Рецепт. 2018; : 848-856
Системный склероз:стандартные подходы и новые горизонты
Аннотация
Системный склероз (СС) - хроническое прогрессирующее заболевание соединительной ткани, основным клиническим проявлением которого является полиорганный фиброз. На сегодняшний день этиология системного склероза точно не установлена. Однако известно, что аутоиммунный патологический процесс при данном заболевании запускают генетические факторы в сочетании с факторами внешней среды. Анализируя современные терапевтические подходы к лечению системного склероза, можно сказать, что, несмотря на существенные достижения, разработку рекомендаций, эффект от применяемых схем лечения при СС остается недостаточным. Предлагаемые новые терапевтические стратегии нуждаются в подтверждении и проведении дальнейших клинических исследований. Перспективным направлением лечения СС на сегодняшний день является изучение эффективности трансплантации стволовых клеток, применения высоких доз глюкокортикоидов при очень ранней форме системного склероза, а также иммуносупрессивных и противовоспалительных свойств синтетических производных каннабиноидов.
Список литературы
1. Kowal-Bielecka O, Fransen J, Avouac J EUSTAR Coauthors, et al. (2017) Update of EULAR recommendations for the treatment of systemic sclerosis. Annals of the Rheumatic Diseases, 76, pp. 1327-1339.
2. Roustit M., Blaise S., Allanore Y., et al. (2013) Phosphodiesterase-5 inhibitors for the treatment of secondary Raynaud’s phenomenon: systematic review and meta-analysis of randomized trials. Ann Rheum Dis, 72 (10), pp. 1696-9.
3. Tashkin D.P., Elashoff R., Clements P.J., et al. (2007) Effects of 1-year treatment with cyclophosphamide on outcomes at 2 years in scleroderma lung disease. Am J Respir Crit Care Med, 176 (10), pp. 1026-34.
4. Sullivan K.M., Goldmuntz E.A., Keyes-Elstein L., McSweeney P.A., Pinckney A., Welch B., et al. (2018) Myeloablative autologous stem cell transplantation for severe scleroderma. N Engl J Med., 378, pp. 35-47. doi: 10.1056/NEJMoa1703327
5. Burt R.K., Shah S.J., Dill K., Grant T., Gheorghiade M., Schroeder J., et al. (2011) Autologous non- myeloablative haemopoietic stem cell transplantation compared with pulse cyclophosphamide once per month for systemic sclerosis (ASSIST): an open-label, randomized phase 2 trial. The Lancet, 378, pp. 498-506. doi: 10.1016/S0140-6736(11)60982-3
6. Van Laar J.M., Farge D., Sont J.K., Naraghi K., Marjanovic Z., Larghero J., et al. (2014) Autologous hematopoietic stem cell transplantation versus intravenous pulse cyclophosphamide in diffuse cutaneous systemic sclerosis: a randomized clinical trial. JAMA, 311, 2490. doi: 10.1001/ jama.2014.6368
7. Guiducci S., Porta F., Saccardi R., Guidi S., Ibba-Manneschi L., Manetti M., et al. (2010) Autologous mesenchymal stem cells foster revascularization of ischemic limbs in systemic sclerosis: a case report. Ann Intern Med., 153 (10), pp. 650-4. doi: 10.7326/0003-4819-153-10-201011160-00007.
8. Christopeit M., Schendel M., Föll J., Müller L.P., Keysser G., Behre G. (2008) Marked improvement of severe progressive systemic sclerosis after transplantation of mesenchymal stem cells from an allogeneic haploidentical related donor mediated by ligation of CD137L. Leukemia, 22 (5), pp. 1062-4. doi: 10.1038/sj.leu.2404996.
9. Keyszer G., Christopeit M., Fick S., Schendel M., Taute B.M., Behre G., et al. (2011) Treatment of severe progressive systemic sclerosis with transplantation of mesenchymal stromal cells from allogeneic related donors: report of five cases. Arthritis Rheum., 63 (8), pp. 2540-2. doi: 10.1002/art.30431.
10. Maria A.T., Toupet K., Bony C., Pirot N., Vozenin M., Petit B., Roger P., Batteux F., Le Quellec A., Jorgensen C., Noël D., Guilpain P. (2016) Antifibrotic, Antioxidant, and Immunomodulatory Effects of Mesenchymal Stem Cells in HOCl-Induced Systemic Sclerosis. Arthritis & Rheumatology, 68, pp. 1013-1025. doi: 10.1002/art.39477
11. Verrecchia F., Laboureau J., Verola O., Roos N., Porcher R., Bruneval P., et al. (2007) Skin involvement in scleroderma - where histological and clinical scores meet. Rheumatology (Oxford), 46 (5), pp. 833-41. doi: 10.1093/rheumatology/kel451.
