A review of hematopoietic stem cell transplantation for autoimmune diseases: multiple sclerosis, systemic sclerosis and Crohn's disease. Position paper of the Brazilian Society of Bone Marrow Transplantation

Oliveira, Maria Carolina; Elias, Juliana Bernardes; Moraes, Daniela A.; Simões, Belinda Pinto; Rodrigues, Morgani; Ribeiro, Andreza Alice Feitosa; Piron-Ruiz, Lílian; Ruiz, Milton A.; Hamerschlak, Nelson

doi:10.1016/j.htct.2020.03.002

Cited by 19 publications

(16 citation statements)

References 134 publications

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“…Most patients in our study presented progressive interstitial lung involvement as indication for HSCT. When the whole cohort of patients was analyzed, we observed stabilization of pulmonary function, which agrees with most international studies [2,5,[20][21][22][23][24][25][26][27][28]. However, when the subgroup of patients with progressive interstitial lung disease as indication for HSCT was analyzed separately, we were able to detect significant improvement of FVC and DLCO after HSCT.…”

Section: Discussionsupporting

confidence: 89%

“…A recent meta-analysis shows that autologous HSCT is superior to conventional therapies in SSc patients, especially in the analyses of risk of death from any cause, skin and lung involvement, and quality of life [24]. Recently, the European League Against Rheumatism (EULAR), the American Society for Transplantation and Cell Therapy (ASCTC) and the Brazilian Society of Bone Marrow Transplantation (SBTMO) have recommended autologous HSCT as treatment for patients with progressive SSc at risk of organ failure [3,25,26].…”

Section: Introductionmentioning

confidence: 99%

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Hematopoietic stem cell transplantation for systemic sclerosis: Brazilian experience

et al. 2021

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Background In the past 20 years, hematopoietic stem cell transplantation (HSCT) has been investigated as treatment for systemic sclerosis (SSc). The goal of HSCT is to eradicate the autoreactive immune system, which is replaced by a new immune repertoire with long-lasting regulation and tolerance to autoantigens. Here, we describe the clinical outcomes of severe and refractory SSc patients that underwent HSCT at a single Brazilian center. Patients and methods This is a longitudinal and retrospective study, including 70 adult SSc patients, with an established diagnosis of SSc, and who underwent autologous HSCT from 2009 to 2016. The procedure included harvesting and cryopreservation of autologous hematopoietic progenitor cells, followed by administration of an immunoablative regimen and subsequent infusion of the previously collected cells. Patients were evaluated immediately before transplantation, at 6 months and then yearly until at least 5-years of post-transplantation follow-up. At each evaluation time point, patients underwent clinical examination, including modified Rodnan’s skin score (mRSS) assessment, echocardiography, high-resolution computed tomography of the lungs and pulmonary function. Results Median (range) age was 35.9 (19–59), with 57 (81.4%) female and median (range) non-Raynaud’s disease duration of 2 (1–7) years. Before transplantation, 96% of the patients had diffuse skin involvement, 84.2%, interstitial lung disease and 67%, positive anti-topoisomerase I antibodies. Skin involvement significantly improved, with a decline in mRSS at all post-transplantation time points until at least 5-years of follow-up. When patients with pre-HSCT interstitial lung disease were analyzed, there was an improvement in pulmonary function (forced vital capacity and diffusing capacity of lung for carbon monoxide) over the 5-year follow-up. Overall survival was 81% and progression-free survival was 70.5% at 8-years after HSCT. Three patients died due to transplant-related toxicity, 9 patients died over follow-up due to disease reactivation and one patient died due to thrombotic thrombocytopenic purpura. Conclusions Autologous hematopoietic progenitor cell transplantation improves skin and interstitial lung involvement. These results are in line with the international experience and support HSCT as a viable therapeutic alternative for patients with severe and progressive systemic sclerosis.

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Section: Discussionsupporting

confidence: 89%

Section: Introductionmentioning

confidence: 99%

Hematopoietic stem cell transplantation for systemic sclerosis: Brazilian experience

et al. 2021

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“…Observation and study on this series of problems will be helpful in exploring future clinical applications regarding cell transplantation. Microglia autophagy serve an important role in the differentiation, survival and homeostasis maintenance of transplanted stem cells (43)(44)(45)(46). Previous studies have demonstrated that bone marrow-derived neural progenitor cells can differentiate into neurons and their transplantation in vivo can effectively promote the motor function of rats following brain injury (25)(26)(27).…”

