On-label and off-label drug use in the treatment of endometriosis

Fertility and Sterility

Volumn 103, Issue 3, 2015, Pages 612-625

  Quaas, Alexander M ^a   Weedin, Elizabeth A ^a   Hansen, Karl R ^a  

^a UNIVERSITY OF OKLAHOMA COLLEGE OF MEDICINE   (United States)

Author keywords

Endometriosis; off label treatment

Indexed keywords

ANGIOGENESIS INHIBITOR; ANTIGESTAGEN; ANTIOXIDANT; AROMATASE INHIBITOR; CONTRACEPTIVE AGENT; CYCLOOXYGENASE 2 INHIBITOR; DANAZOL; GESTAGEN; GESTRINONE; GONADORELIN ANTAGONIST; GOSERELIN; HERBACEOUS AGENT; HYDROXYMETHYLGLUTARYL COENZYME A REDUCTASE INHIBITOR; IMMUNOMODULATING AGENT; LEUPRORELIN; LEVONORGESTREL; MEDROXYPROGESTERONE ACETATE; MELATONIN; NAFARELIN ACETATE; NORETHISTERONE ACETATE; PENTOXIFYLLINE; PEROXISOME PROLIFERATOR ACTIVATED RECEPTOR GAMMA AGONIST; PHYTOESTROGEN; PROGESTERONE RECEPTOR MODULATOR; SELECTIVE ESTROGEN RECEPTOR MODULATOR; VALPROIC ACID; GONADORELIN; ORAL CONTRACEPTIVE AGENT;

DECIDUALIZATION; DRUG APPROVAL; ENDOMETRIOSIS; ENDOMETRIUM; ESTROGEN ACTIVITY; HUMAN; INTRAUTERINE CONTRACEPTIVE DEVICE; META ANALYSIS (TOPIC); OFF LABEL DRUG USE; OVULATION INHIBITION; PHASE 2 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; REVIEW; AGONISTS; DRUG EFFECTS; DRUG LABELING; FEMALE; FOOD AND DRUG ADMINISTRATION; PRESCRIPTION; UNITED STATES;

CONTRACEPTIVES, ORAL, HORMONAL; DRUG LABELING; DRUG PRESCRIPTIONS; ENDOMETRIOSIS; FEMALE; GONADOTROPIN-RELEASING HORMONE; HUMANS; OFF-LABEL USE; OVULATION INHIBITION; PROGESTINS; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 84926135831     PISSN: 00150282     EISSN: 15565653     Source Type: Journal    
DOI: 10.1016/j.fertnstert.2015.01.006     Document Type: Review

References (97)