12. Van Laar J.M., Farge D., Tyndall A. (2005) Autologous Stem cell Transplantation International Scleroderma (ASTIS) trial: hope on the horizon for patients with severe systemic sclerosis. Annals of the Rheumatic Diseases, 64 (10), 1515. Available at: http://doi.org/10.1136/ard.2005.043240
13. Peltzer J., Aletti M., Frescaline N., Busson E., Lataillade J-J., Martinaud C. (2018) Mesenchymal Stromal Cells Based Therapy in Systemic Sclerosis: Rational and Challenges. Front. Immunol., 9: 2013. doi: 10.3389/fimmu.2018.02013
14. Dazzi F., van Laar J.M., Cope A., Tyndall A. (2007) Cell therapy for autoimmune diseases. Arthritis Res Ther., 9 (2), 206. doi: 10.1186/ ar2128.
15. Lalu M.M., McIntyre L., Pugliese C., Fergusson D., Winston B.W., Marshall J.C., et al. (2012) Safety of cell therapy with mesenchymal stromal cells (SafeCell): a systematic review and meta-analysis of clinical trials. PLoS One, 7 (10): e47559. doi: 10.1371/journal.pone. 0047559
16. Cras A., Farge D., Carmoi T., Lataillade J.J., Wang D.D., Sun L. (2015) Update on mesenchymal stem cell-based therapy in lupus and scleroderma. Arthritis Res Ther., 17: 301. doi: 10.1186/s13075- 015-0819-7
17. Caplan A.I. (2015) Adult mesenchymal stem cells: when, where, and how. Stem Cells Int., 628767. doi: 10.1155/2015/628767
18. Coleman S.R. (1997) Facial recontouring with lipostructure. Clin Plast Surg., 24 (2), pp. 347-67.
19. Coleman S.R. (2006) Facial augmentation with structural fat grafting. Clin Plast Surg., 33 (4), pp. 567-77.
20. Magalon G., Daumas A., Sautereau N., Magalon J., Sabatier F., Granel B. (2015) Regenerative approach to scleroderma with fat grafting. Clin Plast Surg., 42 (3), pp. 353-64. viii-ix.
21. Konno M., Hamabe A., Hasegawa S., Ogawa H., FukusumiT., Nishikawa S., Ohta K., KanoY., Ozaki M., Noguchi Y., Sakai D., Kudoh T., Kawamoto K., Eguchi H., Satoh T., Tanemura M., Nagano H., Doki Y., Mori M., Ishii H. (2013) Adipose-derived mesenchymal stem cells and regenerative medicine. Dev Growth Differ., 55 (3), pp. 309-18.
22. Bura A., Planat-Benard V., Bourin P., Silvestre J.S., Gross F., Grolleau J.L., SaintLebese B., Peyrafitte J.A., Fleury S., Gadelorge M., Taurand M., Dupuis-Coronas S., Leobon B., Casteilla L. (2014) Phase I trial: the use of autologous cultured adipose derived stroma/stem cells to treat patients with nonrevascularizable critical limb ischemia. Cytotherapy, 16 (2), pp. 245-57.
23. Dimarino A.M., Caplan A.I., Bonfield T.L. (2013) Mesenchymal stem cells in tissue repair. Front Immunol., 4: 201.
24. Bianco P. (2011) Back to the future: moving beyond “mesenchymal stem cells”. J Cell Biochem.,
25. (7), pp. 1713-21.
26. Nakagami H., Morishita R., Maeda K., Kikuchi Y., Ogihara T., Kaneda Y. (2006) Adipose tissue- derived stromal cells as a novel option for regenerative cell therapy. J Atheroscler Thromb., 13 (2), pp. 77-81.
27. Virzì F., Bianca P., Giammona A., et al. (2017) Combined platelet-rich plasma and lipofilling treatment provides great improvement in facial skin-induced lesion regeneration for scleroderma patients. Stem Cell Research & Therapy, 8: 236. doi: 10.1186/s13287-017-0690-3.
28. Driver V.R., Hanft J., Fylling C.P., Beriou J.M. G. (2006) Autologel Diabetic Foot Ulcer Study. A prospective, randomized, controlled trial of autologous platelet-rich plasma gel for the treatment of diabetic foot ulcers. Ostomy Wound Manage, 52 (6), pp. 68-70. 72, 74 passim.
29. Giuggioli D., Colaci M., Manfredi A., Mariano M., Ferri C. (2012) Platelet gel in the treatment of severe scleroderma skin ulcers. Rheumatol Int., 32 (9), pp. 2929-32.
