstract was awarded the prestigious Van Bekkum Award for best abstract submitted to the medical program. Dr. Selim Corbacioglu, Professor of Pediatrics at the University of Regensburg (Germany) and principal investigator of pediatric prevention trial, accepted the award and presented the final results.
This Phase II / III, randomized, controlled trial evaluated the prophylactic use of defibrotide in pediatric transplant stem cell (SCT) patients at high risk of veno-occulsive (VOD .) In the analysis by intention to treat (ITT), which included 356 patients (180 patients in the prophylaxis group and 176 in the grupo de control), defibrotide demostrado una reducción del 40% en la incidencia de vídeo a la carta dentro de los 30 días después de la SCT, la variable principal de el estudio. la incidencia fue del 20% de Video por Demanda en el grupo control y 12% en el grupo de profilaxis (p = 0,0488 riesgo competitivo; p = 0,0507 de Kaplan-Meier). En el análisis por protocolo, la incidencia fue del 20% de Video por Demanda en el grupo control y 11% en el grupo de profilaxis (p = 0,0225 riesgo competitivo; p = 0,0234 de Kaplan-Meier). Un valor de p igual o inferior a 0,05 se necesita para alcanzar significación estadística.
addition, a pre-specified analysis showed that the incidence and severity of graft versus host disease (GVHD) for 100 days in recipients of allogeneic SCT was significantly reduced from 63% the control group to 45% for the prophylaxis group (p = 0.0046 for the incidence of GVHD and p = 0.0034 for severity). Renal failure was reduced from 6% in the control group to 1% in the defibrotide group (p = 0.0169). With respect to security, defibrotide was well tolerated and no difference in adverse events was observed between the two study arms.
"The resultados del estudio demostraron que defibrotide redujo la incidencia de VOD en alto riesgo de los pacientes pediátricos", declaró el doctor Selim Corbacioglu. "SCT pacientes que desarrollan VOD, independientemente de su gravedad ya pesar de un tratamiento rápido, tienen una tasa de mortalidad cuatro veces mayor que los pacientes sin VOD. También estamos muy entusiasmados de que defibrotide tiene el potencial de reducir la incidencia y severidad de la EICH aguda, una vida principales complicaciones, que es frecuente en SCT y tiene opciones de tratamiento limitadas. Creemos que defibrotide podría tener un papel futuro de la prevención de la GVHD. "
" defibrotide VOD prevention study is the largest study in the context of pediatric SCT and the results have been included in the EBMT press conference, "said Professor Dietger Niederwiesa University of Leipzig, president of the opening session, the chairman of the EBMT, and newly elected president of the Global Network for Blood and Marrow Transplantation (WBMT). "The results of this study, showing a reduction in VOD, and the possible beneficial effects on GVHD and renal failure are consistent with the effects of endothelial protection and confirm the potential of defibrotide in the prevention of transplant-related toxicities. "
"We are very encouraged by the recognition of this study by the leaders of the European Hematology community," said Dr. Massimo Iacobelli, Scientific Director, Gentium. "We are fully committed to the preparation of regulatory filings for the treatment prevencióny video on demand."
defibrotide biological activity and mechanism de acción se destacan en un cartel distinto-presentación, "defibrotide protege las células endoteliales microvasculares de las señales pro-inflamatorias y pro-apoptótica mediada por fludarabina: un microarray (Affimetrix) análisis". Este estudio proporciona nuevos datos biológicos sobre la forma defibrotide podría proteger contra las células endoteliales la activación inducida por la quimioterapia y la muerte celular, sin afectar el efecto de fludarabina antileucémica y sin afectar a endotelio normal.
El simposio satélite "defibrotide for the Treatment of Liver Prevencióny VOD following stem cell transplantation "was well attended, with presentations by opinion leaders Dr. Ernst Holler (University of Regensburg), the Dr. Selim Corbacioglu (University of Regensburg), Dr. Paul Richardson (Dana Farber Cancer Institute, Boston, USA) and Dr. Enric Carreras (University of Barcelona). The symposium was chaired by Dr. Rob Soiffer (Dana Farber Cancer Institute in Boston, USA).