1. R.O. Burney, and L.C. Giudice Pathogenesis and pathophysiology of endometriosis Fertil Steril 98 2012 511 519
2. R.E. Batt, D.C. Martin, and K. Odunsi Endometriosis of the retrocervical septum is proposed to replace the anatomically incorrect term endometriosis of the rectovaginal septum Hum Reprod 29 2014 2603 2605
3. B. Eskenazi, and M.L. Warner Epidemiology of endometriosis Obstet Gynecol Clin North Am 24 1997 235 258
4. X. Gao, J. Outley, M. Botteman, J. Spalding, J.A. Simon, and C.L. Pashos Economic burden of endometriosis Fertil Steril 86 2006 1561 1572
5. J.A. Sampson Peritoneal endometriosis due to the menstrual dissemination of endometrial tissue into the peritoneal cavity Am J Obstet Gynecol 14 1927 422 469
6. S.E. Bulun Endometriosis N Engl J Med 360 2009 268 279
7. N. Panay Advances in the medical management of endometriosis BJOG 115 2008 814 817
8. L.C. Giudice Clinical practice. Endometriosis N Engl J Med 362 2010 2389 2398
9. E. Hughes, J. Brown, J.J. Collins, C. Farquhar, D.M. Fedorkow, and P. Vanderkerchove Ovulation suppression for endometriosis Cochrane Database Syst Rev 2007 CD000155
10. H.N. Sallam, J.A. Garcia-Velasco, S. Dias, and A. Arici Long-term pituitary down-regulation before in vitro fertilization (IVF) for women with endometriosis Cochrane Database Syst Rev 2006 CD004635
11. G.A. Dunselman, N. Vermeulen, C. Becker, C. Calhaz-Jorge, T. d'Hooghe, and B. de Bie ESHRE guideline: management of women with endometriosis Hum Reprod 29 2014 400 412
12. Practice Committee of the American Society for Reproductive Medicine Endometriosis and infertility: a committee opinion Fertil Steril 98 2012 591 598
13. S.R. Soares, A. Martínez-Varea, J.J. Hidalgo-Mora, and A. Pellicer Pharmacologic therapies in endometriosis: a systematic review Fertil Steril 98 2012 529 555
14. M.R. Henzl, S.L. Corson, K. Moghissi, V.C. Buttram, C. Berqvist, and J. Jacobson Administration of nasal nafarelin as compared with oral danazol for endometriosis. A multicenter double-blind comparative clinical trial N Engl J Med 318 1988 485 489
15. A. Prentice, A.J. Deary, S. Goldbeck-Wood, C. Farquhar, and S.K. Smith Gonadotrophin-releasing hormone analogues for pain associated with endometriosis Cochrane Database Syst Rev 2000 CD000346
16. A.M. Dlugi, J.D. Miller, and J. Knittle Lupron depot (leuprolide acetate for depot suspension) in the treatment of endometriosis: a randomized, placebo-controlled, double-blind study. Lupron Study Group Fertil Steril 54 1990 419 427
17. D. Kappou, M. Matalliotakis, and I. Matalliotakis Medical treatments for endometriosis Minerva Ginecol 62 2010 415 432
18. B. Ruhland, A. Agic, J. Krampe, K. Diedrich, and D. Hornung Innovations in conservative endometriosis treatment: an updated review Minerva Ginecol 63 2011 247 259
19. P. Vercellini, E. Somigliana, P. Vigano, A. Abbiati, G. Barbara, and P.G. Crosignani Endometriosis: current therapies and new pharmacological developments Drugs 69 2009 649 675
20. E. Somigliana, P. Vigano, G. Barbara, and P. Vercellini Treatment of endometriosis-related pain: options and outcomes Front Biosci (Elite Ed) 1 2009 455 465
21. I. Gerhard, A.E. Schindler, K. Bühler, U. Winkler, K. Meinen, and D. Mancarella Treatment of endometriosis with leuprorelin acetate depot: a German multicentre study Clin Ther 14 Suppl A 1992 3 16
22. J.S. Johansen, B.J. Riis, C. Hassager, M. Moen, J. Jacobson, and C. Christiansen The effect of a gonadotropin-releasing hormone agonist analog (nafarelin) on bone metabolism J Clin Endocrinol Metab 67 1988 701 706
23. A.J. Friedman, D.I. Hoffman, F. Comite, R.W. Browneller, and J.D. Miller Treatment of leiomyomata uteri with leuprolide acetate depot: a double-blind, placebo-controlled, multicenter study. Leuprolide Study Group Obstet Gynecol 77 1991 720 725
