Botulinum neurotoxins for the treatment of focal dystonias: Review of rating tools used in clinical trials

Toxicon

Volumn 107, Issue , 2015, Pages 89-97

  Del Sorbo, Francesca ^a   Albanese, Alberto ^b,c  

^a DEPARTMENT OF NEUROSURGERY   (Italy)

^b HUMANITAS CLINICAL AND RESEARCH CENTER   (Italy)

^c UNIVERSITÀ CATTOLICA DEL SACRO CUORE   (Italy)

Author keywords

Botulinum neurotoxin; Dystonia; Focal dystonia; Outcome measure; Rating scales

Indexed keywords

BOTULINUM TOXIN; PLACEBO;

ARM DYSTONIA DISABILITY SCALE; ARTICLE; BLEPHAROSPASM; BLEPHAROSPASM DISABILITY INDEX; BLEPHAROSPASM DISABILITY SCALE; BODY CONCEPT SCALE; CERVICAL DYSTONIA; CERVICAL DYSTONIA IMPACT SCALE; CRANIOCERVICAL DYSTONIA QUESTIONNAIRE; DEPRESSION; DISEASE SEVERITY; DRUG EFFICACY; FOCAL DYSTONIA; FUNCTIONAL DISABILITY QUESTIONNAIRE; GLOBAL DYSTONIA RATING SCALE; HUMAN; JANKOVIC RATING SCALE; META ANALYSIS; OROMANDIBULAR DYSTONIA; OROMANDIBULAR DYSTONIA QUESTIONNAIRE; PAIN; PRIORITY JOURNAL; QUALITY OF LIFE; RATING SCALE; SPASMODIC DYSPHONIA; SYSTEMATIC REVIEW; TORONTO WESTERN SPASMODIC TORTICOLLIS RATING SCALE; TREATMENT OUTCOME; TSUI SCALE; TUBIANA CHAMAGNE SCORE; UNIFIED DYSTONIA RATING SCALE; UNIFIED SPASMODIC DYSPHONIA RATING SCALE; UPPER LIMB DYSTONIA; VOCAL PERFORMANCE QUESTIONNAIRE; VOICE HANDICAP INDEX; VOICE RELATED QUALITY OF LIFE SCALE; WRITER CRAMP RATING SCALE; DYSTONIC DISORDERS; PROCEDURES; RANDOMIZED CONTROLLED TRIAL (TOPIC); SEVERITY OF ILLNESS INDEX;

BOTULINUM TOXINS; DYSTONIC DISORDERS; HUMANS; RANDOMIZED CONTROLLED TRIALS AS TOPIC; SEVERITY OF ILLNESS INDEX; TREATMENT OUTCOME;

EID: 84947774874     PISSN: 00410101     EISSN: 18793150     Source Type: Journal    
DOI: 10.1016/j.toxicon.2015.09.010     Document Type: Article

References (71)

