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1.
Rev Bras Enferm ; 73 Suppl 2: e20200476, 2020.
Artigo em Português, Inglês | MEDLINE | ID: mdl-32667574

RESUMO

OBJECTIVE: To describe the experience of nursing, in adopting containment measures, in the care of patients undergoing hematopoietic stem cell transplantation to avoid COVID-19. METHODS: Experience report. RESULTS: Containment measures involve those recommended by major health organizations, such as hand hygiene, social isolation, identification and monitoring of suspected or confirmed cases; and also the local measures implemented in the health service, such as the reduction in the number of hospitalizations for transplantation, clinical screening of outpatients entering the service, monitoring of respiratory signs and symptoms, the allocation of specific isolation rooms for those suspected of the disease and testing of symptomatic patients. Final considerations: The nurse is responsible for the challenge of planning nursing care to prevent the spread of coronavirus in a high-risk population and to implement measures based on available evidence, periodically updated.


Assuntos
Transplante de Medula Óssea/enfermagem , Transplante de Medula Óssea/normas , Infecções por Coronavirus/prevenção & controle , Controle de Infecções/normas , Programas de Rastreamento/normas , Cuidados de Enfermagem/normas , Pandemias/prevenção & controle , Pneumonia Viral/prevenção & controle , Guias de Prática Clínica como Assunto , Betacoronavirus , Humanos
2.
Pain Physician ; 23(2): E85-E131, 2020 03.
Artigo em Inglês | MEDLINE | ID: mdl-32214287

RESUMO

BACKGROUND: The use of bone marrow concentrate (BMC) for treatment of musculoskeletal disorders has become increasingly popular over the last several years, as technology has improved along with the need for better solutions for these pathologies. The use of cellular tissue raises a number of issues regarding the US Food and Drug Administration's (FDA) regulation in classifying these treatments as a drug versus just autologous tissue transplantation. In the case of BMC in musculoskeletal and spine care, this determination will likely hinge on whether BMC is homologous to the musculoskeletal system and spine. OBJECTIVES: The aim of this review is to describe the current regulatory guidelines set in place by the FDA, specifically the terminology around "minimal manipulation" and "homologous use" within Regulation 21 CFR Part 1271, and specifically how this applies to the use of BMC in interventional musculoskeletal medicine. METHODS: The methodology utilized here is similar to the methodology utilized in preparation of multiple guidelines employing the experience of a panel of experts from various medical specialties and subspecialties from differing regions of the world. The collaborators who developed these position statements have submitted their appropriate disclosures of conflicts of interest. Trustworthy standards were employed in the creation of these position statements. The literature pertaining to BMC, its effectiveness, adverse consequences, FDA regulations, criteria for meeting the standards of minimal manipulation, and homologous use were comprehensively reviewed using a best evidence synthesis of the available and relevant literature. RESULTS/Summary of Evidence: In conjunction with evidence-based medicine principles, the following position statements were developed: Statement 1: Based on a review of the literature in discussing the preparation of BMC using accepted methodologies, there is strong evidence of minimal manipulation in its preparation, and moderate evidence for homologous utility for various musculoskeletal and spinal conditions qualifies for the same surgical exemption. Statement 2: Assessment of clinical effectiveness based on extensive literature shows emerging evidence for multiple musculoskeletal and spinal conditions. • The evidence is highest for knee osteoarthritis with level II evidence based on relevant systematic reviews, randomized controlled trials and nonrandomized studies. There is level III evidence for knee cartilage conditions. • Based on the relevant systematic reviews, randomized trials, and nonrandomized studies, the evidence for disc injections is level III. • Based on the available literature without appropriate systematic reviews or randomized controlled trials, the evidence for all other conditions is level IV or limited for BMC injections. Statement 3: Based on an extensive review of the literature, there is strong evidence for the safety of BMC when performed by trained physicians with the appropriate precautions under image guidance utilizing a sterile technique. Statement 4: Musculoskeletal disorders and spinal disorders with related disability for economic and human toll, despite advancements with a wide array of treatment modalities. Statement 5: The 21st Century Cures Act was enacted in December 2016 with provisions to accelerate the development and translation of promising new therapies into clinical evaluation and use. Statement 6: Development of cell-based therapies is rapidly proliferating in a number of disease areas, including musculoskeletal disorders and spine. With mixed results, these therapies are greatly outpacing the evidence. The reckless publicity with unsubstantiated claims of beneficial outcomes having putative potential, and has led the FDA Federal Trade Commission (FTC) to issue multiple warnings. Thus the US FDA is considering the appropriateness of using various therapies, including BMC, for homologous use. Statement 7: Since the 1980's and the description of mesenchymal stem cells by Caplan et al, (now called medicinal signaling cells), the use of BMC in musculoskeletal and spinal disorders has been increasing in the management of pain and promoting tissue healing. Statement 8: The Public Health Service Act (PHSA) of the FDA requires minimal manipulation under same surgical procedure exemption. Homologous use of BMC in musculoskeletal and spinal disorders is provided by preclinical and clinical evidence. Statement 9: If the FDA does not accept BMC as homologous, then it will require an Investigational New Drug (IND) classification with FDA (351) cellular drug approval for use. Statement 10: This literature review and these position statements establish compliance with the FDA's intent and corroborates its present description of BMC as homologous with same surgical exemption, and exempt from IND, for use of BMC for treatment of musculoskeletal tissues, such as cartilage, bones, ligaments, muscles, tendons, and spinal discs. CONCLUSIONS: Based on the review of all available and pertinent literature, multiple position statements have been developed showing that BMC in musculoskeletal disorders meets the criteria of minimal manipulation and homologous use. KEY WORDS: Cell-based therapies, bone marrow concentrate, mesenchymal stem cells, medicinal signaling cells, Food and Drug Administration, human cells, tissues, and cellular tissue-based products, Public Health Service Act (PHSA), minimal manipulation, homologous use, same surgical procedure exemption.


