Lokalizovaná sklerodermie – morfea:
současný stav a možnosti léčby

Česká verzia

Authors: J. Štork
Authors place of work: Dermatovenerologická klinika 1. LF UK a VFN, Praha přednosta prof. MUDr. Jiří Štork, CSc.
Published in the journal: Čes-slov Derm, 91, 2016, No. 6, p. 258-271
Category: Reviews (Continuing Medical Education)

Summary

Localized scleroderma (LS) or morphea are terms encompassing a group of rare chronic inflammatory fibrosing disorders of the skin and underlying structures such as subcutaneous fat tissue, fascia, muscle and bone. As, in contrast to systemic sclerosis, internal organs like GIT, lungs, heart and kidney are not affected and the disease has normal life expectancy, some classifications use term morphea for all subtypes in order to avoid unnecessary confusion with systemic form, other use term LS. The subtypes of the disease include plaque, generalized, linear, deep and mixed variants, some classifications comprise also eosinophilic fasciitis and atrophoderma of Pasini-Pierini. The treatment should start early in the course of the disease before complications occur as these are difficult to treat. The main indication criteria for the treatment are based on disease activity, subtype of the disease and depth of involvement. Current treatment options for superficial forms include phototherapy and topical therapy with corticosteroids, tacrolimus, calcipotriol, alone or in combination with bethamethason dipropionate, some studies suggestt the use of imiquimod. The well- established systemic therapy for severe forms include systemic methotrexate alone or in combination with systemic corticosteroids, in treatment resistant cases the use of mycophenolate mofetil is promissing. It seems that prolonged systemic therapy with methotrexate for 1–2 years reduce relapses of the disease that are observed in one quarter of patients.

Key words:
localized scleroderma – morphea – classifications – therapy – course

Zdroje

1. ARKACHAISRI, T., VILAIYUK, S., LI, S., O’NEIL, K. M. et al. The localized scleroderma skin severity index and physician global assessment of disease activity: a work in progress toward development of localized scleroderma outcome measures. J. Rheumatol., 2009, 36, p. 2819–2829.

2. ARKACHAISRI, T., VILAIYUK, S., TOROK, K. S., MEDSGER, T. A. jr. Development and initial validation of the localized scleroderma skin damage index and physician global assessment of disease damage: a proof-of-concept study. Rheumatology (Oxford), 2010, 49, p. 373–381.

3. BLEASEL, N. R., STAPLETON, K. M., COMMENS, C., AHERN, V. A. Radiation-induced localized scleroderma in breast cancer patients. Australas J. Dermatol., 1999, 40, p. 99–102.

4. COX, D., O’ REGAN, G., COLLINS, S. et al. Juvenile localised scleroderma: a retrospective review of response to systemic treatment. Ir. J. Med. Sci., 2008, 177, p. 343–346.

5. CUNNINGHAM, B. B., LANDELLS, I. D., LANGMAN, C. et al. Topical calcipotriene for morphea/linear scleroderma. J. Am. Acad. Dermatol., 1998, 39, p. 211–215.

6. DYTOC, M., TING, P. T., MAN, J. et al. First case series on the use of imiquimod for morphoea. Br. J. Dermatol., 2005, 153, p. 815–820.

7. DYTOC, M., KOSINTSEVA, I, TING, P. T. First case series on the use of calcipotriol-betamethasone dipropionate for morphoea. Br. J. Dermatol., 2007, 157, p. 615–618.

8. DYTOC, M., WAT, H., CHEUNG-LEE, M. et al. Evaluation of topical imiquimod 5% for plaque-type morphea: a multicenter, prospective, vehicle-controlled trial. J. Cutan. Med. Sur., 2015, 19, p. 132–139.

9. EL-MOFTY, M., MOSTAFA, W., EL-DAROUTY, M. et al. Diffferent low doses of broad-band UVA in the treatment of morphea and systemic sclerosis. Photodermatol. Photoimmunol. Photomed., 2004, 20, p. 148–156.

