BEVACIZUMAB AS TREATMENT IN REFRACTORY HEREDITARY HAEMORRHAGIC TELANGIECTASIA
(Abstract release date: 05/19/16)
EHA Library. Castillo E. 06/09/16; 132544; E995
Dr. Erwing Castillo
Contributions
Contributions
Abstract
Abstract: E995
Type: Eposter Presentation
Background
Hereditary Hemorrhagic Telangiectasia (HHT) is a dominantly inherited vascular disorder characterized by recurrent epistaxis, cutaneous telangiectases and visceral arteriovenous malformations (AVMs) that affect many organs: lungs, gastrointestinal tract, liver and brain. Diagnosis is definitive if at least 3 of the following are fulfilled: spontaneous and recurrent epistaxis, telangiectasia, family history and visceral lesions. Although the disease is highly penetrant, during the first years of life, only 50% of individuals have symptoms.Although 80% of patients have gastric or small bowel telangiectases, only 25-30% of patients develops symptomatic gastrointestinal bleeding. Treatment of these patients is based on iron therapy and secondary use of medications such as Hormone Replacement Therapy, antifibrinolytic, tamoxifen, interferon, sirolimus and endoscopic cauterization.There are few clinical studies that clarifies therapeutic measures in these patients, and that is why the best available scientific evidence is based on case reports.
Aims
This is the case of a patient with refractory HHT, who presented severe gastrointestinal bleeding, which was successfully treated with Bevacizumab
Methods
A 67 year-old man with history of Chronic Obstructive Pulmonary Disease, hypertension, diabetes mellitus, hypothyroidism, right-sided heart failure and HHT which was diagnosed 11 years ago. HHT was initially characterized by epistaxis, skin telangiectases and occasional events of gastrointestinal bleeding. The patient had received repeated nasal and esophagogastric cauterizations, but there was scarce response and it was necessary recurring blood transfusions.Diagnosis was confirmed, and multiple palate, esophagus, stomach and duodenum bleeding angiodysplasias were found. Laboratory tests demonstrated iron deficiency and anemia. The patient was on parenteral iron supplementation every week, without obtaining any benefit.
Results
The patient was admitted every week in emergency service with hemorrhagic shock (Hemoblobin (Hb) levels between 3.5 to 6 g/dL), receiving between 3-4 blood units every week.The patient received oral thalidomide 200 mg/day during 2 months with no benefit, persisting low Hb levels despite numerous transfusions, and he was diagnosed with hepatic and pulmonary AVMs. Bevacizumab was initiated 5 mg/kg every 14 days for 6 doses.Hb rose to14 g/dl during the first week of treatment and it has been stable since then, the patient has been independent of blood transfusions for 8 months, decreasing hepatomegaly and right heart failure.
Conclusion
Bevacizumab is a humanized recombinant monoclonal antibody against VEGF, which plays an important role in angiogenesis. It inhibits endothelial cell proliferation thus preventing vessel growth and causing regression of existing vessels.HHT is characterized by high serum and tissue levels of VEGF, that is why Bevacizumab is a reasonable agent to consider for the treatment of HHT. To our knowledge, this is one of the first cases reported of intractable HHT with gastro-intestinal hemorrhage efficiently treated using bevacizumab. Some cases of epistaxis with anemia successfully treated with bevacizumab has been reported. Others works have reported that administration of bevacizumab in patients with HHT associated with severe hepatic AVMs and high cardiac output, was associated with reversal of high-output cardiac failure related to hepatic shunting. Nevertheless, longer follow-up studies are necessary to determine the duration of HHT efficacy and whether maintenance therapy is required.
Session topic: E-poster
Keyword(s): Antibody targeting, Bleeding disorder, VEGF
Type: Eposter Presentation
Background
Hereditary Hemorrhagic Telangiectasia (HHT) is a dominantly inherited vascular disorder characterized by recurrent epistaxis, cutaneous telangiectases and visceral arteriovenous malformations (AVMs) that affect many organs: lungs, gastrointestinal tract, liver and brain. Diagnosis is definitive if at least 3 of the following are fulfilled: spontaneous and recurrent epistaxis, telangiectasia, family history and visceral lesions. Although the disease is highly penetrant, during the first years of life, only 50% of individuals have symptoms.Although 80% of patients have gastric or small bowel telangiectases, only 25-30% of patients develops symptomatic gastrointestinal bleeding. Treatment of these patients is based on iron therapy and secondary use of medications such as Hormone Replacement Therapy, antifibrinolytic, tamoxifen, interferon, sirolimus and endoscopic cauterization.There are few clinical studies that clarifies therapeutic measures in these patients, and that is why the best available scientific evidence is based on case reports.
