Teprotumumab for thyroid-associated ophthalmopathy

New England Journal of Medicine

Volumn 376, Issue 18, 2017, Pages 1748-1761

  Smith, Terry J ^a   Kahaly, George J ^b   Ezra, Daniel G ^c   Fleming, James C ^d   Dailey, Roger A ^e   Tang, Rosa A ^f   Harris, Gerald J ^g   Antonelli, Alessandro ^h   Salvi, Mario ⁱ   Goldberg, Robert A ^j   Gigantelli, James W ^k   Couch, Steven M ^l   Shriver, Erin M ^m   Hayek, Brent R ⁿ   Hink, Eric M ^o   Woodward, Richard M ^p   Gabriel, Kathleen ^p   Magni, Guido ^p   Douglas, Raymond S ^a  

^a UNIVERSITY OF MICHIGAN MEDICAL SCHOOL   (United States)

^b UNIVERSITY MEDICAL CENTER MAINZ   (Germany)

^c MOORFIELDS EYE HOSPITAL   (United Kingdom)

^d UNIVERSITY OF TENNESSEE HEALTH SCIENCE CENTER   (United States)

^e OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

^f Ey Wellness Center   (United States)

^g MEDICAL COLLEGE OF WISCONSIN   (United States)

^h UNIVERSITY OF PISA   (Italy)

ⁱ UNIVERSITY OF MILAN   (Italy)

^j JULES STEIN EYE INSTITUTE   (United States)

^k UNIVERSITY OF NEBRASKA MEDICAL CENTER   (United States)

^l BARNES JEWISH HOSPITAL   (United States)

^m UNIVERSITY OF IOWA HOSPITALS AND CLINICS   (United States)

ⁿ EMORY UNIVERSITY SCHOOL OF MEDICINE   (United States)

^o UNIVERSITY OF COLORADO SCHOOL OF MEDICINE   (United States)

^p River Vision Development   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

DRUG ANTIBODY; NEUTRALIZING ANTIBODY; PLACEBO; TEPROTUMUMAB; IMMUNOLOGIC FACTOR; MONOCLONAL ANTIBODY; R-1507 MONOCLONAL ANTIBODY; SOMATOMEDIN C;

ADULT; AGED; ALOPECIA; ARTICLE; CONTROLLED STUDY; DEATH; DIABETES MELLITUS; DIARRHEA; DISEASE SEVERITY; DOUBLE BLIND PROCEDURE; DRUG EFFICACY; DRUG RESPONSE; DRUG SAFETY; DRY SKIN; DYSGEUSIA; ENDOCRINE OPHTHALMOPATHY; EXOPHTHALMOS; FEMALE; HASHIMOTO ENCEPHALOPATHY; HEADACHE; HEARING IMPAIRMENT; HUMAN; HYPERGLYCEMIA; INFLAMMATORY BOWEL DISEASE; MAJOR CLINICAL STUDY; MALE; MENTAL DISEASE; MULTICENTER STUDY; MUSCLE SPASM; NAUSEA; OPTIC NERVE DISEASE; PARESTHESIA; PRIORITY JOURNAL; QUALITY OF LIFE; QUESTIONNAIRE; RANDOMIZED CONTROLLED TRIAL; SCORING SYSTEM; SEPSIS; SIDE EFFECT; TREATMENT DURATION; URINE RETENTION; WEIGHT REDUCTION; ANTAGONISTS AND INHIBITORS; CHEMICALLY INDUCED; CLINICAL TRIAL; COMPLICATION; DIABETIC COMPLICATION; GRAVES OPHTHALMOPATHY; INTENTION TO TREAT ANALYSIS; MIDDLE AGED; STATISTICAL MODEL;

ADULT; AGED; ANTIBODIES, MONOCLONAL; DIABETES COMPLICATIONS; DOUBLE-BLIND METHOD; EXOPHTHALMOS; FEMALE; GRAVES OPHTHALMOPATHY; HUMANS; HYPERGLYCEMIA; IMMUNOLOGIC FACTORS; INSULIN-LIKE GROWTH FACTOR I; INTENTION TO TREAT ANALYSIS; LOGISTIC MODELS; MALE; MIDDLE AGED; QUALITY OF LIFE;

EID: 85019065038     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMoa1614949     Document Type: Article

References (40)