1. N. Aaronson, J. Alonso, A. Burnam, K.N. Lohr, D.L. Patrick, E. Perrin, and R.E.K. Stein Assessing health status and quality-of-life instruments: attributes and review criteria Qual. Life Res. 11 2002 193 205
2. S.G. Adams, E.J. Hunt, J.C. Irish, D.A. Charles, A.E. Lang, L.C. Durkin, and D.L. Wong Comparison of botulinum toxin injection procedures in adductor spasmodic dysphonia J. Otolaryngol. 24 1995 345 351
3. C.H. Adler, S.F. Bansberg, K. Krein-Jones, and J.G. Hentz Safety and efficacy of botulinum toxin type B (Myobloc) in adductor spasmodic dysphonia Mov. Disord. 19 2004 1075 1079
4. A. Albanese Deep brain stimulation for cervical dystonia Lancet Neurol. 13 2014 856 857
5. A. Albanese, F. Asmus, K.P. Bhatia, A.E. Elia, B. Elibol, G. Filippini, T. Gasser, J.K. Krauss, N. Nardocci, A. Newton, and J. Valls-Sole EFNS guidelines on diagnosis and treatment of primary dystonias Eur. J. Neurol. 18 2011 5 18
6. A. Albanese, F. Del Sorbo, C. Comella, H.A. Jinnah, J.W. Mink, B. Post, M. Vidailhet, J. Volkmann, T.T. Warner, A.F. Leentjens, P. Martinez-Martin, G.T. Stebbins, C.G. Goetz, and A. Schrag Dystonia rating scales: critique and recommendations Mov. Disord. 28 2013 874 883
7. A. Albanese, L.M. Romito, and D. Calandrella Therapeutic advances in dystonia Mov. Disord. 30 2015 1547 1556
8. I. Atroshi, W.C. Breidenbach, and S.J. McCabe Assessment of the carpal tunnel outcome instrument in patients with nerve-compression symptoms J. Hand Surg. Am. 22 1997 222 227
9. R. Benecke, W.H. Jost, P. Kanovsky, E. Ruzicka, G. Comes, and S. Grafe A new botulinum toxin type A free of complexing proteins for treatment of cervical dystonia Neurology 64 2005 1949 1951
10. S. Bielamowicz, and C.L. Ludlow Effects of botulinum toxin on pathophysiology in spasmodic dysphonia Ann. Otol. Rhinol. Laryngol. 109 2000 194 203
11. S. Bielamowicz, S. Squire, K. Bidus, and C.L. Ludlow Assessment of posterior cricoarytenoid botulinum toxin injections in patients with abductor spasmodic dysphonia Ann. Otol. Rhinol. Laryngol. 110 2001 406 412
12. M.H. Boyle, G. McGwin Jr., C.E. Flanagan, M.G. Vicinanzo, and J.A. Long High versus low concentration botulinum toxin A for benign essential blepharospasm: does dilution make a difference? Ophthal. Plast. Reconstr. Surg. 25 2009 81 84
13. J.W. Brans, R. Lindeboom, J.W. Snoek, M.J. Zwarts, T.W. van Weerden, E.R. Brunt, J.J. van Hilten, W. van der Kamp, M.H. Prins, and J.D. Speelman Botulinum toxin versus trihexyphenidyl in cervical dystonia: a prospective, randomized, double-blind controlled trial Neurology 46 1996 1066 1072
14. A. Brashear, M.F. Lew, D.D. Dykstra, C.L. Comella, S.A. Factor, R.L. Rodnitzky, R. Trosch, C. Singer, M.F. Brin, J.J. Murray, J.D. Wallace, A. Willmer-Hulme, and M. Koller Safety and efficacy of NeuroBloc (botulinum toxin type B) in type A-responsive cervical dystonia Neurology 53 1999 1439 1446
15. M.F. Brin, J. Jankovic, C. Comella, A. Blitzer, J.K. Tsui, and S.L. Pullman Treatment of dystonia using botulinum toxin R. Kurlan, Treatment of Movement Disorders 1995 Lippincott New York 183 246
16. M.F. Brin, M.F. Lew, C.H. Adler, C.L. Comella, S.A. Factor, J. Jankovic, C. O'Brien, J.J. Murray, J.D. Wallace, A. Willmer-Hulme, and M. Koller Safety and efficacy of NeuroBloc (botulinum toxin type B) in type A-resistant cervical dystonia Neurology 53 1999 1431 1438
17. R.E. Burke, S. Fahn, C.D. Marsden, S.B. Bressman, C. Moskowitz, and J. Friedman Validity and reliability of a rating scale for the primary torsion dystonias Neurology 35 1985 73 77
18. S.J. Cano, T.T. Warner, J.M. Linacre, K.P. Bhatia, A.J. Thompson, R. Fitzpatrick, and J.C. Hobart Capturing the true burden of dystonia on patients: the Cervical Dystonia Impact Profile (CDIP-58) Neurology 63 2004 1629 1633