30. Gentile P., Orlandi A., Scioli M.G., Di Pasquali C., Bocchini I., Curcio C.B., Floris M., Fiaschetti V., Floris R., Cervell V. (2012) A comparative translational study: the combined use of enhanced stromal vascular fraction and platelet-rich plasma improves fat grafting maintenance in breast reconstruction. Stem Cells Transl Med., 1 (4), pp. 341-51.
31. Rubio-Azpeitia E., Andia I. (2014) Partnership between platelet-rich plasma and mesenchymal stem cells: in vitro experience. Muscles Ligaments Tendons J., 4 (1), pp. 52-62.
32. Tashkin D.P., Roth M.D., Clements P.J., et al. (2016) Mycophenolate Mofetil versus Oral Cyclophosphamide in Scleroderma-related Interstitial Lung Disease: Scleroderma Lung Study II (SLS-II), a double-blind, parallel group, randomized controlled trial. The Lancet Respiratory medicine, 4 (9), pp. 708-719. doi: 10.1016/S2213-2600(16)30152-7.
33. Volkmann E.R., Tashkin D.P. (2016) Treatment of Systemic Sclerosis-related Interstitial Lung Disease: A Review of Existing and Emerging Therapies. Annals of the American Thoracic Society, 13 (11), pp. 2045-2056. doi: 10.1513/AnnalsATS.201606-426FR.
34. Ueda T. et al. (2017) Mycophenolate mofetil as a therapeutic agent for interstitial lung diseases in systemic sclerosis. Respiratory Investigation. Available at: https://doi.org/10.1016/j. resinv.2017.11.004
35. Burstein S.H. (2018) Ajulemic acid: potential treatment for chronic inflammation. Pharmacol Res Perspect., e00394. Available at: https://doi.org/10.1002/prp2.394
36. Chmiel J.F., Elborn J.S., Constantine S., White B. (2017) A Phase 2 study of the safety, pharmacokinetics, and efficacy of anabasum (JBT-101) in cystic fibrosis (CF). J Cyst Fibros., 16S1: S01.05.
37. Stebulis J.A., Johnson D.R., Rossetti R.G., Burstein S.H., Zurier R.B. (2008) Ajulemic acid, a synthetic cannabinoid acid, induces an anti-inflammatory profile of eicosanoids in human synovial cells. Life Sci., 83, pp. 666-670.
38. Van den Hombergh W.M.T., Kersten B.E., Knaapen-Hans H.K.A., et al. (2018) Hit hard and early: analyzing the effects of high-dose methylprednisolone on nailfold capillary changes and biomarkers in very early systemic sclerosis: study protocol for a 12-week randomized controlled trial. Trials, 19: 449. doi: 10.1186/s13063-018-2798-x.
Recipe. 2018; : 848-856
Systemic sclerosis: standard approaches and new horizons
Abstract
References
1. Kowal-Bielecka O, Fransen J, Avouac J EUSTAR Coauthors, et al. (2017) Update of EULAR recommendations for the treatment of systemic sclerosis. Annals of the Rheumatic Diseases, 76, pp. 1327-1339.
2. Roustit M., Blaise S., Allanore Y., et al. (2013) Phosphodiesterase-5 inhibitors for the treatment of secondary Raynaud’s phenomenon: systematic review and meta-analysis of randomized trials. Ann Rheum Dis, 72 (10), pp. 1696-9.
3. Tashkin D.P., Elashoff R., Clements P.J., et al. (2007) Effects of 1-year treatment with cyclophosphamide on outcomes at 2 years in scleroderma lung disease. Am J Respir Crit Care Med, 176 (10), pp. 1026-34.
4. Sullivan K.M., Goldmuntz E.A., Keyes-Elstein L., McSweeney P.A., Pinckney A., Welch B., et al. (2018) Myeloablative autologous stem cell transplantation for severe scleroderma. N Engl J Med., 378, pp. 35-47. doi: 10.1056/NEJMoa1703327
5. Burt R.K., Shah S.J., Dill K., Grant T., Gheorghiade M., Schroeder J., et al. (2011) Autologous non- myeloablative haemopoietic stem cell transplantation compared with pulse cyclophosphamide once per month for systemic sclerosis (ASSIST): an open-label, randomized phase 2 trial. The Lancet, 378, pp. 498-506. doi: 10.1016/S0140-6736(11)60982-3
6. Van Laar J.M., Farge D., Sont J.K., Naraghi K., Marjanovic Z., Larghero J., et al. (2014) Autologous hematopoietic stem cell transplantation versus intravenous pulse cyclophosphamide in diffuse cutaneous systemic sclerosis: a randomized clinical trial. JAMA, 311, 2490. doi: 10.1001/ jama.2014.6368
7. Guiducci S., Porta F., Saccardi R., Guidi S., Ibba-Manneschi L., Manetti M., et al. (2010) Autologous mesenchymal stem cells foster revascularization of ischemic limbs in systemic sclerosis: a case report. Ann Intern Med., 153 (10), pp. 650-4. doi: 10.7326/0003-4819-153-10-201011160-00007.