About EBMT and Van Bekkum Award
The European Group of Blood and Marrow (EBMT) es una organización europea sin ánimo de lucro que fue establecida en 1974 para permitir a los científicos y médicos implicados en el trasplante clínico de médula ósea para compartir su experiencia y desarrollar los estudios cooperativos. En representación de 527 centros de trasplante a través de 57 países dentro y fuera de Europa, la EBMT promueve todas las actividades encaminadas a mejorar trasplante de células madre o la terapia celular.
Anuncios Google BeadChip HumanOmni1-Quad
Obtener la mejor potencia para estudios de asociación. Www.illumina.com/dna/omni/
Van Bekkum
The award is named for Dr. Dirk Van Bekkum, who is recognized internationally for his work in transplanting bone marrow, among other achievements, his laboratory was the first to identify what is now known as graft versus host.
About the Phase II / III European trial of pediatric prevention
EBMT Phase II / III European trial of pediatric prevention was a prospective, multicenter, randomized, open to evaluate the prophylactic use of defibrotide in patients under 18 years of age who underwent stem cell transplantation (SCT) and were at high risk of Veno-Occlusive Disease (VOD). Patients randomized to the prophylaxis group received 25 mg / kg / day in four divided doses defibrotide at the time of initiation of conditioning until 30 days after transplantation. Patients assigned to the control arm did not receive prophylactic therapy VOD. Patients received defibrotide as therapy if they developed VOD. The primary study endpoint was the development of VOD within 30 days after of the SCT, based on the modified criteria of Seattle. A blinded independent review committee of three experts hematologists confirmed the diagnosis of VOD.
About
VOD
veno-occlusive disease is a potentially life-threatening, which normally occurs as a major complication of stem cell transplantation. Some high-dose conditioning regimens used as part of SCT can damage the cells lining the hepatic blood vessels and thus result in VOD, a blockage of small veins of the liver that leads to liver failure and can result in significant dysfunction in other organs such as kidneys and lungs ( Call severe VOD). SCT is a treatment modality often used following high-dose chemotherapy and radiation therapy for blood cancers and other conditions in both adults and children. Currently, no approved agent for the treatment or prevention of VOD in the U.S. or the EU.
GVHD About
Graft versus host disease (GVHD) is a complication that frequently occurs after allogeneic stem cell where the blood-forming cells to an entirely new immune system is transplanted from a donor. The differences between donor and recipient T cells frequently (a subtype of white blood cells) from the donor recognize the recipient's body tissues as foreign. When this happens, the newly transplanted immune system attacks the transplant recipient's body. Increased risk GVHD with the degree of mismatch between donor and recipient.
About Gentium Gentium SpA
, located in Como, Italy, is a biopharmaceutical company focused on the development and manufacture of medicines to treat and prevent various diseases and conditions, including diseases vascular related to cancer and its treatments. Defibrotide, the candidate of the company's lead product, is an investigational drug that has been granted orphan drug status by the U.S. FDA and Orphan Drug Designation por la Comisión Europea, tanto para tratar y prevenir el VOD y Fast Track Designación por la FDA de EE.UU. para el tratamiento de VOD .
Advertencia sobre las declaraciones prospectivas
Este comunicado de prensa contiene "declaraciones prospectivas". En algunos casos, se puede identificar estas declaraciones prospectivas palabras como "puede", "podría", "hará", "debería", "espera", "planea", "anticipa", "Believe," "estimate," "predicts," "potential" or "continue" the negative of these terms and other comparable terminology. These statements are not historical facts but instead represent the Company's belief regarding future results, many of which, by their nature, are inherently uncertain and outside the Company's control. It is possible that actual results, including with respect to the possibility of future regulatory approval, may differ materially from those anticipated in these forward-looking statements. For a discussion of some of the risks and important factors that could affect future results, see the discussion in our Form 20-F filed with the Securities and Exchange Commission under the title "Risk Factors."