19. R. Chen, B.I. Karp, S.R. Goldstein, W. Bara-Jimenez, Z. Yaseen, and M. Hallett Effect of muscle activity immediately after botulinum toxin injection for writer's cramp Mov. Disord. 14 1999 307 312
20. R. Cole, M. Hallett, and L.G. Cohen Double-blind trial of botulinum toxin for treatment of focal hand dystonia Mov. Disord. 10 1995 466 471
21. C.L. Comella, J. Jankovic, K.M. Shannon, J. Tsui, M. Swenson, S. Leurgans, and W. Fan Comparison of botulinum toxin serotypes A and B for the treatment of cervical dystonia Neurology 65 2005 1423 1429
22. ®, botulinum neurotoxin type A, without accessory proteins) in patients with cervical dystonia J. Neurol. Sci. 308 2011 103 109
23. C.L. Comella, S. Leurgans, J. Wuu, G.T. Stebbins, and T. Chmura Rating scales for dystonia: a multicenter assessment Mov. Disord. 18 2003 303 312
24. E. Consky, A. Basinski, L. Belle, R. Ranawaya, and A. Lang The Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS): assessment of validity and inter-rater reliability Neurology 40 Suppl. 1 1990 445
25. M.F. Contarino, J.J. Kruisdijk, L. Koster, B.W. Ongerboer de Visser, J.D. Speelman, and J.H. Koelman Sensory integration in writer's cramp: comparison with controls and evaluation of botulinum toxin effect Clin. Neurophysiol. 118 2007 2195 2206
26. A.F. de Bruin, L.P. de Witte, F. Stevens, and J.P. Diederiks Sickness impact profile: the state of the art of a generic functional status measure Soc. Sci. Med. 35 1992 1003 1014
27. R.A. Deyo, P. Diehr, and D.L. Patrick Reproducibility and responsiveness of health status measures. Statistics and strategies for evaluation Control Clin. Trials 12 Suppl. l 1991 142S 158S
28. S. Fahn Assessment of the primary dystonias T. Munsat, The Quantification of Neurological Deficit 1989 Butterworths Boston 241 270
29. E.M. Finnegan, E.S. Luschei, J.D. Gordon, J.M. Barkmeier, and H.T. Hoffman Increased stability of airflow following botulinum toxin injection Laryngoscope 109 1999 1300 1306
30. R. Fitzpatrick, C. Davey, M.J. Buxton, and D.R. Jones Evaluating patient-based outcome measures for use in clinical trials Health Technol. Assess. 2 1998 1 74
31. C. Geenen, E. Consky, and P. Ashby Localizing muscles for botulinum toxin treatment of focal hand dystonia Can. J. Neurol. Sci. 23 1996 194 197
32. P. Girlanda, A. Quartarone, S. Sinicropi, C. Nicolosi, and C. Messina Unilateral injection of botulinum toxin in blepharospasm: single fiber electromyography and blink reflex study Mov. Disord. 11 1996 27 31
33. R.A. Gross, and K.C. Johnston Levels of evidence: taking neurology to the next level Neurology 72 2009 8 10
34. M. Hallett, A. Albanese, D. Dressler, K.R. Segal, D.M. Simpson, D. Truong, and J. Jankovic Evidence-based review and assessment of botulinum neurotoxin for the treatment of movement disorders Toxicon 67 2013 94 114
35. J. Jankovic, C. Comella, A. Hanschmann, and S. Grafe Efficacy and safety of incobotulinumtoxinA (NT 201, Xeomin) in the treatment of blepharospasm-a randomized trial Mov. Disord. 26 2011 1521 1528
36. J. Jankovic, C. Kenney, S. Grafe, R. Goertelmeyer, and G. Comes Relationship between various clinical outcome assessments in patients with blepharospasm Mov. Disord. 24 2009 407 413
37. J. Jankovic, and J. Orman Botulinum A toxin for cranial-cervical dystonia: a double-blind, placebo-controlled study Neurology 37 1987 616 623
38. C. Jenkinson, L. Wright, and A. Coulter The SF 36 health survey questionnaire.if used within its limits BMJ 307 1993 449
39. H.A. Jinnah, and S.A. Factor Diagnosis and treatment of dystonia Neurol. Clin. 33 2015 77 100
