Executive summary of the 12th HHT international scientific conference

Andrejecsk, Jillian W.; Hosman, Anna E; Botella, Luisa María; Shovlin, CL; Arthur, Helen M.; Dupuis‐Girod, Sophie; Buscarini, Elisabetta; Hughes, Christopher C.W.; Lebrin, Franck; Mummery, Christine L.; Post, Marco C.; Mager, Johannes J.

doi:10.1007/s10456-017-9585-2

Cited by 11 publications

(11 citation statements)

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“…This hemodynamic improvement has already been outlined by Dupuis-Girod et al in a prospective cohort study of 24 patients in which 3 patients had complete response and 17 patients achieved partial response at three months after starting treatment [24]. Bevacizumab has been proposed as a bridge therapy for patients awaiting LT [31,32] or as a supportive treatment when transplantation is not an alternative or it is rejected by the patient [33]. In the present study, bevacizumab treatment allowed a better clinical performance in two patients awaiting for LT, one of whom was under anticoagulation treatment.…”

Section: Discussionmentioning

confidence: 56%

Bevacizumab for treating Hereditary Hemorrhagic Telangiectasia patients with severe hepatic involvement or refractory anemia

et al. 2020

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ObjectiveTo report our clinical experience with bevacizumab in a cohort of Hereditary Hemorrhagic Telangiectasia (HHT) patients with severe hepatic involvement and/or refractory anemia. MethodsObservational, ambispective study of the Institutional Registry of HHT at Hospital Italiano de Buenos Aires. Patients were treated with bevacizumab due to iron deficiency refractory anemia secondary to nasal/gastrointestinal bleeding and/or high output cardiac failure. We describe basal clinical data, bevacizumab schedules, efficacy outcomes and adverse events. Wilcoxon signed ranks test and longitudinal analysis were conducted. ResultsTwenty adult patients were included from July 2013 to June 2019. Clinical indications were: 13 for anemia, 4 for heart failure and 3 for both. In the anemia group, median pretreatment hemoglobin was 8.1 g/dl [IQR: 7.2-8.4] and median transfusion requirement was 4 units [2][3][4][5][6]. In heart failure group, pretreatment median cardiac index was 4.5 L/min/m 2 [4.1-5.6] and cardiac output was 8.3 L/min [7.5-9.2]. Bevacizumab 5 mg/kg/dose every 2 weeks for 6 applications was scheduled. By the end of induction, median hemoglobin at 3 months was 10.9 g/dl [9.5-12.8] (p = 0.01) and median transfusion requirement 0 units [0-1] (p<0.01), and this effect was more or less sustained during a year. Regarding heart failure group, two patients had complete hemodynamic response and achieved liver transplantation and two had partial response. No serious adverse events were registered.

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Section: Discussionmentioning

confidence: 56%

Bevacizumab for treating Hereditary Hemorrhagic Telangiectasia patients with severe hepatic involvement or refractory anemia

et al. 2020

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“…Epistaxis severity may change according to several individual and environmental factors, for example, age, season, blood pressure, etc. (6) . It could be mild and infrequent, never requiring medical attention, or severe and life-threatening, leading to anaemia, transfusion dependence and several surgical treatments.…”

Section: Frequencymentioning

confidence: 99%

“…The clinical spectrum related to epistaxis ranges from mild and rare nosebleeds to life-threatening episodes due to massive blood loss and need for hospitalization, transfusions and, in some selected case, surgical treatment. Epistaxis is also the main complaint of patients and represents the most common cause of disability and social impairment (5,6) . taxis, therefore it is of paramount importance for the clinician to effectively grade this symptom.…”

Section: Introductionmentioning

confidence: 99%

FID Score: an effective tool in Hereditary Haemorrhagic Telangiectasia - related epistaxis

Pagella¹,

Maiorano²,

Matti³

et al. 2020

Rhin

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Background: Hereditary haemorrhagic telangiectasia (HHT) is a rare disease characterized by a multisystemic vascular dysplasia and epistaxis, that is the most common cause of disability and social impairment. Patient management strictly depends on the severity of this symptom; therefore, it is of paramount importance for the clinicians to effectively grade epistaxis severity. The aim of this report was to validate the Frequency, Intensity and Duration score (FID) for grading epistaxis severity in patients with HHT; we studied repeatability and external validity comparing FID score with Epistaxis Severity Score (ESS). Methods: This is a descriptive, observational study that included 264 adult HHT patients with epistaxis. Diagnosis of HHT was established with Curaçao criteria or positivity at genetic testing. Nosebleed severity was evaluated according to the FID score and the ESS. The first 30 patients were included in the validation of the FID score, which was graded on days 0, 1, 3 and 7. In the remaining 234 patients, a comparison between the ESS and FID score was performed. Results: The statistical analysis performed in order to validate the FID score showed very good agreement between scores calculated on different days; analysis comparing the FID score with the ESS revealed a high correlation between the two grading systems. Conclusions: The FID score is a quick, easy and precise tool for evaluating HHT-related epistaxis and could be a possible alternative to the ESS. The FID score meets the need for an intuitive and smart grading system that is easy to manage in clinicians' hands.

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“…Patients with HHT type 1, especially women who have not been screened and treated preemptively, have a slightly lower life expectancy than family members without HHT and severe epistaxis can result in a decreased quality of life [ 18 ]. However, preliminary data show that a normal life expectancy can be achieved when patients are screened and treated appropriately [ 19 ].…”

Section: Hereditary Haemorrhagic Telangiectasiamentioning

confidence: 99%

Pulmonary Arterial Hypertension and Hereditary Haemorrhagic Telangiectasia

Vorselaars

Hosman

Westermann

et al. 2018

IJMS

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Hereditary haemorrhagic telangiectasia (HHT) is an autosomal dominant inherited disease characterised by multisystemic vascular dysplasia. Heritable pulmonary arterial hypertension (HPAH) is a rare but severe complication of HHT. Both diseases can be the result of genetic mutations in ACVLR1 and ENG encoding for proteins involved in the transforming growth factor-beta (TGF-β) superfamily, a signalling pathway that is essential for angiogenesis. Changes within this pathway can lead to both the proliferative vasculopathy of HPAH and arteriovenous malformations seen in HHT. Clinical signs of the disease combination may not be specific but early diagnosis is important for appropriate treatment. This review describes the molecular mechanism and management of HPAH and HHT.

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Executive summary of the 12th HHT international scientific conference

Cited by 11 publications

References 50 publications

Bevacizumab for treating Hereditary Hemorrhagic Telangiectasia patients with severe hepatic involvement or refractory anemia

Bevacizumab for treating Hereditary Hemorrhagic Telangiectasia patients with severe hepatic involvement or refractory anemia

FID Score: an effective tool in Hereditary Haemorrhagic Telangiectasia - related epistaxis

Pulmonary Arterial Hypertension and Hereditary Haemorrhagic Telangiectasia

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