Assuntos
Transplante de Medula Óssea/normas , Medicina Baseada em Evidências/normas , Doenças Musculoesqueléticas/terapia , Manejo da Dor/normas , Médicos/normas , Sociedades Médicas/normas , Medula Óssea/fisiologia , Transplante de Medula Óssea/métodos , Medicina Baseada em Evidências/métodos , Humanos , Doenças Musculoesqueléticas/diagnóstico , Doenças Musculoesqueléticas/epidemiologia , Dor/diagnóstico , Dor/epidemiologia , Manejo da Dor/métodos , Ensaios Clínicos Controlados Aleatórios como Assunto/métodos , Resultado do Tratamento , Estados Unidos , United States Food and Drug Administration/normas
3.
Rev. bras. enferm ; 73(supl.2): e20200476, 2020.
Artigo em Inglês | LILACS, BDENF - Enfermagem | ID: biblio-1115412

RESUMO

ABSTRACT Objective: To describe the experience of nursing, in adopting containment measures, in the care of patients undergoing hematopoietic stem cell transplantation to avoid COVID-19. Methods: Experience report. Results: Containment measures involve those recommended by major health organizations, such as hand hygiene, social isolation, identification and monitoring of suspected or confirmed cases; and also the local measures implemented in the health service, such as the reduction in the number of hospitalizations for transplantation, clinical screening of outpatients entering the service, monitoring of respiratory signs and symptoms, the allocation of specific isolation rooms for those suspected of the disease and testing of symptomatic patients. Final considerations: The nurse is responsible for the challenge of planning nursing care to prevent the spread of coronavirus in a high-risk population and to implement measures based on available evidence, periodically updated.