40. P. Kanovsky, and R.L. Rosales Debunking the pathophysiological puzzle of dystonia-with special reference to botulinum toxin therapy Parkinsonism. Relat. Disord. 17 Suppl. 1 2011 S11 S14
41. J.J. Kruisdijk, J.H. Koelman, B.W. Ongerboer de Visser, R.J. De Haan, and J.D. Speelman Botulinum toxin for writer's cramp: a randomised, placebo-controlled trial and 1-year follow-up J. Neurol. Neurosurg. Psychiatry 78 2007 264 270
42. S.J. Lee, W.D. McCall Jr., Y.K. Kim, S.C. Chung, and J.W. Chung Effect of botulinum toxin injection on nocturnal bruxism: a randomized controlled trial Am. J. Phys. Med. Rehabil. 89 2010 16 23
43. M.F. Lew, B.T. Adornato, D.D. Duane, D.D. Dykstra, S.A. Factor, J.M. Massey, M.F. Brin, J. Jankovic, R.L. Rodnitzky, C. Singer, M.R. Swenson, D. Tarsy, J.J. Murray, M. Koller, and J.D. Wallace Botulinum toxin type B: a double-blind, placebo-controlled, safety and efficacy study in cervical dystonia Neurology 49 1997 701 707
44. R. Lindeboom, R. De Haan, M. Aramideh, and J.D. Speelman The blepharospasm disability scale: an instrument for the assessment of functional health in blepharospasm Mov. Disord. 10 1995 444 449
45. C.L. Ludlow, R.F. Naunton, S.E. Sedory, G.M. Schulz, and M. Hallett Effects of botulinum toxin injections on speech in adductor spasmodic dysphonia Neurology 38 1988 1220 1225
46. R.I. Merz, J. Deakin, and M.R. Hawthorne Oromandibular dystonia questionnaire (OMDQ-25): a valid and reliable instrument for measuring health-related quality of life Clin. Otolaryngol. 35 2010 390 396
47. S. Messick Validation of inferences from persons' responses and performances as scientific enquiry into score meaning Am. Psychol. 50 1995 741 749
48. D. Moher, K.F. Schulz, and D. Altman The CONSORT statement: revised recommendations for improving the quality of reports of parallel-group randomized trials JAMA 285 2001 1987 1991
49. F.M. Molloy, H.A. Shill, A. Kaelin-Lang, and B.I. Karp Accuracy of muscle localization without EMG: implications for treatment of limb dystonia Neurology 58 2002 805 807
50. S.A. Montgomery, and M. Asberg A new depression scale designed to be sensitive to change Br. J. Psychiatry 134 1979 382 389
51. J. Muller, J. Wissel, G. Kemmler, B. Voller, T. Bodner, A. Schneider, G.K. Wenning, and W. Poewe Craniocervical dystonia questionnaire (CDQ-24): development and validation of a disease-specific quality of life instrument J. Neurol. Neurosurg. Psychiatry 75 2004 749 753
52. Z. Nussgens, and P. Roggenkamper Comparison of two botulinum-toxin preparations in the treatment of essential blepharospasm Graefes Arch. Clin. Exp. Ophthalmol. 235 1997 197 199
53. S. Ochudlo, P. Bryniarski, and G. Opala Botulinum toxin improves the quality of life and reduces the intensification of depressive symptoms in patients with blepharospasm Parkinsonism. Relat. Disord. 13 2007 505 508
54. T. Odergren, H. Hjaltason, S. Kaakkola, G. Solders, J. Hanko, C. Fehling, R.J. Marttila, H. Lundh, S. Gedin, I. Westergren, A. Richardson, C. Dott, and H. Cohen A double blind, randomised, parallel group study to investigate the dose equivalence of Dysport and Botox in the treatment of cervical dystonia J. Neurol. Neurosurg. Psychiatry 64 1998 6 12
55. E.J. Pappert, and T. Germanson Botulinum toxin type B vs. type A in toxin-naive patients with cervical dystonia: randomized, double-blind, noninferiority trial Mov. Disord. 23 2008 510 517
56. W. Poewe, G. Deuschl, A. Nebe, E. Feifel, J. Wissel, R. Benecke, K.R. Kessler, A.O. Ceballos-Baumann, A. Ohly, W. Oertel, and G. Kunig What is the optimal dose of botulinum toxin A in the treatment of cervical dystonia? Results of a double blind, placebo controlled, dose ranging study using Dysport. German Dystonia Study Group J. Neurol. Neurosurg. Psychiatry 64 1998 13 17