1. Smith TJ Heged L. Graves' disease. N Engl J Med 2016; 375:1552-65.
2. Sandler HM, Rubenstein JH, Fowble BL, Sergott RC, Savino PJ, Bosley TM. Results of radiotherapy for thyroid ophthalmopathy. Int J Radiat Oncol Biol Phys 1989; 17: 823-7.
3. Bartalena L, Krassas GE, Wiersinga W, et al. Efficacy and safety of three different cumulative doses of intravenous methylprednisolone for moderate to severe and active Graves' orbitopathy. J Clin Endocrinol Metab 2012; 97: 4454-63.
4. Zang S, Ponto KA, Kahaly GJ. Clinical review: Intravenous glucocorticoids for Graves' orbitopathy: Efficacy and morbidity. J Clin Endocrinol Metab 2011; 96: 320-32.
5. Sisti E, Coco B, Menconi F, et al. Intravenous glucocorticoid therapy for Graves' ophthalmopathy and acute liver damage: An epidemiological study. Eur J Endocrinol 2015; 172: 269-76.
6. Tanda ML, Bartalena L. Efficacy and safety of orbital radiotherapy for Graves' orbitopathy. J Clin Endocrinol Metab 2012; 97: 3857-65.
7. Marcocci C, Bartalena L, Tanda ML, et al. Comparison of the effectiveness and tolerability of intravenous or oral glucocorticoids associated with orbital radiotherapy in the management of severe Graves' ophthalmopathy: Results of a prospective, single-blind, randomized study. J Clin Endocrinol Metab 2001; 86: 3562-7.
8. Morshed SA, Davies TF. Graves' disease mechanisms: The role of stimulating, blocking, and cleavage region TSH receptor antibodies. Horm Metab Res 2015; 47: 727-34.
9. Diana T Wter C Kanitz M Kahaly GJ. Highly variable sensitivity of five binding and two bio-assays for TSHreceptor antibodies. J Endocrinol Invest 2016;39: 1159-65.
10. Feliciello A Porcellini A Ciullo I Bonavolont G Avvedimento EV Fenzi G. Expression of thyrotropin-receptor mRNA in healthy and Graves' disease retro-orbital tissue. Lancet1993; 342: 337-8.
11. Bahn RS, Dutton CM, Natt N, Joba W, Spitzweg C, Heufelder AE. Thyrotropin receptor expression in Graves' orbital adipose/connective tissues: Potential autoantigen in Graves' ophthalmopathy. J Clin Endocrinol Metab 1998; 83: 998-1002.
12. Douglas RS, Afifiyan NF, Hwang CJ, et al. Increased generation of fibrocytes in thyroid-associated ophthalmopathy. J Clin Endocrinol Metab 2010; 95: 430-8.
13. Tabasum A, Khan I, Taylor P, Das G, Okosieme OE. Thyroid antibody-negative euthyroid Graves' ophthalmopathy. Endocrinol Diabetes Metab Case Rep 2016; 2016: 160008.
14. Pritchard J, Han R, Horst N, Cruikshank WW, Smith TJ. Immunoglobulin activation of T cell chemoattractant expression in fibroblasts from patients with Graves' disease is mediated through the insulin-like growth factor I receptor pathway. J Immunol 2003; 170: 6348-54.
15. Tramontano D, Cushing GW, Moses AC, Ingbar SH. Insulin-like growth factor- I stimulates the growth of rat thyroid cells in culture and synergizes the stimulation of DNA synthesis induced by TSH and Graves'-IgG. Endocrinology 1986; 119: 940-2.
16. Gallagher EJ, LeRoith D. Minireview: IGF, insulin, and cancer. Endocrinology 2011; 152: 2546-51.
17. Smith TJ. Insulin-like growth factor-I regulation of immune function: A potential therapeutic target in autoimmune diseases? Pharmacol Rev 2010; 62: 199-236.
18. Tsui S, Naik V, Hoa N, et al. Evidence for an association between thyroid-stimulating hormone and insulin-like growth factor 1 receptors: A tale of two antigens implicated in Graves' disease. J Immunol 2008; 181: 4397-405.
19. Douglas RS, Gianoukakis AG, Kamat S, Smith TJ. Aberrant expression of the insulin-like growth factor-1 receptor by T cells from patients with Graves' disease may carry functional consequences for disease pathogenesis. J Immunol 2007; 178: 3281-7.
20. Douglas RS, Naik V, Hwang CJ, et al. B cells from patients with Graves' disease aberrantly express the IGF-1 receptor: Implications for disease pathogenesis. J Immunol 2008; 181: 5768-74.
21. Chen H, Mester T, Raychaudhuri N, et al. Teprotumumab, an IGF-1R blocking monoclonal antibody inhibits TSH and IGF-1 action in fibrocytes. J Clin Endocrinol Metab 2014; 99: E1635-E1640.