24. M. Sagsveen, J.E. Farmer, A. Prentice, and A. Breeze Gonadotrophin-releasing hormone analogues for endometriosis: bone mineral density Cochrane Database Syst Rev 2003 CD001297
25. D.L. Olive, and E.A. Pritts Treatment of endometriosis N Engl J Med 345 2001 266 275
26. K.S. Moghissi Medical treatment of endometriosis Clin Obstet Gynecol 42 1999 620 632
27. R.L. Barbieri Hormone treatment of endometriosis: the estrogen threshold hypothesis Am J Obstet Gynecol 166 1992 740 745
28. J. Jain, A.J. Jakimiuk, F.R. Bode, D. Ross, and A.M. Kaunitz Contraceptive efficacy and safety of DMPA-SC Contraception 70 2004 269 275
29. P. Vercellini, I. Cortesi, and P.G. Crosignani Progestins for symptomatic endometriosis: a critical analysis of the evidence Fertil Steril 68 1997 393 401
30. D. Scholes, A.Z. LaCroix, L.E. Ichikawa, W.E. Barlow, and S.M. Ott Change in bone mineral density among adolescent women using and discontinuing depot medroxyprogesterone acetate contraception Arch Pediatr Adolesc Med 159 2005 139 144
31. V. Selak, C. Farquhar, A. Prentice, and A. Singla Danazol for pelvic pain associated with endometriosis Cochrane Database Syst Rev 2007 CD000068
32. S. Telimaa, J. Puolakka, and A. Kauppila Placebo-controlled comparison of danazol and high-dose medroxyprogesterone acetate in the treatment of endometriosis Gynecol Endocrinol 1 1987 13 23
33. M.R. Laufer, J. Sanfilippo, and G. Rose Adolescent endometriosis: diagnosis and treatment approaches J Pediatr Adolesc Gynecol 16 3 Suppl 2003 S3 S11
34. T. Harada, M. Momoeda, Y. Taketani, H. Hoshiai, and N. Terakawa Low-dose oral contraceptive pill for dysmenorrhea associated with endometriosis: a placebo-controlled, double-blind, randomized trial Fertil Steril 90 2008 1583 1588
35. P. Vercellini, G. Frontino, O. de Giorgi, G. Pietropaolo, R. Pasin, and P.G. Crosignani Continuous use of an oral contraceptive for endometriosis-associated recurrent dysmenorrhea that does not respond to a cyclic pill regimen Fertil Steril 80 2003 560 563
36. N.G. Mahutte, and A. Arici Medical management of endometriosis-associated pain Obstet Gynecol Clin North Am 30 2003 133 150
37. T. Strowitzki, J. Marr, C. Gerlinger, T. Faustmann, and C. Seitz Dienogest is as effective as leuprolide acetate in treating the painful symptoms of endometriosis: a 24-week, randomized, multicentre, open-label trial Hum Reprod 25 2010 633 641
38. T. Strowitzki, J. Marr, C. Gerlinger, T. Faustmann, and C. Seitz Detailed analysis of a randomized, multicenter, comparative trial of dienogest versus leuprolide acetate in endometriosis Int J Gynaecol Obstet 117 2012 228 233
39. K.L. Whalen, and R. Rose Estradiol valerate/dienogest: a novel oral contraceptive Ann Pharmacother 45 2011 1256 1261
40. W. Küpker, R.E. Felberbaum, M. Krapp, T. Schill, E. Malik, and K. Diedrich Use of GnRH antagonists in the treatment of endometriosis Reprod Biomed Online 5 2002 12 16
41. R.S. Struthers, A.J. Nicholls, J. Grundy, T. Chen, R. Jimenez, and S.S. Yen Suppression of gonadotropins and estradiol in premenopausal women by oral administration of the nonpeptide gonadotropin-releasing hormone antagonist elagolix J Clin Endocrinol Metab 94 2009 545 551
42. M.P. Diamond, B. Carr, W.P. Dmowski, W. Koltun, C. O'Brien, and P. Jiang Elagolix treatment for endometriosis-associated pain: results from a phase 2, randomized, double-blind, placebo-controlled study Reprod Sci 21 2014 363 371
43. C. Allen, S. Hopewell, A. Prentice, and C. Allen Nonsteroidal antiinflammatory drugs for pain in women with endometriosis Cochrane Database Syst Rev 2009 CD004753