This Phase II / III, randomized, controlled trial evaluated the prophylactic use of defibrotide in pediatric transplant stem cell (SCT) patients at high risk of veno-occulsive (VOD .) In the analysis by intention to treat (ITT), which included 356 patients (180 patients in the prophylaxis group and 176 in the grupo de control), defibrotide demostrado una reducción del 40% en la incidencia de vídeo a la carta dentro de los 30 días después de la SCT, la variable principal de el estudio. la incidencia fue del 20% de Video por Demanda en el grupo control y 12% en el grupo de profilaxis (p = 0,0488 riesgo competitivo; p = 0,0507 de Kaplan-Meier). En el análisis por protocolo, la incidencia fue del 20% de Video por Demanda en el grupo control y 11% en el grupo de profilaxis (p = 0,0225 riesgo competitivo; p = 0,0234 de Kaplan-Meier). Un valor de p igual o inferior a 0,05 se necesita para alcanzar significación estadística.
addition, a pre-specified analysis showed that the incidence and severity of graft versus host disease (GVHD) for 100 days in recipients of allogeneic SCT was significantly reduced from 63% the control group to 45% for the prophylaxis group (p = 0.0046 for the incidence of GVHD and p = 0.0034 for severity). Renal failure was reduced from 6% in the control group to 1% in the defibrotide group (p = 0.0169). With respect to security, defibrotide was well tolerated and no difference in adverse events was observed between the two study arms.
"The resultados del estudio demostraron que defibrotide redujo la incidencia de VOD en alto riesgo de los pacientes pediátricos", declaró el doctor Selim Corbacioglu. "SCT pacientes que desarrollan VOD, independientemente de su gravedad ya pesar de un tratamiento rápido, tienen una tasa de mortalidad cuatro veces mayor que los pacientes sin VOD. También estamos muy entusiasmados de que defibrotide tiene el potencial de reducir la incidencia y severidad de la EICH aguda, una vida principales complicaciones, que es frecuente en SCT y tiene opciones de tratamiento limitadas. Creemos que defibrotide podría tener un papel futuro de la prevención de la GVHD. "
" defibrotide VOD prevention study is the largest study in the context of pediatric SCT and the results have been included in the EBMT press conference, "said Professor Dietger Niederwiesa University of Leipzig, president of the opening session, the chairman of the EBMT, and newly elected president of the Global Network for Blood and Marrow Transplantation (WBMT). "The results of this study, showing a reduction in VOD, and the possible beneficial effects on GVHD and renal failure are consistent with the effects of endothelial protection and confirm the potential of defibrotide in the prevention of transplant-related toxicities. "
"We are very encouraged by the recognition of this study by the leaders of the European Hematology community," said Dr. Massimo Iacobelli, Scientific Director, Gentium. "We are fully committed to the preparation of regulatory filings for the treatment prevencióny video on demand."
defibrotide biological activity and mechanism de acción se destacan en un cartel distinto-presentación, "defibrotide protege las células endoteliales microvasculares de las señales pro-inflamatorias y pro-apoptótica mediada por fludarabina: un microarray (Affimetrix) análisis". Este estudio proporciona nuevos datos biológicos sobre la forma defibrotide podría proteger contra las células endoteliales la activación inducida por la quimioterapia y la muerte celular, sin afectar el efecto de fludarabina antileucémica y sin afectar a endotelio normal.
El simposio satélite "defibrotide for the Treatment of Liver Prevencióny VOD following stem cell transplantation "was well attended, with presentations by opinion leaders Dr. Ernst Holler (University of Regensburg), the Dr. Selim Corbacioglu (University of Regensburg), Dr. Paul Richardson (Dana Farber Cancer Institute, Boston, USA) and Dr. Enric Carreras (University of Barcelona). The symposium was chaired by Dr. Rob Soiffer (Dana Farber Cancer Institute in Boston, USA).