8. Christopeit M., Schendel M., Föll J., Müller L.P., Keysser G., Behre G. (2008) Marked improvement of severe progressive systemic sclerosis after transplantation of mesenchymal stem cells from an allogeneic haploidentical related donor mediated by ligation of CD137L. Leukemia, 22 (5), pp. 1062-4. doi: 10.1038/sj.leu.2404996.
9. Keyszer G., Christopeit M., Fick S., Schendel M., Taute B.M., Behre G., et al. (2011) Treatment of severe progressive systemic sclerosis with transplantation of mesenchymal stromal cells from allogeneic related donors: report of five cases. Arthritis Rheum., 63 (8), pp. 2540-2. doi: 10.1002/art.30431.
10. Maria A.T., Toupet K., Bony C., Pirot N., Vozenin M., Petit B., Roger P., Batteux F., Le Quellec A., Jorgensen C., Noël D., Guilpain P. (2016) Antifibrotic, Antioxidant, and Immunomodulatory Effects of Mesenchymal Stem Cells in HOCl-Induced Systemic Sclerosis. Arthritis & Rheumatology, 68, pp. 1013-1025. doi: 10.1002/art.39477
11. Verrecchia F., Laboureau J., Verola O., Roos N., Porcher R., Bruneval P., et al. (2007) Skin involvement in scleroderma - where histological and clinical scores meet. Rheumatology (Oxford), 46 (5), pp. 833-41. doi: 10.1093/rheumatology/kel451.
12. Van Laar J.M., Farge D., Tyndall A. (2005) Autologous Stem cell Transplantation International Scleroderma (ASTIS) trial: hope on the horizon for patients with severe systemic sclerosis. Annals of the Rheumatic Diseases, 64 (10), 1515. Available at: http://doi.org/10.1136/ard.2005.043240
13. Peltzer J., Aletti M., Frescaline N., Busson E., Lataillade J-J., Martinaud C. (2018) Mesenchymal Stromal Cells Based Therapy in Systemic Sclerosis: Rational and Challenges. Front. Immunol., 9: 2013. doi: 10.3389/fimmu.2018.02013
14. Dazzi F., van Laar J.M., Cope A., Tyndall A. (2007) Cell therapy for autoimmune diseases. Arthritis Res Ther., 9 (2), 206. doi: 10.1186/ ar2128.
15. Lalu M.M., McIntyre L., Pugliese C., Fergusson D., Winston B.W., Marshall J.C., et al. (2012) Safety of cell therapy with mesenchymal stromal cells (SafeCell): a systematic review and meta-analysis of clinical trials. PLoS One, 7 (10): e47559. doi: 10.1371/journal.pone. 0047559
16. Cras A., Farge D., Carmoi T., Lataillade J.J., Wang D.D., Sun L. (2015) Update on mesenchymal stem cell-based therapy in lupus and scleroderma. Arthritis Res Ther., 17: 301. doi: 10.1186/s13075- 015-0819-7
17. Caplan A.I. (2015) Adult mesenchymal stem cells: when, where, and how. Stem Cells Int., 628767. doi: 10.1155/2015/628767
18. Coleman S.R. (1997) Facial recontouring with lipostructure. Clin Plast Surg., 24 (2), pp. 347-67.
19. Coleman S.R. (2006) Facial augmentation with structural fat grafting. Clin Plast Surg., 33 (4), pp. 567-77.
20. Magalon G., Daumas A., Sautereau N., Magalon J., Sabatier F., Granel B. (2015) Regenerative approach to scleroderma with fat grafting. Clin Plast Surg., 42 (3), pp. 353-64. viii-ix.
21. Konno M., Hamabe A., Hasegawa S., Ogawa H., FukusumiT., Nishikawa S., Ohta K., KanoY., Ozaki M., Noguchi Y., Sakai D., Kudoh T., Kawamoto K., Eguchi H., Satoh T., Tanemura M., Nagano H., Doki Y., Mori M., Ishii H. (2013) Adipose-derived mesenchymal stem cells and regenerative medicine. Dev Growth Differ., 55 (3), pp. 309-18.