44. A. Kauppila, and L. Ronnberg Naproxen sodium in dysmenorrhea secondary to endometriosis Obstet Gynecol 65 1985 379 383
45. E.C. Hayes, and J.A. Rock COX-2 inhibitors and their role in gynecology Obstet Gynecol Surv 57 2002 768 780
46. L. Cobellis, S. Razzi, S. De Simone, A. Sartini, A. Fava, and S. Danero The treatment with a COX-2 specific inhibitor is effective in the management of pain related to endometriosis Eur J Obstet Gynecol Reprod Biol 116 2004 100 102
47. M.B. Moravek, E.A. Ward, and D.I. Lebovic Thiazolidinediones as therapy for endometriosis: a case series Gynecol Obstet Invest 68 2009 167 170
48. S. Alborzi, S. Ghotbi, M.E. Parsanezhad, S. Dehbashi, S. Alborzi, and M. Alborzi Pentoxifylline therapy after laparoscopic surgery for different stages of endometriosis: a prospective, double-blind, randomized, placebo-controlled study J Minim Invasive Gynecol 14 2007 54 58
49. M. Creus, F. Fábregues, F. Carmona, M. del Pino, D. Manau, and J. Balasch Combined laparoscopic surgery and pentoxifylline therapy for treatment of endometriosis-associated infertility: a preliminary trial Hum Reprod 23 2008 1910 1916
50. H. Kamencic, and J.A. Thiel Pentoxifylline after conservative surgery for endometriosis: a randomized, controlled trial J Minim Invasive Gynecol 15 2008 62 66
51. J. Mier-Cabrera, T. Aburto-Soto, S. Burrola-Méndez, L. Jiménez-Zamudio, M.C. Tolentino, and E. Casanueva Women with endometriosis improved their peripheral antioxidant markers after the application of a high antioxidant diet Reprod Biol Endocrinol 7 2009 54
52. E.R. Simpson, M.S. Mahendroo, G.D. Means, M.W. Kilgore, M.M. Hinshelwood, and S. Graham-Lorence Aromatase cytochrome P450, the enzyme responsible for estrogen biosynthesis Endocr Rev 15 1994 342 355
53. S.E. Bulun, K. Zeitoun, K. Takayama, L. Noble, D. Michael, and E. Simpson Estrogen production in endometriosis and use of aromatase inhibitors to treat endometriosis Endocr Relat Cancer 6 1999 293 301
54. K.M. Zeitoun, and S.E. Bulun Aromatase: a key molecule in the pathophysiology of endometriosis and a therapeutic target Fertil Steril 72 1999 961 969
55. F. Abushahin, K.N. Goldman, E. Barbieri, M. Milad, A. Rademaker, and S.E. Bulun Aromatase inhibition for refractory endometriosis-related chronic pelvic pain Fertil Steril 96 2011 939 942
56. S. Soysal, M.E. Soysal, S. Ozer, N. Gul, and T. Gezgin The effects of post-surgical administration of goserelin plus anastrozole compared to goserelin alone in patients with severe endometriosis: a prospective randomized trial Hum Reprod 19 2004 160 167
57. R.K. Ailawadi, S. Jobanputra, M. Kataria, B. Gurates, and S.E. Bulun Treatment of endometriosis and chronic pelvic pain with letrozole and norethindrone acetate: a pilot study Fertil Steril 81 2004 290 296
58. V. Remorgida, L.H. Abbamonte, N. Ragni, E. Fulcheri, and S. Ferrero Letrozole and desogestrel-only contraceptive pill for the treatment of stage IV endometriosis Aust N Z J Obstet Gynaecol 47 2007 222 225
59. T. Tulandi, J. Martin, R. Al-Fadhli, N. Kabli, R. Forman, and J. Hitkari Congenital malformations among 911 newborns conceived after infertility treatment with letrozole or clomiphene citrate Fertil Steril 85 2006 1761 1765
60. R.S. Legro, R.G. Brzyski, M.P. Diamond, C. Coutifaris, W.D. Schlaff, and R. Alvero The Pregnancy in Polycystic Ovary Syndrome II study: baseline characteristics and effects of obesity from a multicenter randomized clinical trial Fertil Steril 101 2014 258 269.e8
61. R.S. Legro, R.G. Brzyski, M.P. Diamond, C. Coutifaris, W.D. Schlaff, and P. Casson Letrozole versus clomiphene for infertility in the polycystic ovary syndrome N Engl J Med 371 2014 119 129
