ELLIPSE Study: A Phase 1 study evaluating the tolerance of bevacizumab nasal spray in the treatment of epistaxis in hereditary hemorrhagic telangiectasia

mAbs

Volumn 6, Issue 3, 2014, Pages 794-799

  Dupuis Girod, Sophie ^a   Ambrun, Alexis ^b   Decullier, Evelyne ^a,c,d   Samson, Géraldine ^a,c,d   Roux, Adeline ^a,c,d   Fargeton, Anne Emmanuelle ^a   Rioufol, Catherine ^e,f   Schwiertz, Verane ^e   Disant, François ^b   Chapuis, François ^a,c,d   Donazzolo, Yves ^e   Paintaud, Gilles ^g   Edery, Patrick ^a   Faure, Frederic ^b  

^a HOSPICES CIVILS DE LYON   (France)

^b EDOUARD HERRIOT HOSPITAL   (France)

^c UNIVERSITÉ DE LYON   (France)

^d UNIVERSITÉ LYON 1   (France)

^e CENTRE HOSPITALIER LYON SUD   (France)

^f UNIVERSITÉ CLAUDE BERNARD LYON 1   (France)

^g UNIVERSITY HOSPITAL OF TOURS   (France)

Author keywords

Bevacizumab; Epistaxis; Hereditary hemorrhagic telangiectasia; Nasal spray; Phase 1 clinical study; Rare diseases

Indexed keywords

ACTIVIN RECEPTOR LIKE KINASE 1; BEVACIZUMAB; ENDOGLIN; NOSE SPRAY; PLACEBO; ANGIOGENESIS INHIBITOR; MONOCLONAL ANTIBODY;

ADULT; ANEMIA; ARTICLE; ASTHENIA; BLOOD TRANSFUSION; CLINICAL ARTICLE; CONTROLLED STUDY; DISEASE DURATION; DOUBLE BLIND PROCEDURE; DRUG ABSORPTION; DRUG BIOAVAILABILITY; DRUG DOSE ESCALATION; DRUG DOSE INCREASE; DRUG EFFICACY; DRUG ERUPTION; DRUG INDUCED HEADACHE; DRUG SAFETY; DRUG TOLERANCE; EPISTAXIS; ERYTHEMA; FEMALE; GENE MUTATION; HUMAN; HYPERTENSION; LIMIT OF QUANTITATION; MALE; NAUSEA; NOSE MUCOSA; PHASE 1 CLINICAL TRIAL; PROSPECTIVE STUDY; RANDOMIZED CONTROLLED TRIAL; RENDU OSLER WEBER DISEASE; RHINOPHARYNGITIS; VOMITING; AGED; CLINICAL TRIAL; COMPLICATION; DOSE RESPONSE; MIDDLE AGED; TELANGIECTASIA, HEREDITARY HEMORRHAGIC; TREATMENT OUTCOME;

ADULT; AGED; ANGIOGENESIS INHIBITORS; ANTIBODIES, MONOCLONAL, HUMANIZED; BEVACIZUMAB; BLOOD TRANSFUSION; DOSE-RESPONSE RELATIONSHIP, IMMUNOLOGIC; DOUBLE-BLIND METHOD; EPISTAXIS; FEMALE; HUMANS; MALE; MIDDLE AGED; NASAL SPRAYS; TELANGIECTASIA, HEREDITARY HEMORRHAGIC; TREATMENT OUTCOME;

EID: 84899713100     PISSN: 19420862     EISSN: 19420870     Source Type: Journal    
DOI: 10.4161/mabs.28025     Document Type: Article

References (25)