22. Kurzrock R, Patnaik A, Aisner J, et al. A phase I study of weekly R1507, a human monoclonal antibody insulin-like growth factor-I receptor antagonist, in patients with advanced solid tumors. Clin Cancer Res 2010; 16: 2458-65.
23. Wiersinga WM, Perros P, Kahaly GJ, et al. Clinical assessment of patients with Graves' orbitopathy: The European Group on Graves' Orbitopathy recommendations to generalists, specialists and clinical researchers. Eur J Endocrinol 2006; 155: 387-9.
24. Terwee CB, Gerding MN, Dekker FW, Prummel MF, Wiersinga WM. Development of a disease specific quality of life questionnaire for patients with Graves' ophthalmopathy: The GO-QOL. Br J Ophthalmol 1998; 82: 773-9.
25. Rootman DB, Golan S, Pavlovich P, Rootman J. Postoperative changes in strabismus, ductions, exophthalmometry, and eyelid retraction after orbital decompression for thyroid orbitopathy. Ophthal Plast Reconstr Surg 2016 August 1 (Epub ahead of print).
26. Wu CY, Niziol LM, Musch DC, Kahana A. Thyroid-related orbital decompression surgery: A multivariate analysis of risk factors and outcomes. Ophthal Plast Reconstr Surg 2016 April 19 (Epub ahead of print).
27. Terwee CB, Dekker FW, Mourits MP, et al. Interpretation and validity of changes in scores on the Graves' ophthalmopathy quality of life questionnaire (GO-QOL) after different treatments. Clin Endocrinol (Oxf) 2001; 54: 391-8.
28. Baldeschi L, Lupetti A, Vu P, Wakelkamp IM, Prummel MF, Wiersinga WM. Reactivation of Graves' orbitopathy after rehabilitative orbital decompression. Ophthalmology 2007; 114: 1395-402.
29. Weightman DR, Perros P, Sherif IH, Kendall-Taylor P. Autoantibodies to IGF-1 binding sites in thyroid associated ophthalmopathy. Autoimmunity 1993; 16: 251-7.
30. Smith TJ, Hoa N. Immunoglobulins from patients with Graves' disease induce hyaluronan synthesis in their orbital fibroblasts through the self-antigen, IGF-1 receptor. J Clin Endocrinol Metab 2004; 89: 5076-80.
31. Varewijck AJ, Boelen A, Lamberts SW, et al. Circulating IgGs may modulate IGF-I receptor stimulating activity in a subset of patients with Graves' ophthalmopathy. J Clin Endocrinol Metab 2013; 98: 769-76.
32. Smith TJ. Is IGF-I receptor a target for autoantibody generation in Graves' disease? J Clin Endocrinol Metab 2013; 98: 515-8.
33. Minich WB, Dehina N, Welsink T, et al. Autoantibodies to the IGF1 receptor in Graves' orbitopathy. J Clin Endocrinol Metab 2013; 98: 752-60.
34. Krieger CC, Place RF, Bevilacqua C, et al. TSH/IGF-1 receptor cross talk in Graves' ophthalmopathy pathogenesis. J Clin Endocrinol Metab 2016; 101: 2340-7.
35. Pappo AS, Patel SR, Crowley J, et al. R1507, a monoclonal antibody to the insulin- like growth factor 1 receptor, in patients with recurrent or refractory Ewing sarcoma family of tumors: Results of a phase II Sarcoma Alliance for Research through Collaboration study. J Clin Oncol 2011; 29: 4541-7.
36. Ramalingam SS, Spigel DR, Chen D, et al. Randomized phase II study of erlotinib in combination with placebo or R1507, a monoclonal antibody to insulinlike growth factor-1 receptor, for advancedstage non-small-cell lung cancer. J Clin Oncol 2011; 29: 4574-80.
37. Ma H, Zhang T, Shen H, Cao H, Du J. The adverse events profile of anti-IGF-1R monoclonal antibodies in cancer therapy. Br J Clin Pharmacol 2014; 77: 917-28.
38. Stan MN, Garrity JA, Carranza Leon BG, Prabin T, Bradley EA, Bahn RS. Randomized controlled trial of rituximab in patients with Graves' orbitopathy. J Clin Endocrinol Metab 2015; 100: 432-41.
39. Salvi M Vannucchi G Curr N et al. Efficacy of B-cell targeted therapy with rituximab in patients with active moderate to severe Graves' orbitopathy: A randomized controlled study. J Clin Endocrinol Metab 2015; 100: 422-31.
40. Pritchard J, Tsui S, Horst N, Cruikshank WW, Smith TJ. Synovial fibroblasts from patients with rheumatoid arthritis, like fibroblasts from Graves' disease, express high levels of IL-16 when treated with Igs against insulinlike growth factor-1 receptor. J Immunol 2004; 173: 3564-9.