62. P. Vercellini, G. Frontino, O. de Giorgi, G. Aimi, B. Zaina, and P.G. Crosignani Comparison of a levonorgestrel-releasing intrauterine device versus expectant management after conservative surgery for symptomatic endometriosis: a pilot study Fertil Steril 80 2003 305 309
63. C.A. Petta, R.A. Ferriani, M.S. Abrao, D. Hassan, J.C.R. de Silva, and S. Podgaec Randomized clinical trial of a levonorgestrel-releasing intrauterine system and a depot GnRH analogue for the treatment of chronic pelvic pain in women with endometriosis Hum Reprod 20 2005 1993 1998
64. F.B. Lockhat, J.O. Emembolu, and J.C. Konje The efficacy, side-effects and continuation rates in women with symptomatic endometriosis undergoing treatment with an intra-uterine administered progestogen (levonorgestrel): a 3 year follow-up Hum Reprod 20 2005 789 793
65. L.M. Kettel, A.A. Murphy, A.E. Morales, A. Ulmann, E.E. Baulieu, and S.S. Yen Treatment of endometriosis with the antiprogesterone mifepristone (RU486) Fertil Steril 65 1996 23 28
66. L.M. Kettel, A.A. Murphy, A.J. Morales, and S.S. Yen Preliminary report on the treatment of endometriosis with low-dose mifepristone (RU 486) Am J Obstet Gynecol 178 1998 1151 1156
67. D. Morris, J. Podolski, A. Kirsch, R. Wiehle, and L. Fleckenstein Population pharmacokinetics of telapristone (CDB-4124) and its active monodemethylated metabolite CDB-4453, with a mixture model for total clearance AAPS J 13 2011 665 673
68. O.B. Ioffe, R.J. Zaino, and G.L. Mutter Endometrial changes from short-term therapy with CDB-4124, a selective progesterone receptor modulator Mod Pathol 22 2009 450 459
69. I. Streuli, D. de Ziegler, P. Santulli, L. Marcellin, B. Borghese, and F. Batteux An update on the pharmacological management of endometriosis Expert Opin Pharmacother 14 2013 291 305
70. C.A. Huniadi, O.L. Pop, T.A. Antal, and F. Stamatian The effects of ulipristal on Bax/Bcl-2, cytochrome c, Ki-67 and cyclooxygenase-2 expression in a rat model with surgically induced endometriosis Eur J Obstet Gynecol Reprod Biol 169 2013 360 365
71. P. Stratton, N. Sinaii, J. Segars, D. Koziol, R. Wesley, and C. Zimmer Return of chronic pelvic pain from endometriosis after raloxifene treatment: a randomized controlled trial Obstet Gynecol 111 2008 88 96
72. K. Nasu, A. Yuge, A. Tsuno, and H. Narahara Simvastatin inhibits the proliferation and the contractility of human endometriotic stromal cells: a promising agent for the treatment of endometriosis Fertil Steril 92 2009 2097 2099
73. F. Almassinokiani, A. Mehdizadeh, E. Sariri, M. Rezaei, A. Almasi, and H. Akbari Effects of simvastatin in prevention of pain recurrences after surgery for endometriosis Med Sci Monit 19 2013 534 539
74. D. Altintas, A. Kokcu, B. Kandemir, M.B. Cetinkaya, and M. Tosun Efficacy of imiquimod, an immunomodulatory agent, on experimental endometriosis Fertil Steril 90 2008 401 405
75. D. Altintas, A. Kokcu, M. Tosun, M.B. Cetinkaya, and B. Kandemir Efficacy of recombinant human interferon alpha-2b on experimental endometriosis Eur J Obstet Gynecol Reprod Biol 139 2008 95 99
76. K. Shakiba, and T. Falcone Tumour necrosis factor-alpha blockers: potential limitations in the management of advanced endometriosis? A case report Hum Reprod 21 2006 2417 2420
77. P.R. Koninckx, M. Craessaerts, D. Timmerman, F. Cornillie, and S. Kennedy Anti-TNF-alpha treatment for deep endometriosis-associated pain: a randomized placebo-controlled trial Hum Reprod 23 2008 2017 2023
78. K. May, and C.M. Becker Endometriosis and angiogenesis Minerva Ginecol 60 2008 245 254
79. C.M. Becker, D.A. Sampson, M.A. Rupnick, R.M. Rohan, J.A. Efstathiou, and S.M. Short Endostatin inhibits the growth of endometriotic lesions but does not affect fertility Fertil Steril 84 Suppl 2 2005 1144 1155