10. FETT, N., WERTH, V. P. Update on morphea: part I. Epidemiology, clinical presentation, and pathogenesis. J. Am. Acad. Dermatol., 2011, 64, p. 217–228.

11. FETT, N., WERTH, V. P. Update on morphea: part II. Outcome measures and treatment. J. Am. Acad. Dermatol., 2011, 64, p. 231–242.

12. FITCH, P. G., RETTIG, P., BURNHAM, J. M. et al. Treatment of pediatric localized scleroderma with methotrexate. J. Rheumatol., 2006, 33, p. 609–614.

13. GORDON, S., PRATT, E. A., GORCEY, L. V., SOTER, N. A. et al. Phototherapy, photodynamic therapy and photopheresis in the treatment of connective tissues diseases: a review. Brit. J. Dermatol., 2015, 173, p. 19–30.

14. GRABELL, D., HSIEH, C., ANDREW, R. et al. The role of skin trauma in the distribution of morphea lesions: A cross-sectional survey of the morphea in adults and children cohort IV. J. Am. Acad. Dermatol., 2014 71, p. 493–498.

15. HERRICK, A. L., ENNIS, H., BHUSHAN, M. et al. Incidence of childhood linear scleroderma and systemic sclerosis in the UK and Ireland. Arthritis Care Res. (Hoboken), 2010, 62, p. 213–218.

16. IHN, H., SATO, S., FUJIMOTO, M., KIKUCHI, K., TAKEHARA, K. Demonstration of interleukin-2, interleukin-4 and interleukin-6 in sera from patients with localized scleroderma. Arch. Dermatol. Res., 1995, 287, p. 193–197.

17. JOHNSON, W., JACOBE, H. Morphea in adults and children cohort II: Patients with morphea experience delay in diagnosis and large variation in treatment. J. Am. Acad. Dermatol., 2012, 67, 5, p. 881–889.

18. JOLY, P., BAMBERGER, N., CRICKX, B. et al. Treatment of severe forms of localized scleroderma with oral corticosteroids: follow-up study on 17 patients. Arch. Dermatol., 1994, 130, 5, p. 663–664.

19. KELSEY, C. E., TOROK, K. S. The Localized Scleroderma Cutaneous Assessment Tool: responsiveness to change in a pediatric clinical population. J. Am. Acad. Dermatol., 2013, 69, p. 214–220.

20. KREUTER, A., GAMBICHLER, T., AVERMAETE, A. et al. Combined treatment with calcipotriol ointment and low-dose ultraviolet A1 phototherapy in childhood morphea. Pediatr. Dermatol., 2001, 18, p. 241–245.

21. KREUTER, A., GAMBICHLER, T., BREUCKMANN, F. et al. Pulsed high-dose corticosteroids combined with low-dose methotrexate in severe localized scleroderma. Arch. Dermatol., 2005, 141, p. 847–852.

22. KREUTER, A., HYUN, J., STÜCKER, M. et al. A randomized controlled study of low-dose UVA1, medium-dose UVA1, and narrowband UVB phototherapy in the treatment of localized scleroderma. J. Am. Acad. Dermatol., 2006, 54, 3, p. 440–447.

23. KREUTER, A., HYUN, J., SKRYGAN, M. et al. Ultraviolet A1-induced downregulation of human beta-defensins and interleukin-6 and interleukin-8 correlates with clinical improvement in localized scleroderma. Br. J. Dermatol., 2006, 155, p. 600–607.

24. KREUTER, A. Localized scleroderma. Dermatol. Therapy, 2012, 25, p. 135–147.

25. KREUTER, A., KRIEG, T., WORM, M. et al. German guidelines for the diagnosis and therapy of circumscribed scleroderma. J. Dtsch. Dermatol. Ges., 2016, 14, 2, p. 199–216.