1. Shovlin CL, Guttmacher AE, Buscarini E, Faughnan ME, Hyland RH, Westermann CJ, Kjeldsen AD, Plauchu H. Diagnostic criteria for hereditary hemorrhagic telangiectasia (Rendu-Osler-Weber syndrome). Am J Med Genet 2000; 91:66-7; PMID:10751092; http://dx.doi.org/10.1002/(SICI)1096-8628(20000306)91: 1〈66::AID-AJMG12〉3.0.CO;2-P
2. Haitjema T, Balder W, Disch FJ, Westermann CJ. Epistaxis in hereditary haemorrhagic telangiectasia. Rhinology 1996; 34:176-8; PMID:8938889
3. Karapantzos I, Tsimpiris N, Goulis DG, Van Hoecke H, Van Cauwenberge P, Danielides V. Management of epistaxis in hereditary hemorrhagic telangiectasia by Nd:YAG laser and quality of life assessment using the HR-QoL questionnaire. Eur Arch Otorhinolaryngol 2005; 262:830-3; PMID:15739086; http://dx.doi.org/10. 1007/s00405-004-0911-0
4. Folz BJ, Tennie J, Lippert BM, Werner JA. Natural history and control of epistaxis in a group of German patients with Rendu-Osler-Weber disease. Rhinology 2005; 43:40-6; PMID:15844501
5. McAllister KA, Grogg KM, Johnson DW, Gallione CJ, Baldwin MA, Jackson CE, Helmbold EA, Markel DS, McKinnon WC, Murrell J, et al. Endoglin, a TGF-beta binding protein of endothelial cells, is the gene for hereditary haemorrhagic telangiectasia type 1. Nat Genet 1994; 8:345-51; PMID:7894484; http://dx.doi.org/10.1038/ng1294-345
6. Johnson DW, Berg JN, Baldwin MA, Gallione CJ, Marondel I, Yoon SJ, Stenzel TT, Speer M, Pericak-Vance MA, Diamond A, et al. Mutations in the activin receptor-like kinase 1 gene in hereditary haemorrhagic telangiectasia type 2. Nat Genet 1996; 13:189-95; PMID:8640225; http://dx.doi.org/10.1038/ ng0696-189
7. Gallione CJ, Repetto GM, Legius E, Rustgi AK, Schelley SL, Tejpar S, Mitchell G, Drouin E, Westermann CJ, Marchuk DA. A combined syndrome of juvenile polyposis and hereditary haemorrhagic telangiectasia associated with mutations in MADH4 (SMAD4). Lancet 2004; 363:852-9; PMID:15031030; http://dx.doi.org/10. 1016/S0140-6736(04)15732-2
8. David L, Mallet C, Mazerbourg S, Feige JJ, Bailly S. Identification of BMP9 and BMP10 as functional activators of the orphan activin receptor-like kinase 1 (ALK1) in endothelial cells. Blood 2007; 109:1953-61; PMID:17068149; http://dx.doi.org/10.1182/blood-2006-07-034124
9. Dupuis-Girod S, Ginon I, Saurin JC, Marion D, Guillot E, Decullier E, Roux A, Carette MF, Gilbert-Dussardier B, Hatron PY, et al. Bevacizumab in patients with hereditary hemorrhagic telangiectasia and severe hepatic vascular malformations and high cardiac output. JAMA 2012; 307:948-55; PMID:22396517; http://dx.doi.org/10.1001/jama.2012.250
10. Samson G, García de la Calera A, Dupuis-Girod S, Faure F, Decullier E, Paintaud G, Vignault C, Scoazec JY, Pivot C, Plauchu H, et al. Ex vivo study of bevacizumab transport through porcine nasal mucosa. Eur J Pharm Biopharm 2012; 80:465-9; PMID:22120685; http://dx.doi.org/10.1016/j.ejpb.2011. 11.004
11. Simonds J, Miller F, Mandel J, Davidson TM. The effect of bevacizumab (Avastin) treatment on epistaxis in hereditary hemorrhagic telangiectasia. Laryngoscope 2009; 119:988-92; PMID:19194865; http://dx.doi.org/10.1002/lary. 20159
12. Rohrmeier C, Sachs HG, Kuehnel TS. A retrospective analysis of low dose, intranasal injected bevacizumab (Avastin) in hereditary haemorrhagic telangiectasia. Eur Arch Otorhinolaryngol 2012; 269:531-6; PMID:21805356; http://dx.doi.org/10.1007/s00405-011-1721-9