RESUMEN Objetivo: Describir la experiencia de la enfermería, en la adopción de medidas de contención, en la atención de pacientes sometidos al trasplante de células madre hematopoyéticas para evitar la COVID-19. Métodos: Relato de experiencia. Resultados: Las medidas de contención envuelven aquellas recomendadas por las principales organizaciones de salud, como la higiene de manos, aislamiento social, identificación y monitoreo de casos sospechosos o confirmados; y aún las medidas locales implementadas en el servicio de salud, como la reducción en el número de internamientos para el trasplante, selección clínica de pacientes ambulatorios que adentran el servicio, monitoreo de señales y síntomas respiratorios, destinación de cuartos de aislamiento específicos para los sospechosos de la enfermedad y análisis de pacientes sintomáticos. Consideraciones finales: Compete al enfermero el desafío de planear el cuidado de enfermería para la prevención de la diseminación del coronavirus en población de alto riesgo y para la implementación de medidas pautadas en las evidencias disponibles, periódicamente actualizadas.


RESUMO Objetivo: Descrever a experiência da enfermagem, na adoção de medidas de contenção, no atendimento de pacientes submetidos ao transplante de células-tronco hematopoiéticas para evitar a COVID-19. Métodos: Relato de experiência. Resultados: As medidas de contenção envolvem aquelas recomendadas pelas principais organizações de saúde, como a higiene de mãos, isolamento social, identificação e monitoramento de casos suspeitos ou confirmados; e ainda as medidas locais implementadas no serviço de saúde, como a redução no número de internações para o transplante, triagem clínica de pacientes ambulatoriais que adentram o serviço, monitoramento de sinais e sintomas respiratórios, destinação de quartos de isolamento específicos para os suspeitos da doença e testagem de pacientes sintomáticos. Considerações finais: Compete ao enfermeiro o desafio de planejar o cuidado de enfermagem para a prevenção da disseminação do coronavírus em população de alto risco e para a implementação de medidas pautadas nas evidências disponíveis, periodicamente atualizadas.


Assuntos
Humanos , Pneumonia Viral/prevenção & controle , Programas de Rastreamento/normas , Transplante de Medula Óssea/enfermagem , Transplante de Medula Óssea/normas , Controle de Infecções/normas , Guias de Prática Clínica como Assunto , Infecções por Coronavirus/prevenção & controle , Pandemias/prevenção & controle , Cuidados de Enfermagem/normas , Betacoronavirus
4.
Sultan Qaboos Univ Med J ; 19(1): e15-e18, 2019 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-31198590

RESUMO

Objectives: Haematopoietic stem cell transplantation (HSCT) in Oman started in 1994 at Sultan Qaboos University Hospital (SQUH), Muscat, Oman. Previous studies have suggested that longer driving time to the transplant centre (DTC) independently correlates with worse overall survival (OS). Therefore, this study aimed to examine the impact of DTC on OS and acute graft-versus-host disease (aGvHD). Methods: This retrospective study included all patients who underwent HSCT between February 2006 and December 2016 at SQUH. The DTC was determined using Google Maps (Google LLC., Mountain View, California, USA). The probability of OS was estimated using a Kaplan-Meier estimator and the impact of DTC on OS was compared using a Cox model. Results: A total of 170 patients were included in this study of which 52% were male and 28% were from the Al Batinah region. The mean age was 14.2 ± 12.2 years. The mean haemoglobin, platelet and white blood cell counts before the HSCT were 10.3 ± 1.7 g/dL, 207 ± 131 × 109/L and 5.1 ± 5.9 × 109/L, respectively. The median DTC for those with aGvHD was 84 minutes, which is similar to patients without aGvHD (P = 0.918). The hazard ratio for DTC as a predictor of OS was 1.0 (P = 0.901). Conclusion: In this single centre study, DTC did not impact aGvHD or OS in patients post-HSCT. The study was limited by its retrospective design and the small sample size. It is recommended that these results be confirmed in a prospective study.