Aims
This is the case of a patient with refractory HHT, who presented severe gastrointestinal bleeding, which was successfully treated with Bevacizumab
Methods
A 67 year-old man with history of Chronic Obstructive Pulmonary Disease, hypertension, diabetes mellitus, hypothyroidism, right-sided heart failure and HHT which was diagnosed 11 years ago. HHT was initially characterized by epistaxis, skin telangiectases and occasional events of gastrointestinal bleeding. The patient had received repeated nasal and esophagogastric cauterizations, but there was scarce response and it was necessary recurring blood transfusions.Diagnosis was confirmed, and multiple palate, esophagus, stomach and duodenum bleeding angiodysplasias were found. Laboratory tests demonstrated iron deficiency and anemia. The patient was on parenteral iron supplementation every week, without obtaining any benefit.
Results
The patient was admitted every week in emergency service with hemorrhagic shock (Hemoblobin (Hb) levels between 3.5 to 6 g/dL), receiving between 3-4 blood units every week.The patient received oral thalidomide 200 mg/day during 2 months with no benefit, persisting low Hb levels despite numerous transfusions, and he was diagnosed with hepatic and pulmonary AVMs. Bevacizumab was initiated 5 mg/kg every 14 days for 6 doses.Hb rose to14 g/dl during the first week of treatment and it has been stable since then, the patient has been independent of blood transfusions for 8 months, decreasing hepatomegaly and right heart failure.
Conclusion
Bevacizumab is a humanized recombinant monoclonal antibody against VEGF, which plays an important role in angiogenesis. It inhibits endothelial cell proliferation thus preventing vessel growth and causing regression of existing vessels.HHT is characterized by high serum and tissue levels of VEGF, that is why Bevacizumab is a reasonable agent to consider for the treatment of HHT. To our knowledge, this is one of the first cases reported of intractable HHT with gastro-intestinal hemorrhage efficiently treated using bevacizumab. Some cases of epistaxis with anemia successfully treated with bevacizumab has been reported. Others works have reported that administration of bevacizumab in patients with HHT associated with severe hepatic AVMs and high cardiac output, was associated with reversal of high-output cardiac failure related to hepatic shunting. Nevertheless, longer follow-up studies are necessary to determine the duration of HHT efficacy and whether maintenance therapy is required.
Session topic: E-poster
Keyword(s): Antibody targeting, Bleeding disorder, VEGF
Abstract: E995
Type: Eposter Presentation
Background
Hereditary Hemorrhagic Telangiectasia (HHT) is a dominantly inherited vascular disorder characterized by recurrent epistaxis, cutaneous telangiectases and visceral arteriovenous malformations (AVMs) that affect many organs: lungs, gastrointestinal tract, liver and brain. Diagnosis is definitive if at least 3 of the following are fulfilled: spontaneous and recurrent epistaxis, telangiectasia, family history and visceral lesions. Although the disease is highly penetrant, during the first years of life, only 50% of individuals have symptoms.Although 80% of patients have gastric or small bowel telangiectases, only 25-30% of patients develops symptomatic gastrointestinal bleeding. Treatment of these patients is based on iron therapy and secondary use of medications such as Hormone Replacement Therapy, antifibrinolytic, tamoxifen, interferon, sirolimus and endoscopic cauterization.There are few clinical studies that clarifies therapeutic measures in these patients, and that is why the best available scientific evidence is based on case reports.
Aims
This is the case of a patient with refractory HHT, who presented severe gastrointestinal bleeding, which was successfully treated with Bevacizumab
Methods
A 67 year-old man with history of Chronic Obstructive Pulmonary Disease, hypertension, diabetes mellitus, hypothyroidism, right-sided heart failure and HHT which was diagnosed 11 years ago. HHT was initially characterized by epistaxis, skin telangiectases and occasional events of gastrointestinal bleeding. The patient had received repeated nasal and esophagogastric cauterizations, but there was scarce response and it was necessary recurring blood transfusions.Diagnosis was confirmed, and multiple palate, esophagus, stomach and duodenum bleeding angiodysplasias were found. Laboratory tests demonstrated iron deficiency and anemia. The patient was on parenteral iron supplementation every week, without obtaining any benefit.
Results
The patient was admitted every week in emergency service with hemorrhagic shock (Hemoblobin (Hb) levels between 3.5 to 6 g/dL), receiving between 3-4 blood units every week.The patient received oral thalidomide 200 mg/day during 2 months with no benefit, persisting low Hb levels despite numerous transfusions, and he was diagnosed with hepatic and pulmonary AVMs. Bevacizumab was initiated 5 mg/kg every 14 days for 6 doses.Hb rose to14 g/dl during the first week of treatment and it has been stable since then, the patient has been independent of blood transfusions for 8 months, decreasing hepatomegaly and right heart failure.