26. KROFT, E. B., GROENEVELD, T. J., SEYGER, M. M. B. et al. Efficacy of topical tacrolimus 0.1% in active plaque morphea. Randomized, double-blind, emollient-controlled pilot study. Am. J. Clin. Dermatol., 2009, 10, p. 181.

27. KROFT, E. B., CREEMERS, M. C., VANDEN HOOGEN, F. H. et al. Effectiveness, side-effects and period of remission after treatment with methotrexate in localized scleroderma and related sclerotic skin diseases: an inception cohort study. Br. J. Dermatol., 2009, 160, p. 1075–1082.

28. LAXER, R. M., ZULIAN, F. Localized scleroderma. Curr. Opin. Rheumatol., 2006, 18, p. 606–613.

29. LEASK, A., ABRAHAM, D. J. TGF-beta signaling and the fibrotic response. FASEB J, 2004, 18, p. 816–827.

30. LEITENBERGER, J. J., CAYCE, R. L., HALEY, R. W., ADAMS-HUET, B., BERGSTRESSER, P. R., JACOBE, H. T. Distinct autoimmune syndromes in morphea: a review of 245 adult and pediatric cases. Arch. Dermatol., 2009, 145, 5, p. 545.

31. LI, S. C., TOROK, K, S., POPE, E. et al. Development of consensus treatment plans for juvenile localized scleroderma: a roadmap toward comparative effectiveness studies in juvenile localized scleroderma. Arthritis Care Res (Hoboken) 2012, 64, p. 1175–1185.

32. MANCUSO, G., BERDONDINI, R, M. Localized scleroderma: response to occlusive treatment with tacrolimus ointment. Br. J. Dermatol., 2005, 152, p. 180–182.

33. MARSOL, I. B. Update on the classification and treatment of localized scleroderma. Acta Dermatosilifiliogr., 2013, 104, 8, p. 654–666.

34. MARTINI, G., RAMANAN, A. V., FALCINI, F. et al. Successful treatment of severe or methotrexate-resistant juvenile localized scleroderma with mycophenolate mofetil. Rheumatology (Oxford), 2009, 48, p. 1410–1413.

35. MERTENS, J. S., SEYGER, M. M. B., KIEVIT, W. et al. Disease recurrence in localized scleroderma: a retrospective analysis of 344 patients with paediatric- or adult-onset disease. Br. J. Dermatol., 2015, 172, p. 722–778.

36. MERTENS, J. S., MARSMAN, D. S., VAN DE KERKHOF, P. C. M. et al. Use of mycophenolate mofetil in patients with severe localized scleroderma resistant or intolerant to methotrexate. Acta Dermato-Venereologica, 2016, 96, p. 510–513.

37. MURRAY, K. J., LAXER, R. M. Scleroderma in children and adolescents. Rheum. Dis. Clin. N. Am., 2002, 28, 3, p. 603–624.

38. NOURI, S., JACOBE, H. Recent developments in diagnosis and assessment of morphea. Curr. Rheumatol. Rep., 2013, 15, p. 308–315.

39. PAVLOTSKY, F., SAKKA, N., LOZINSKI, A. et al. Bath psoralen-UVA photochemotherapy for localized scleroderma: experience from a single center. Photodermatol. Photoimmunol. Photomed., 2013, 29, p. 247–252.

40. PETERSON, L. S., NELSON, A. M., SU, W. P. et al. The epidemiology of morphea (localized scleroderma) in Olmsted County 1960–1993. J. Rheumatol., 1997, 24, 1, p. 73–80.

41. Pope, E., Doria, A.S., Theriault, M., et al. Topical imiquimod 5% cream for mediatric plaque morphea. Dermatology, 2011, 223, p. 363-369.

42. RICE, M., PADILLA, C. M., MCLAUGHLIN, S. R. et al. Fresolimumab treatment decreases biomarkers and improves clinical symptoms in systemic sclerosis patients. J. Clin. Invest., 2015, 125, 7, p. 2795–2807.