57. P. Roggenkamper, W.H. Jost, K. Bihari, G. Comes, and S. Grafe Efficacy and safety of a new botulinum toxin type A free of complexing proteins in the treatment of blepharospasm J. Neural Transm. 113 2006 303 312
58. C. Sampaio, J.J. Ferreira, F. Simoes, M.J. Rosas, M. Magalhaes, A.P. Correia, A. Bastos-Lima, R. Martins, and A. Castro-Caldas DYSBOT: a single-blind, randomized parallel study to determine whether any differences can be detected in the efficacy and tolerability of two formulations of botulinum toxin type A-Dysport and Botox-assuming a ratio of 4:1 Mov. Disord. 12 1997 1013 1018
59. A.L. Stewart, R.D. Hays, and J.E. Ware Jr. The MOS short-form general health survey. Reliability and validity in a patient population Med. Care 26 1988 724 735
60. D.D. Troung, M. Rontal, M. Rolnick, A.E. Aronson, and K. Mistura Double-blind controlled study of botulinum toxin in adductor spasmodic dysphonia Laryngoscope 101 1991 630 634
61. D. Truong, M. Brodsky, M. Lew, A. Brashear, J. Jankovic, E. Molho, O. Orlova, and S. Timerbaeva Long-term efficacy and safety of botulinum toxin type A (Dysport) in cervical dystonia Parkinsonism. Relat. Disord. 16 2010 316 323
62. D. Truong, C. Comella, H.H. Fernandez, and W.G. Ondo Efficacy and safety of purified botulinum toxin type A (Dysport) for the treatment of benign essential blepharospasm: a randomized, placebo-controlled, phase II trial Parkinsonism. Relat. Disord. 14 2008 407 414
63. D. Truong, D.D. Duane, J. Jankovic, C. Singer, L.C. Seeberger, C.L. Comella, M.F. Lew, R.L. Rodnitzky, F.O. Danisi, J.P. Sutton, P.D. Charles, R.A. Hauser, and G.L. Sheean Efficacy and safety of botulinum type A toxin (Dysport) in cervical dystonia: results of the first US randomized, double-blind, placebo-controlled study Mov. Disord. 20 2005 783 791
64. J.K. Tsui, M. Bhatt, S. Calne, and D.B. Calne Botulinum toxin in the treatment of writer's cramp: a double-blind study Neurology 43 1993 183 185
65. J.K. Tsui, A. Eisen, A.J. Stoessl, S. Calne, and D.B. Calne Double-blind study of botulinum toxin in spasmodic torticollis Lancet 2 1986 245 247
66. J. Volkmann, J. Mueller, G. Deuschl, A.A. Kuhn, J.K. Krauss, W. Poewe, L. Timmermann, D. Falk, A. Kupsch, A. Kivi, G.H. Schneider, A. Schnitzler, M. Sudmeyer, J. Voges, A. Wolters, M. Wittstock, J.U. Muller, S. Hering, W. Eisner, J. Vesper, T. Prokop, M. Pinsker, C. Schrader, M. Kloss, K. Kiening, K. Boetzel, J. Mehrkens, I.M. Skogseid, J. Ramm-Pettersen, G. Kemmler, K.P. Bhatia, J.L. Vitek, and R. Benecke Pallidal neurostimulation in patients with medication-refractory cervical dystonia: a randomised, sham-controlled trial Lancet Neurol. 13 2014 875 884
67. B. Wabbels, G. Reichel, A. Fulford-Smith, N. Wright, and P. Roggenkamper Double-blind, randomised, parallel group pilot study comparing two botulinum toxin type A products for the treatment of blepharospasm J. Neural Transm. 118 2011 233 239
68. P. Warrick, C. Dromey, J.C. Irish, L. Durkin, A. Pakiam, and A. Lang Botulinum toxin for essential tremor of the voice with multiple anatomical sites of tremor: a crossover design study of unilateral versus bilateral injection Laryngoscope 110 8 2000 Aug 1366 1374
69. J. Wissel, C. Kabus, R. Wenzel, S. Klepsch, U. Schwarz, A. Nebe, L. Schelosky, U. Scholz, and W. Poewe Botulinum toxin in writer's cramp: objective response evaluation in 31 patients J. Neurol. Neurosurg. Psychiatry 61 1996 172 175
70. D.L. Wong, S.G. Adams, J.C. Irish, L.C. Durkin, E.J. Hunt, and M.P. Charlton Effect of neuromuscular activity on the response to botulinum toxin injections in spasmodic dysphonia J. Otolaryngol. 24 1995 209 216
71. D.M. Yoshimura, M.J. Aminoff, and R.K. Olney Botulinum toxin therapy for limb dystonias Neurology 42 1992 627 630