Conclusion
Bevacizumab is a humanized recombinant monoclonal antibody against VEGF, which plays an important role in angiogenesis. It inhibits endothelial cell proliferation thus preventing vessel growth and causing regression of existing vessels.HHT is characterized by high serum and tissue levels of VEGF, that is why Bevacizumab is a reasonable agent to consider for the treatment of HHT. To our knowledge, this is one of the first cases reported of intractable HHT with gastro-intestinal hemorrhage efficiently treated using bevacizumab. Some cases of epistaxis with anemia successfully treated with bevacizumab has been reported. Others works have reported that administration of bevacizumab in patients with HHT associated with severe hepatic AVMs and high cardiac output, was associated with reversal of high-output cardiac failure related to hepatic shunting. Nevertheless, longer follow-up studies are necessary to determine the duration of HHT efficacy and whether maintenance therapy is required.
Session topic: E-poster
Keyword(s): Antibody targeting, Bleeding disorder, VEGF
Type: Eposter Presentation
Background
Hereditary Hemorrhagic Telangiectasia (HHT) is a dominantly inherited vascular disorder characterized by recurrent epistaxis, cutaneous telangiectases and visceral arteriovenous malformations (AVMs) that affect many organs: lungs, gastrointestinal tract, liver and brain. Diagnosis is definitive if at least 3 of the following are fulfilled: spontaneous and recurrent epistaxis, telangiectasia, family history and visceral lesions. Although the disease is highly penetrant, during the first years of life, only 50% of individuals have symptoms.Although 80% of patients have gastric or small bowel telangiectases, only 25-30% of patients develops symptomatic gastrointestinal bleeding. Treatment of these patients is based on iron therapy and secondary use of medications such as Hormone Replacement Therapy, antifibrinolytic, tamoxifen, interferon, sirolimus and endoscopic cauterization.There are few clinical studies that clarifies therapeutic measures in these patients, and that is why the best available scientific evidence is based on case reports.
Aims
This is the case of a patient with refractory HHT, who presented severe gastrointestinal bleeding, which was successfully treated with Bevacizumab
Methods
A 67 year-old man with history of Chronic Obstructive Pulmonary Disease, hypertension, diabetes mellitus, hypothyroidism, right-sided heart failure and HHT which was diagnosed 11 years ago. HHT was initially characterized by epistaxis, skin telangiectases and occasional events of gastrointestinal bleeding. The patient had received repeated nasal and esophagogastric cauterizations, but there was scarce response and it was necessary recurring blood transfusions.Diagnosis was confirmed, and multiple palate, esophagus, stomach and duodenum bleeding angiodysplasias were found. Laboratory tests demonstrated iron deficiency and anemia. The patient was on parenteral iron supplementation every week, without obtaining any benefit.
Results
The patient was admitted every week in emergency service with hemorrhagic shock (Hemoblobin (Hb) levels between 3.5 to 6 g/dL), receiving between 3-4 blood units every week.The patient received oral thalidomide 200 mg/day during 2 months with no benefit, persisting low Hb levels despite numerous transfusions, and he was diagnosed with hepatic and pulmonary AVMs. Bevacizumab was initiated 5 mg/kg every 14 days for 6 doses.Hb rose to14 g/dl during the first week of treatment and it has been stable since then, the patient has been independent of blood transfusions for 8 months, decreasing hepatomegaly and right heart failure.
Conclusion
Bevacizumab is a humanized recombinant monoclonal antibody against VEGF, which plays an important role in angiogenesis. It inhibits endothelial cell proliferation thus preventing vessel growth and causing regression of existing vessels.HHT is characterized by high serum and tissue levels of VEGF, that is why Bevacizumab is a reasonable agent to consider for the treatment of HHT. To our knowledge, this is one of the first cases reported of intractable HHT with gastro-intestinal hemorrhage efficiently treated using bevacizumab. Some cases of epistaxis with anemia successfully treated with bevacizumab has been reported. Others works have reported that administration of bevacizumab in patients with HHT associated with severe hepatic AVMs and high cardiac output, was associated with reversal of high-output cardiac failure related to hepatic shunting. Nevertheless, longer follow-up studies are necessary to determine the duration of HHT efficacy and whether maintenance therapy is required.
Session topic: E-poster
Keyword(s): Antibody targeting, Bleeding disorder, VEGF
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