Epidermolysis Bullosa: Innovative Treatment with Stem Cells, a Systematic Review
DOI:
https://doi.org/10.56294/saludcyt20241193Keywords:
Treatment, Stem Cells, Regenerative Therapy, Innovation, Skin, Genetic DiseaseAbstract
Introduction: epidermolysis bullosa is a set of genetic disorders that originate from modifications in certain skin proteins that alter the union and structure of the dermis and epidermis. Clinically, it manifests itself through blisters. However, these lesions can spread to other areas. areas of the body, thus causing the appearance of chronic wounds. The conventional treatment of Epidermolysis Bullosa has not been shown to improve the long-term quality of life of those who suffer from it, which is why researchers propose new therapies that allow improvements in the quality of life of patients, being one of the most promising, the use of stem cells.
Methods: randomized clinical trials were selected that evaluated the efficacy and safety of stem cell treatment in patients with Epidermolysis bullosa compared to usual therapy and its cost-effectiveness.
Results: based on the selected articles, several variables were measured. Four articles demonstrated the effectiveness of stem cell treatment compared to conventional treatment; two articles measured the economic considerations and access to this new treatment; four articles compared the benefits and disadvantages of using stem cells in patients with Epidermolysis bullosa; and finally, five articles demonstrated the impact that stem cells have in improving the quality of life of patients with Epidermolysis bullosa who undergo this type of therapy.
Conclusions: at a global level, the effectiveness of the use of stem cells opens a promising research perspective that seeks to benefit the most vulnerable population affected by this disease, since it plays a key role in the significant reduction of blistering lesions, this represents a positive advance. in the management of the disease, as well as the improvement in its biological, psychological and social sphere, allowing an early introduction to daily activities, generating significant savings of resources for caregivers and for the State. Because, in Latin America, to date there is little research in this area, it should be considered a very promising line of research in the field of Dermatology. Similar situation in the case of Ecuador, where since there are no statistics on patients with this disease, nor data that contribute to the treatment of this vulnerable group, these investigations will provide a solid basis for researchers to adopt this new therapy
References
1. Isis Elianis Rubio Rojas D, Mariana López Moro D. REVISION BIBLIOGRAFICA TITULO: LAS ENFERMEDADES RARAS. EPIDERMÓLISIS BULLOSA [Internet]. 2023. Available from: https://genmed.sld.cu/index.php/gemed23/2023/paper/view/36
2. Hon KL, Chu S, Leung AKC. Epidermolysis Bullosa: Pediatric Perspectives. Curr Pediatr Rev [Internet]. 2022 Jun 14 [cited 2024 Mar 22];18(3):182–190. Available from: https://pubmed.ncbi.nlm.nih.gov/34036913/
3. Amanda Vázquez Núñez M, Eloy Santiesteban Alejo R, Inés Ferrer Mora Y. Epidermólisis ampollosa o bullosa congénita. Actualización clínica Congenital Bullous Epidermolysis. Clinical Update. 2021 [cited 2023 Dec 27]; Available from: http://scielo.sld.cu/scielo.php?script=sci_arttext&pid=S2221-24342021000100074
4. Hou PC, Wang HT, Abhee S, Tu WT, McGrath JA, Hsu CK. Investigational Treatments for Epidermolysis Bullosa. Am J Clin Dermatol [Internet]. 2021 Nov 22 [cited 2024 Mar 22];22(6):801–817. Available from: https://pubmed.ncbi.nlm.nih.gov/34292508/
5. Bardhan A, Bruckner-Tuderman L, Chapple ILC, Fine JD, Harper N, Has C, Magin TM, Marinkovich MP, Marshall JF, McGrath JA, Mellerio JE, Polson R, Heagerty AH. Epidermolysis bullosa. Nat Rev Dis Primers [Internet]. 2020 Sep 24 [cited 2024 Mar 22];6(1):78. Available from: https://pubmed.ncbi.nlm.nih.gov/32973163/
6. De la Rosa J, Zamora R, Vásquez G, López A. Epidermólisis ampollosa, reporte de un caso. Redalyc [Internet]. 2021 Jul 5 [cited 2024 Mar 22];17. Available from: https://www.redalyc.org/articulo.oa?id=638268499021
7. Tabor A, LeQuang JAK, Pergolizzi J. Epidermolysis Bullosa: Practical Clinical Tips From the Field. Cureus [Internet]. 2024 Feb 7 [cited 2024 Mar 22]; Available from: https://pubmed.ncbi.nlm.nih.gov/38465068/
8. Maldonado G, Durán C, Orozco L, Palacios C, Del Mar M, García M. Epidermólisis ampollosa: nuevos conceptos clínicos y moleculares para clasificación y diagnóstico. Artículo de revisión. Dermatología Cosmética, Médica y Quirúrgica [Internet]. 2016 [cited 2024 Mar 22];14. Available from: https://www.medigraphic.com/pdfs/cosmetica/dcm-2016/dcm164f.pdf
9. Ministerio de Salud Pública del Ecuador. Por primera vez en Ecuador contamos con un registro único de personas que viven con enfermedades raras [Internet]. Por primera vez en Ecuador contamos con un registro único de personas que viven con enfermedades raras. 2022 [cited 2024 Mar 22]. Available from: https://www.salud.gob.ec/por-primera-vez-en-ecuador-contamos-con-un-registro-unico-de-personas-que-viven-con-enfermedades-raras/
10. Diario Salud. UNAS 106 ENFERMEDADES RARAS SON RECONOCIDAS EN ECUADOR [Internet]. 2022 [cited 2024 Mar 22]. Available from: https://diariosalud.com.ec/2022/02/28/unas-106-enfermedades-raras-son-reconocidas-en-ecuador/
11. Bullosa E. Best Practice Guidelines Skin and wound care in [Internet]. 2017. Available from: www.woundsinternational.com
12. Martin K, Geuens S, Asche JK, Bodan R, Browne F, Downe A, García García N, Jaega G, Kennedy B, Mauritz PJ, Pérez F, Soon K, Zmazek V, Mayre-Chilton KM. Psychosocial recommendations for the care of children and adults with epidermolysis bullosa and their family: Evidence based guidelines. Orphanet Journal of Rare Diseases. BioMed Central Ltd.; 2019. PMID: 31186066
13. Felipe J, Paramo A, Gómez N, Codirector S. DISPOSITIVO MÉDICO PREVENTIVO DE LESIONES PARA PERSONAS CON EPIDERMÓLISIS AMPOLLOSA MODALIDAD: Exploratorio AUTOR. 2021.