Section: Discussionmentioning

confidence: 99%

Bone marrow stromal cells‑derived exosomes target DAB2IP to induce microglial cell autophagy, a new strategy for neural stem cell transplantation in brain injury

et al. 2020

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Bone marrow stromal cells (MSCs) are a useful source of stem cells for the treatment of various brain injury diseases due to their abundant supply and fewer ethical problems compared with transplant treatment. However, the clinical application of MSCs is limited due to allograft rejection and immunosuppression in the process of MSCs transplantation. According to previous studies, microglial cell autophagy occurs following co-culture with MSCs. In the present study, exosomes were obtained from MSCs and subsequently characterized using transmission electron microscopy, atomic force microscopy and dynamic light scattering particle size analysis. The type of microRNAs (miRs) found in the exosomes was then analyzed via gene chip. The results demonstrated that microglial cell autophagy could be induced by exosomes. This mechanism was therefore investigated further via reverse transcription-quantitative PCR, western blotting and luciferase assays. These results demonstrated that exosomes from MSCs could induce microglial cell autophagy through the miR-32-mediated regulation of disabled homolog 2-interacting protein, thus providing a theoretical basis for the clinical application of miRs in MSCs.

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“…The future of HSCT for CD is to continue to tweak the regimen to make it safer and to achieve even longer and more long-lasting results without evidence of disease, that is, durable clinical remission on no drug therapy, and imaging, endoscopic, and histologic remission. Based on the literature and data cited, we consider that there is a place for autologous HSCT in the treatment of patients with severe and refractory CD where the death rate is zero and toxicity is low and that position was highlighted in a recent SBTMO position and a correspondence paper[ 42 - 52 ]. It is well known that autologous HSCT provided treatment-free remission in a large number of patients, but the majority persisted with histological evidence of disease.…”

Section: Discussionmentioning

confidence: 99%

“…Recently SBTMO wrote a position paper accepted for publication. The suggestion is to introduce HSCT into clinical practice for those diseases that are considered experimental in the country[ 42 ].…”

Section: And Hsct In Brazilmentioning

confidence: 99%

Medical, ethical, and legal aspects of hematopoietic stem cell transplantation for Crohn’s disease in Brazil

Ruiz¹,

Kaiser²,

Piron-Ruiz³

et al. 2020

WJSC

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Crohn's disease (CD) is a chronic inflammatory bowel disease that can affect any part of the gastrointestinal tract. The etiology of CD is unknown; however, genetic, epigenetic, environmental, and lifestyle factors could play an essential role in the onset and establishment of the disease. CD results from immune dysregulation due to loss of the healthy symbiotic relationship between host and intestinal flora and or its antigens. It affects both sexes equally with a male to female ratio of 1.0, and its onset can occur at any age, but the diagnosis is most commonly observed in the range of 20 to 40 years of age. CD diminishes quality of life, interferes with social activities, traumatizes due to the stigma of incontinence, fistulae, strictures, and colostomies, and in severe cases, affects survival when compared to the general population. Symptoms fluctuate between periods of remission and activity in which complications such as fistulas, strictures, and the need for bowel resection, surgery, and colostomy implantation make up the most severe aspects of the disease. CD can be progressive and the complications recurrent despite treatment with anti-inflammatory drugs, corticosteroids, immunosuppressants, and biological agents. However, over time many patients become refractory without treatment alternatives, and in this scenario, hematopoietic stem cell transplantation (HSCT) has emerged as a potential treatment option. The rationale for the use of HSCT for CD is anchored in animal studies and human clinical trials where HSCT could reset a patient's immune system by eliminating disease-causing effector cells and upon immune recovery increase regulatory and suppressive immune cells. Autologous HSCT using a non-myeloablative regimen of cyclophosphamide and anti-thymocyte globulin without CD34+ selection has been to date the most common transplant conditioning regimen adopted. In this review we will address the current situation regarding CD treatment with HSCT and emphasize the medical, ethical, and legal aspects that permeate the procedure in Brazil.

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A review of hematopoietic stem cell transplantation for autoimmune diseases: multiple sclerosis, systemic sclerosis and Crohn's disease. Position paper of the Brazilian Society of Bone Marrow Transplantation

Cited by 19 publications

References 134 publications

Hematopoietic stem cell transplantation for systemic sclerosis: Brazilian experience

Hematopoietic stem cell transplantation for systemic sclerosis: Brazilian experience

Bone marrow stromal cells‑derived exosomes target DAB2IP to induce microglial cell autophagy, a new strategy for neural stem cell transplantation in brain injury

Medical, ethical, and legal aspects of hematopoietic stem cell transplantation for Crohn’s disease in Brazil

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