Guidelines of the Brazilian Society of Bone Marrow Transplantation on hematopoietic stem cell transplantation as a treatment for the autoimmune diseases systemic sclerosis and multiple sclerosis

Oliveira, Maria Carolina; Hamerschlak, Nelson; Moraes, Daniela A.; Simões, Belinda Pinto; Rodrigues, Morgani; Ribeiro, Andreza Alice Feitosa; Voltarelli, Júlio César

doi:10.5581/1516-8484.20130035

Cited by 12 publications

(11 citation statements)

References 92 publications

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“…In 2013 Rodrigues et al , 39 on behalf of the Brazilian Society of Bone Marrow Transplantation, developed recommendations on the use of HSCT as a treatment for SSc.…”

Section: State Of the Art On Clinical Practice Guidelinesmentioning

confidence: 99%

Systemic sclerosis: state of the art on clinical practice guidelines

et al. 2018

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Systemic sclerosis (SSc) is an orphan disease characterised by autoimmunity, fibrosis of the skin and internal organs, and vasculopathy. SSc may be associated with high morbidity and mortality. In this narrative review we summarise the results of a systematic literature research, which was performed as part of the European Reference Network on Rare and Complex Connective Tissue and Musculoskeletal Diseases project, aimed at evaluating existing clinical practice guidelines or recommendations. Only in the domains ‘Vascular & Ulcers’ (ie, non-pharmacological approach to digital ulcer), ‘PAH’ (ie, screening and treatment), ‘Treatment’ and ‘Juveniles’ (ie, evaluation of juveniles with Raynaud’s phenomenon) evidence-based and consensus-based guidelines could be included. Hence there is a preponderance of unmet needs in SSc referring to the diagnosis and (non-)pharmacological treatment of several SSc-specific complications. Patients with SSc experience significant uncertainty concerning SSc-related taxonomy, management (both pharmacological and non-pharmacological) and education. Day-to-day impact of the disease (loss of self-esteem, fatigue, sexual dysfunction, and occupational, nutritional and relational problems) is underestimated and needs evaluation.

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“…In 2013 Rodrigues et al , 39 on behalf of the Brazilian Society of Bone Marrow Transplantation, developed recommendations on the use of HSCT as a treatment for SSc.…”

Section: State Of the Art On Clinical Practice Guidelinesmentioning

confidence: 99%

Systemic sclerosis: state of the art on clinical practice guidelines

et al. 2018

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“…Autologous HSCT has been employed in the treatment of some forms of MS, even though it is not considered to be the standard treatment of the disease [1,2,3]. There are still controversies about its indication, which range between total skepticism and opinions about the unethical behavior of neurologists not offering it to patients with MS [4].…”

Section: Discussionmentioning

confidence: 99%

“…The risk of transplant-related mortality in HSCT for MS was conventionally considered very high but has declined since 2001 to less than 1.3% [2,3,4,5,6]; this may be due to the result of the changes in the conditioning regimens, thus reducing toxicity. Recent data, with more than 700 autologous transplants for MS in Europe, showed an overall survival (OS) of 92% in 5 years and a progression-free survival of 46%, the main cause of mortality and morbidity being the recurrence of the autoimmune disease [2,3,4,5,6]. The consensus provides an indication of HSCT in patients with progressive MS unresponsive to conventional therapy and Expanded Disability Status Scale (EDSS) [1] between 3 and 6.…”

Section: Introductionmentioning

confidence: 99%

“…It mainly affects young adults and is twice as common in women than it is in men [1]. Studies published from the 1990s brought animal models and theoretical considerations of hematopoietic stem cell transplantation (HSCT) being useful in the prevention and treatment of autoimmune diseases, with clinical responses in some patients, suggesting that high-dose chemotherapy followed by HSCT rescue could “reset” the immunological changes through the control of autoreactive clones, followed by immunological tolerance after immune reconstitution [2]; this led to the conclusion that HSCT may be a viable therapeutic option for MS [1,2,3,4,5,6]. Autologous HSCT have been given to patients with MS since 1996 and more than 700 HSCTs have been performed around the world [1,2,3,4,5,6].…”

Section: Introductionmentioning

confidence: 99%

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A Feasibility Study of the Full Outpatient Conduction of Hematopoietic Transplants in Persons with Multiple Sclerosis Employing Autologous Non-Cryopreserved Peripheral Blood Stem Cells

et al. 2017

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Background: With the goal of achieving immune system reset, autologous hematopoietic stem cell transplantations have been performed in patients with multiple sclerosis (MS). Material and Methods: Two hundred and eighty-six consecutive patients with MS were autografted in a single center using non-frozen peripheral blood stem cells (PBSCs), on an outpatient basis and conditioning with cyclophosphamide and rituximab. The protocol was registered in ClinicalTrials.gov identifier NCT02674217. Results: One hundred and ninety-four females and 92 males were included; the median age was 47. All procedures were started on an outpatient basis and only 8 persons needed to be admitted to the hospital during the procedure. In order to obtain at least 1 × 10⁶/kg viable CD34 cells, 1-4 aphereses were performed (median 1). The total number of viable CD34+ cells infused ranged between 1 and 19.2 × 10⁶/kg (median 4.6). Patients recovered above 0.5 × 10⁹/L absolute granulocytes on median day 8 (range 0-12). Two individuals needed red blood cells but none needed platelet transfusions. There were no transplant-related deaths and the 128-month overall survival of the patients is 100%. In 82 persons followed up for 3 or more months, the Expanded Disability Status Scale diminished from a mean of 5.2-4.9, the best results being obtained in relapsing-remitting and primary progressive MS. Conclusions: It is possible to conduct autotransplants for patients with MS employing non-frozen PBSCs and outpatient conduction. Additional information is needed to assess the efficacy of these procedures in the treatment of patients with MS.

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“…Every three years, a clinical guidelines meeting occurs, and one of the subjects is autoimmune diseases. In addition to the annual SBTMO meeting, reports and position papers are prepared and published from these events[ 41 ]. Among the most cited autoimmune diseases recommended for HSCT are multiple sclerosis, systemic sclerosis, and CD.…”

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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Guidelines of the Brazilian Society of Bone Marrow Transplantation on hematopoietic stem cell transplantation as a treatment for the autoimmune diseases systemic sclerosis and multiple sclerosis

Cited by 12 publications

References 92 publications

Systemic sclerosis: state of the art on clinical practice guidelines

Systemic sclerosis: state of the art on clinical practice guidelines

A Feasibility Study of the Full Outpatient Conduction of Hematopoietic Transplants in Persons with Multiple Sclerosis Employing Autologous Non-Cryopreserved Peripheral Blood Stem Cells

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

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