14. Biasutto M, Martínez MF, Angles MV, Mazzuoccolo LD. Nuevos tratamientos para el manejo de la epidermólisis ampollar. Dermatología Argentina. 2023 Apr 21;29(1):02–08.
15. Facio-Olvera OE. Procuración de injertos estándar frente a extendidos. Experiencia en el Hospital de Especialidades La Raza. Revista Médica del Instituto Mexicano del Seguro Social. Publicidad Permanyer, SLU; 2021 Mar 23;58(5).
16. José M, Toledano E. Empleo de células madre mesenquimales para el tratamiento de la EB [Internet]. 2018. Available from: http://www.unicolmayor.edu.co/invest_nova/NOVA/
17. De Rosa L, Carulli S, Cocchiarella F, Quaglino D, Enzo E, Franchini E, Giannetti A, De Santis G, Recchia A, Pellegrini G, De Luca M. Long-term stability and safety of transgenic cultured epidermal stem cells in gene therapy of junctional epidermolysis bullosa. Stem Cell Reports. 2014 Jan 14;2(1):1–8. PMID: 24511464
18. Paz y Miño C. Terapia gratuita con Células Madre. Quito; 2012 Mar [cited 2024 Mar 22]; Available from: https://www.udla.edu.ec/2012/03/terapia-gratuita-con-celulas-madre/
19. Niti A, Koliakos G, Michopoulou A. Stem Cell Therapies for Epidermolysis Bullosa Treatment. Bioengineering. MDPI; 2023.
20. Lwin SM, Syed F, Di WL, Kadiyirire T, Liu L, Guy A, Petrova A, Abdul-Wahab A, Reid F, Phillips R, Elstad M, Georgiadis C, Aristodemou S, Lovell PA, McMillan JR, Mee J, Miskinyte S, Titeux M, Ozoemena L, Pramanik R, Serrano S, Rowles R, Maurin C, Orrin E, Martinez-Queipo M, Rashidghamat E, Tziotzios C, Onoufriadis A, Chen M, Chan L, Farzaneh F, Rio M Del, Tolar J, Bauer JW, Larcher F, Antoniou MN, Hovnanian A, Thrasher AJ, Mellerio JE, Qasim W, McGrath JA. Safety and early efficacy outcomes for lentiviral fibroblast gene therapy in recessive dystrophic epidermolysis bullosa. JCI Insight. American Society for Clinical Investigation; 2019 Jun 6;4(11). PMID: 31167965
21. Rashidghamat E, Kadiyirire T, Ayis S, Petrof G, Liu L, Pullabhatla V, Ainali C, Guy A, Aristodemou S, McMillan JR, Ozoemena L, Mee J, Pramanik R, Saxena A, Nuamah R, de Rinaldis E, Serrano S, Maurin C, Martinez-Queipo M, Lwin SM, Ilic D, Martinez A, Dazzi F, Slaper-Cortenbach I, Westinga K, Zeddies S, van den Broek M, Onoufriadis A, Mellerio JE, McGrath JA. Phase I/II open-label trial of intravenous allogeneic mesenchymal stromal cell therapy in adults with recessive dystrophic epidermolysis bullosa. J Am Acad Dermatol. Mosby Inc.; 2020 Aug 1;83(2):447–454. PMID: 31786163
22. Picón-Jaimes YA, López-Medina IL, Carmona-Gómez JK, Filorio-Campos YP. Epidermólisis bullosa distrófica recesiva: avances e impacto del desarrollo de terapias génicas y alogénicas. 2022
23. Debbe Esquivel A, Soto Cordero C. Terapia celular como tratamiento para mejorar la curación de heridas en pacientes con epidermólisis ampollosa distrófica recesiva. Revista de la Facultad de Medicina de la Universidad de Iberoamérica. Universidad de Iberoamérica; 2023 Jan 1;1(1):33–44.
24. Petrof G, Lwin SM, Martínez-Queipo M, Abdul-Wahab A, Tso S, Mellerio JE, Slaper-Cortenbach I, Boelens JJ, Tolar J, Veys P, Ofuya M, Peacock JL, Martínez AE, McGrath JA. Potential of Systemic Allogeneic Mesenchymal Stromal Cell Therapy for Children with Recessive Dystrophic Epidermolysis Bullosa. Journal of Investigative Dermatology. 2015 Sep;135(9):2319–2321.
25. Hirsch T, Rothoeft T, Teig N, Bauer JW, Pellegrini G, De Rosa L, Scaglione D, Reichelt J, Klausegger A, Kneisz D, Romano O, Seconetti AS, Contin R, Enzo E, Jurman I, Carulli S, Jacobsen F, Luecke T, Lehnhardt M, Fischer M, Kueckelhaus M, Quaglino D, Morgante M, Bicciato S, Bondanza S, De Luca M. Regeneration of the entire human epidermis using transgenic stem cells. Nature. Nature Publishing Group; 2017 Nov 16;551(7680):327–332.
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