80. C.M. Becker, D.A. Sampson, S.M. Short, K. Javaherian, J. Folkman, and R.J. D'Amato Short synthetic endostatin peptides inhibit endothelial migration in vitro and endometriosis in a mouse model Fertil Steril 85 2006 71 77
81. C.M. Becker, R.D. Wright, R. Satchi-Fainaro, T. Funakoshi, J. Folkman, and A.L. Kung A novel noninvasive model of endometriosis for monitoring the efficacy of antiangiogenic therapy Am J Pathol 168 2006 2074 2084
82. T.M. d'Hooghe, N.P. Nugent, S. Cuneo, D.C. Chai, F. Deer, and S. Debrock Recombinant human TNFRSF1A (r-hTBP1) inhibits the development of endometriosis in baboons: a prospective, randomized, placebo- and drug-controlled study Biol Reprod 74 2006 131 136
83. R.N. Taylor, J. Yu, P.B. Torres, A.C. Schickedanz, J.K. Park, and M.D. Mueller Mechanistic and therapeutic implications of angiogenesis in endometriosis Reprod Sci 16 2009 140 146
84. R. Gómez, A. Abad, F. Delgado, S. Tamarit, C. Simón, and A. Pellicer Effects of hyperprolactinemia treatment with the dopamine agonist quinagolide on endometriotic lesions in patients with endometriosis-associated hyperprolactinemia Fertil Steril 95 2011 882 888.e1
85. E.M. Coutinho Treatment of endometriosis with gestrinone (R-2323), a synthetic antiestrogen, antiprogesterone Am J Obstet Gynecol 144 1982 895 898
86. P.L. Venturini, S. Bertolini, M.C. Brunenghi, A. Daga, V. Fasce, and A. Marcenaro Endocrine, metabolic, and clinical effects of gestrinone in women with endometriosis Fertil Steril 52 1989 589 595
87. Y. Wu, and S.W. Guo Histone deacetylase inhibitors trichostatin A and valproic acid induce cell cycle arrest and p21 expression in immortalized human endometrial stromal cells Eur J Obstet Gynecol Reprod Biol 137 2008 198 203
88. X. Liu, and S.W. Guo A pilot study on the off-label use of valproic acid to treat adenomyosis Fertil Steril 89 2008 246 250
89. R.J. Reiter, D.X. Tan, and M.D. Maldonado Melatonin as an antioxidant: physiology versus pharmacology J Pineal Res 39 2005 215 216
90. G. Yildirim, R. Attar, F. Ozkan, B. Kumbak, C. Ficicioglu, and N. Yesildaglar The effects of letrozole and melatonin on surgically induced endometriosis in a rat model: a preliminary study Fertil Steril 93 2010 1787 1792
91. A. Schwertner, C.C. Conceição dos Santos, G.D. Costa, A. Deitos, A. de Souza, and I.C.C. de Souza Efficacy of melatonin in the treatment of endometriosis: a phase II, randomized, double-blind, placebo-controlled trial Pain 154 2013 874 881
92. M.W. Laschke, C. Schwender, B. Vollmar, and M.D. Menger Genistein does not affect vascularization and blood perfusion of endometriotic lesions and ovarian follicles in dorsal skinfold chambers of Syrian golden hamsters Reprod Sci 17 2010 568 577
93. E. Yavuz, M. Oktem, I. Esinler, S.A. Toru, and H.B. Zeyneloglu Genistein causes regression of endometriotic implants in the rat model Fertil Steril 88 4 Suppl 2007 1129 1134
94. T. Kohama, K. Herai, and M. Inoue Effect of French maritime pine bark extract on endometriosis as compared with leuprorelin acetate J Reprod Med 52 2007 703 708
95. H. Cox, L. Henderson, R. Wood, and G. Cagliarini Learning to take charge: women's experiences of living with endometriosis Complement Ther Nurs Midwifery 9 2003 62 68
96. A. Fugh-Berman, and F. Kronenberg Complementary and alternative medicine (CAM) in reproductive-age women: a review of randomized controlled trials Reprod Toxicol 17 2003 137 152
97. F. Wieser, M. Cohen, A. Gaeddert, J. Yu, C. Burks-Wicks, and S.L. Berga Evolution of medical treatment for endometriosis: back to the roots? Hum Reprod Update 13 2007 487 499