About EBMT and Van Bekkum Award
The European Group of Blood and Marrow (EBMT) es una organización europea sin ánimo de lucro que fue establecida en 1974 para permitir a los científicos y médicos implicados en el trasplante clínico de médula ósea para compartir su experiencia y desarrollar los estudios cooperativos. En representación de 527 centros de trasplante a través de 57 países dentro y fuera de Europa, la EBMT promueve todas las actividades encaminadas a mejorar trasplante de células madre o la terapia celular.
Anuncios Google BeadChip HumanOmni1-Quad
Obtener la mejor potencia para estudios de asociación. Www.illumina.com/dna/omni/
Van Bekkum
The award is named for Dr. Dirk Van Bekkum, who is recognized internationally for his work in transplanting bone marrow, among other achievements, his laboratory was the first to identify what is now known as graft versus host.
About the Phase II / III European trial of pediatric prevention
EBMT Phase II / III European trial of pediatric prevention was a prospective, multicenter, randomized, open to evaluate the prophylactic use of defibrotide in patients under 18 years of age who underwent stem cell transplantation (SCT) and were at high risk of Veno-Occlusive Disease (VOD). Patients randomized to the prophylaxis group received 25 mg / kg / day in four divided doses defibrotide at the time of initiation of conditioning until 30 days after transplantation. Patients assigned to the control arm did not receive prophylactic therapy VOD. Patients received defibrotide as therapy if they developed VOD. The primary study endpoint was the development of VOD within 30 days after of the SCT, based on the modified criteria of Seattle. A blinded independent review committee of three experts hematologists confirmed the diagnosis of VOD.
About
VOD
veno-occlusive disease is a potentially life-threatening, which normally occurs as a major complication of stem cell transplantation. Some high-dose conditioning regimens used as part of SCT can damage the cells lining the hepatic blood vessels and thus result in VOD, a blockage of small veins of the liver that leads to liver failure and can result in significant dysfunction in other organs such as kidneys and lungs ( Call severe VOD). SCT is a treatment modality often used following high-dose chemotherapy and radiation therapy for blood cancers and other conditions in both adults and children. Currently, no approved agent for the treatment or prevention of VOD in the U.S. or the EU.
GVHD About
Graft versus host disease (GVHD) is a complication that frequently occurs after allogeneic stem cell where the blood-forming cells to an entirely new immune system is transplanted from a donor. The differences between donor and recipient T cells frequently (a subtype of white blood cells) from the donor recognize the recipient's body tissues as foreign. When this happens, the newly transplanted immune system attacks the transplant recipient's body. Increased risk GVHD with the degree of mismatch between donor and recipient.
About Gentium Gentium SpA
, located in Como, Italy, is a biopharmaceutical company focused on the development and manufacture of medicines to treat and prevent various diseases and conditions, including diseases vascular related to cancer and its treatments. Defibrotide, the candidate of the company's lead product, is an investigational drug that has been granted orphan drug status by the U.S. FDA and Orphan Drug Designation por la Comisión Europea, tanto para tratar y prevenir el VOD y Fast Track Designación por la FDA de EE.UU. para el tratamiento de VOD .
Advertencia sobre las declaraciones prospectivas
Este comunicado de prensa contiene "declaraciones prospectivas". En algunos casos, se puede identificar estas declaraciones prospectivas palabras como "puede", "podría", "hará", "debería", "espera", "planea", "anticipa", "Believe," "estimate," "predicts," "potential" or "continue" the negative of these terms and other comparable terminology. These statements are not historical facts but instead represent the Company's belief regarding future results, many of which, by their nature, are inherently uncertain and outside the Company's control. It is possible that actual results, including with respect to the possibility of future regulatory approval, may differ materially from those anticipated in these forward-looking statements. For a discussion of some of the risks and important factors that could affect future results, see the discussion in our Form 20-F filed with the Securities and Exchange Commission under the title "Risk Factors."
0 comments:
Post a Comment