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@article{Buscarini:2019:10.1186/s13023-018-0982-4,
author = {Buscarini, E and Botella, LM and Geisthoff, U and Kjeldsen, AD and Mager, HJ and Pagella, F and Suppressa, P and Zarrabeitia, R and Dupuis-Girod, S and Shovlin, CL and VASCERN-HHT},
doi = {10.1186/s13023-018-0982-4},
journal = {Orphanet Journal of Rare Diseases},
title = {Safety of thalidomide and bevacizumab in patients with hereditary hemorrhagic telangiectasia},
url = {http://dx.doi.org/10.1186/s13023-018-0982-4},
volume = {14},
year = {2019}
}

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TY  - JOUR
AB  - BackgroundHereditary hemorrhagic telangiectasia (HHT) is a multisystemic inherited vascular dysplasia that leads to nosebleeds and visceral arteriovenous malformations (AVMs). Anti-angiogenic drugs thalidomide and bevacizumab have been increasingly used off-label with variable results. The HHT working group within the ERN for Rare Multisystemic Vascular Diseases (VASCERN), developed a questionnaire-based retrospective capture of adverse events (AEs) classified using the Common Terminology Criteria for Adverse Events.ResultsSixty-nine HHT patients received bevacizumab, 37 (50.6%) for high output cardiac failure/hepatic AVMs, and 32 (49.4%) for bleeding; the 69 patients received bevacizumab for a mean of 11 months for a total of 63.8 person/years treatment. 67 received thalidomide, all for epistaxis and/or gastrointestinal bleeding; they received thalidomide for a mean of 13.4 months/patient for a total of 75 person/years treatment. AEs were reported in 58 patients, 33 with bevacizumab, 37 with thalidomide. 32 grade 1–3 AEs related to bevacizumab were reported with an average incidence rate of 50 per 100 person-years. 34 grade 1–3 AEs related to thalidomide were reported with an average incidence rate of 45.3 per 100 person-years. Bevacizumab AEs were more common in females (27 AEs in 46 women) than males (6 in 23, p < 0.001). Thalidomide AEs occurred at more similar rates in males (25 AEs in 41 men, 60.9%) and females (12 in 26 (46.2%), but were more common in ENG patients (17 in 17) than in ACVRL1 (14 in 34, p < 0.0001). For bevacizumab, the most common reports were of joint pains (7/69, 10%), headache (3/69, 4.4%) and proteinuria (2/69, 3%), and for thalidomide, peripheral neuropathy (12/67, 18%); drowsiness (8/67, 12%); and dizziness (6/67, 9%). Fatal adverse events were more common in males (p = 0.009), and in patients with ENG pathogenic variants (p = 0.012). One fatal AE was
AU  - Buscarini,E
AU  - Botella,LM
AU  - Geisthoff,U
AU  - Kjeldsen,AD
AU  - Mager,HJ
AU  - Pagella,F
AU  - Suppressa,P
AU  - Zarrabeitia,R
AU  - Dupuis-Girod,S
AU  - Shovlin,CL
AU  - VASCERN-HHT
DO  - 10.1186/s13023-018-0982-4
PY  - 2019///
SN  - 1750-1172
TI  - Safety of thalidomide and bevacizumab in patients with hereditary hemorrhagic telangiectasia
T2  - Orphanet Journal of Rare Diseases
UR  - http://dx.doi.org/10.1186/s13023-018-0982-4
UR  - http://hdl.handle.net/10044/1/67482
VL  - 14
ER  -

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Claire L. Shovlin PhD FRCP

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