13. Davidson TM, Olitsky SE, Wei JL. Hereditary hemorrhagic telangiectasia/avastin. Laryngoscope 2010; 120:432-5; PMID:19998344
14. Karnezis TT, Davidson TM. Efficacy of intranasal Bevacizumab (Avastin) treatment in patients with hereditary hemorrhagic telangiectasia-associated epistaxis. Laryngoscope 2011; 121:636-8; PMID:21344445; http://dx.doi.org/10. 1002/lary.21415
15. th, Karnezis T, Davidson TM. Safety of intranasal Bevacizumab (Avastin) treatment in patients with hereditary hemorrhagic telangiectasia-associated epistaxis. Laryngoscope 2011; 121:644-6; PMID:21344447; http://dx.doi.org/10.1002/lary.21345
16. Ivy SP, Siu LL, Garrett-Mayer E, Rubinstein L. Approaches to phase 1 clinical trial design focused on safety, efficiency, and selected patient populations: a report from the clinical trial design task force of the national cancer institute investigational drug steering committee. Clin Cancer Res 2010; 16:1726-36; PMID:20215542; http://dx.doi.org/10.1158/1078-0432.CCR-09-1961
17. Ternant D, Cézé N, Lecomte T, Degenne D, Duveau AC, Watier H, Dorval E, Paintaud G. An enzymelinked immunosorbent assay to study bevacizumab pharmacokinetics. Ther Drug Monit 2010; 32:647-52; PMID:20720519; http://dx.doi.org/10.1097/FTD.0b013e3181ef582a
18. Saif MW, Mehra R. Incidence and management of bevacizumab-related toxicities in colorectal cancer. Expert Opin Drug Saf 2006; 5:553-66; PMID:16774493; http://dx.doi.org/10.1517/14740338.5.4.553
19. Traina TA, Norton L, Drucker K, Singh B. Nasal septum perforation in a bevacizumab-treated patient with metastatic breast cancer. Oncologist 2006; 11:1070-1; PMID:17110625; http://dx.doi.org/10.1634/theoncologist.11-10-1070
20. Fakih MG, Lombardo JC. Bevacizumab-induced nasal septum perforation. Oncologist 2006; 11:85-6; PMID:16401718; http://dx.doi.org/10.1634/ theoncologist.11-1-85
21. Ruiz N, Fernandez-Martos C, Romero I, Pla A, Maiquez J, Calatrava A, Guillem V. Invasive fungal infection and nasal septum perforation with bevacizumab-based therapy in advanced colon cancer. J Clin Oncol 2007; 25:3376-7; PMID:17664487; http://dx.doi.org/10.1200/JCO.2007.12.0006
22. Karnezis TT, Davidson TM. Treatment of hereditary hemorrhagic telangiectasia with submucosal and topical bevacizumab therapy. Laryngoscope 2012; 122:495-7; PMID:22147664; http://dx.doi.org/10.1002/lary.22501
23. Bitonti AJ, Dumont JA. Pulmonary administration of therapeutic proteins using an immunoglobulin transport pathway. Adv Drug Deliv Rev 2006; 58:1106-18; PMID:16997417; http://dx.doi.org/10.1016/j.addr.2006.07.015
24. Kaja S, Hilgenberg JD, Everett E, Olitsky SE, Gossage J, Koulen P. Effects of dilution and prolonged storage with preservative in a polyethylene container on Bevacizumab (Avastin™) for topical delivery as a nasal spray in anti-hereditary hemorrhagic telangiectasia and related therapies. Hum Antibodies 2011; 20:95-101; PMID:22129679
25. Guldmann R, Dupret A, Nivoix Y, Schultz P, Debry C. Bevacizumab nasal spray: Noninvasive treatment of epistaxis in patients with Rendu-Osler disease. Laryngoscope 2012; 122:953-5; PMID:22447341; http://dx.doi.org/10.1002/lary. 23230