43. SEYGER, M. M., VANDEN HOOGEN, F. H., DEBOO, T., DEJONG, E. M. Low-dose methotrexate in the treatment of widespread morphea. J. Am. Acad. Dermatol., 1998, 39, p. 220–225.

44. SILMAN, A., JANNINI, S., SYMMONS, D., BACON, P. An epidemiological study of scleroderma in the West Midlands. Br. J. Rheumatol., 1988, 27, p. 286–290.

45. SPALEK, M., JONSKA-GMYREK, J., GAŁECKI, J. Radiation-induced morphea – a literature review. J. Eur. Acad. Dermatol. Venereol., 2015, 29, p. 197–202.

46. STEFANAKI, CH., STEFANAKI, K., KONTOCHRISTOPOULOS, G. et al. Topical tacrolimus 0.1% ointment in the treatment of localized scleroderma. J. Derm., 2008, 35, p. 712–718.

47. TOLKACHJOV, S. N., PATEL, N. G., TOLLEFSON, M. M. Progressive hemifacial atrophy: a review. Orphanet Journal of Rare Diseases, 2015, DOI 10.1186/s13023-015-0250-9.

48. TOLLEFSON, M. M., WITMAN, P. M. En coup de sabre morphea and Parry-Romberg syndrome: a retrospective review of 54 patients. J. Am. Acad. Dermatol., 2007, 56, p. 257–263.

49. TOROK, K. S., ARAKACHAISRI, T. Methotrexate and corticosteroids in the treatment of localized scleroderma: a standardized prospective longitudinal single center study. J. Rheumatol., 2012, 39, 2, p. 286–294.

50. TOROK, K. S., KURZINSKI, K., KELSEY, H. C. et al. Peripheral blood cytokine and chemokine profiles in juvenile localized scleroderma: T-helper cell-associated cytokine profiles. Sem. Arthritis Rheum., 2015, 45, p. 284–293.

51. UZIEL, Y., KRAFCHIK, B. R., SILVERMAN, E. D. et al. Localized scleroderma in childhood: a report of 30 cases. Semin. Arthritis Rheum., 1994, 23, p. 328–340.

52. UZIEL, Y., FELDMAN, B., KRAFCHIK, B. et al. Methotrexate and corticosteroid therapy for pediatric localized scleroderma. J. Pediatr., 2000, 136, p. 91–95.

53. WEIBEL L., SAMPAIO, M. C., VISENTIN, M. T. et al. Evaluation of methotrexate and corticosteroids for the treatment of localized scleroderma (morphoea) in children. Br. J. Dermatol., 2006, 155, 1013–1120.

54. WU, E. Y., LI, S. C., TOROK, K. S. et al. A28: Description of the Juvenile Localized Scleroderma Subgroup of the CARRA Registry. Arthritis Rheumatol., 2014, 66, S3, p. S43–S44.

55. ZANNIN, M. E., MARTINI, G., ATHREYA, B. H. et al. Ocular involvement in children with localised scleroderma: a multi-centre study. Br. J. Ophthalmol., 2007, 91, p. 1311–1314.

56. ZULIAN, F., ATHREYA, B. H., LAXER, R. et al. Juvenile localized scleroderma: clinical and epidemiological features in 750 children. An international study. Rheumatology (Oxford), 2006, 45, p. 614–620.

57. ZULIAN, F., MARTINI, G., VALLONGO, C. et al. Methotrexate treatment in juvenile localized scleroderma: a randomized, double-blind, placebo-controlled trial. Arthritis Rheum., 2011, 63, p. 1998–2006.

58. ZULIAN, F., VALLONGO, C., PATRIZI, A. et al. A long-term follow-up study of methotrexate in juvenile localized scleroderma (morphea). J. Am. Acad. Dermatol., 2012, 67, p. 1151–1156.