22. Bura A., Planat-Benard V., Bourin P., Silvestre J.S., Gross F., Grolleau J.L., SaintLebese B., Peyrafitte J.A., Fleury S., Gadelorge M., Taurand M., Dupuis-Coronas S., Leobon B., Casteilla L. (2014) Phase I trial: the use of autologous cultured adipose derived stroma/stem cells to treat patients with nonrevascularizable critical limb ischemia. Cytotherapy, 16 (2), pp. 245-57.
23. Dimarino A.M., Caplan A.I., Bonfield T.L. (2013) Mesenchymal stem cells in tissue repair. Front Immunol., 4: 201.
24. Bianco P. (2011) Back to the future: moving beyond “mesenchymal stem cells”. J Cell Biochem.,
25. (7), pp. 1713-21.
26. Nakagami H., Morishita R., Maeda K., Kikuchi Y., Ogihara T., Kaneda Y. (2006) Adipose tissue- derived stromal cells as a novel option for regenerative cell therapy. J Atheroscler Thromb., 13 (2), pp. 77-81.
27. Virzì F., Bianca P., Giammona A., et al. (2017) Combined platelet-rich plasma and lipofilling treatment provides great improvement in facial skin-induced lesion regeneration for scleroderma patients. Stem Cell Research & Therapy, 8: 236. doi: 10.1186/s13287-017-0690-3.
28. Driver V.R., Hanft J., Fylling C.P., Beriou J.M. G. (2006) Autologel Diabetic Foot Ulcer Study. A prospective, randomized, controlled trial of autologous platelet-rich plasma gel for the treatment of diabetic foot ulcers. Ostomy Wound Manage, 52 (6), pp. 68-70. 72, 74 passim.
29. Giuggioli D., Colaci M., Manfredi A., Mariano M., Ferri C. (2012) Platelet gel in the treatment of severe scleroderma skin ulcers. Rheumatol Int., 32 (9), pp. 2929-32.
30. Gentile P., Orlandi A., Scioli M.G., Di Pasquali C., Bocchini I., Curcio C.B., Floris M., Fiaschetti V., Floris R., Cervell V. (2012) A comparative translational study: the combined use of enhanced stromal vascular fraction and platelet-rich plasma improves fat grafting maintenance in breast reconstruction. Stem Cells Transl Med., 1 (4), pp. 341-51.
31. Rubio-Azpeitia E., Andia I. (2014) Partnership between platelet-rich plasma and mesenchymal stem cells: in vitro experience. Muscles Ligaments Tendons J., 4 (1), pp. 52-62.
32. Tashkin D.P., Roth M.D., Clements P.J., et al. (2016) Mycophenolate Mofetil versus Oral Cyclophosphamide in Scleroderma-related Interstitial Lung Disease: Scleroderma Lung Study II (SLS-II), a double-blind, parallel group, randomized controlled trial. The Lancet Respiratory medicine, 4 (9), pp. 708-719. doi: 10.1016/S2213-2600(16)30152-7.
33. Volkmann E.R., Tashkin D.P. (2016) Treatment of Systemic Sclerosis-related Interstitial Lung Disease: A Review of Existing and Emerging Therapies. Annals of the American Thoracic Society, 13 (11), pp. 2045-2056. doi: 10.1513/AnnalsATS.201606-426FR.
34. Ueda T. et al. (2017) Mycophenolate mofetil as a therapeutic agent for interstitial lung diseases in systemic sclerosis. Respiratory Investigation. Available at: https://doi.org/10.1016/j. resinv.2017.11.004
35. Burstein S.H. (2018) Ajulemic acid: potential treatment for chronic inflammation. Pharmacol Res Perspect., e00394. Available at: https://doi.org/10.1002/prp2.394
36. Chmiel J.F., Elborn J.S., Constantine S., White B. (2017) A Phase 2 study of the safety, pharmacokinetics, and efficacy of anabasum (JBT-101) in cystic fibrosis (CF). J Cyst Fibros., 16S1: S01.05.
37. Stebulis J.A., Johnson D.R., Rossetti R.G., Burstein S.H., Zurier R.B. (2008) Ajulemic acid, a synthetic cannabinoid acid, induces an anti-inflammatory profile of eicosanoids in human synovial cells. Life Sci., 83, pp. 666-670.
38. Van den Hombergh W.M.T., Kersten B.E., Knaapen-Hans H.K.A., et al. (2018) Hit hard and early: analyzing the effects of high-dose methylprednisolone on nailfold capillary changes and biomarkers in very early systemic sclerosis: study protocol for a 12-week randomized controlled trial. Trials, 19: 449. doi: 10.1186/s13063-018-2798-x.