Assuntos
Transplante de Medula Óssea/métodos , Cuidado Transicional/normas , Resultado do Tratamento , Adolescente , Adulto , Transplante de Medula Óssea/normas , Criança , Pré-Escolar , Feminino , Serviços de Assistência Domiciliar/normas , Humanos , Masculino , Omã , Modelos de Riscos Proporcionais , Estudos Prospectivos , Estudos Retrospectivos , Análise de Sobrevida
5.
Bull Cancer ; 106(1S): S83-S91, 2019 Jan.
Artigo em Francês | MEDLINE | ID: mdl-30528618

RESUMO

JACIE (Joint Accreditation Committee ISTC EBMT) regulations and standards impose a quality and safety requirement for graft reinjection by nurses. However, the standards do not provide a step-by-step graft reinjection procedure. Because of high medical team turnover, the opening of new transplant centers, and continual questions from colleagues trying to decipher the JACIE standards, the need for a specific procedure goes without saying. We collected graft reinjection procedures from each SFGM-TC center that participated in our survey, thus creating an inventory of the different steps that make up graft reinjection. In addition to reviewing the main regulatory texts and JACIE standards, we sought advice from medical and cellular therapy experts. We observed that most centers use a mix of practices and some unjustified practices. In some transplant units, it is still standard practice to defrost cell therapy products in the transplant unit. Caregivers are aware of the need for a rigorous application of the regulatory requirements and are willing to administer a procedure that provides specific steps for each stage of the process. In this workshop, we questioned each stage of the graft reinjection procedure, which helped us define clear methods of implementation. In the form of a checklist, we offer bone marrow and stem cell transplant units a step-by-step procedure.


Assuntos
Transplante de Medula Óssea/normas , Transplante de Células-Tronco Hematopoéticas/normas , Retratamento/normas , Transplante de Medula Óssea/legislação & jurisprudência , Transplante de Medula Óssea/métodos , Criopreservação , França , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Transplante de Células-Tronco Hematopoéticas/legislação & jurisprudência , Transplante de Células-Tronco Hematopoéticas/métodos , Humanos , Sistemas de Identificação de Pacientes/métodos , Pré-Medicação/métodos , Pré-Medicação/normas , Retratamento/efeitos adversos , Retratamento/métodos , Sociedades Médicas , Temperatura
6.
Bull Cancer ; 106(1S): S1-S9, 2019 Jan.
Artigo em Francês | MEDLINE | ID: mdl-30580913

RESUMO

Allogeneic hematopoietic cell transplantation (HCT) is part of the standard of care for many hematological diseases. Over the last decades, significant advances in patient and donor selection, conditioning regimens as well as supportive care of patients undergoing allogeneic HCT leading to improved overall survival have been made. In view of many new treatment options in cellular and molecular targeted therapies, the place of allogeneic transplantation in therapy concepts must be reviewed. Most aspects of HCT are well standardized by national guidelines or laws as well as by certification labels such as FACT-JACIE. However, the requirements for human resources, construction and layout of a unit treating patients during the transplantation procedure and for different complications are not well defined. Here, we describe the process of planning a transplant unit in order to open a discussion that could lead to more precise guidelines in the field of personnel and infrastructural requirements for hospitals caring for people with severe immunosuppression.


Assuntos
Transplante de Medula Óssea/normas , Ambiente de Instituições de Saúde/normas , Doenças Hematológicas/terapia , Transplante de Células-Tronco Hematopoéticas/normas , Ar/normas , Terapia Baseada em Transplante de Células e Tecidos/normas , Dieta Saudável/normas , Seleção do Doador/normas , França , Pessoal de Saúde/normas , Unidades Hospitalares/normas , Humanos , Higiene , Imunossupressão/normas , Monitorização Fisiológica/métodos , Roupa de Proteção/normas , Sociedades Médicas , Esterilização/normas , Transplante Homólogo/normas , Visitas a Pacientes
7.
Vox Sang ; 113(8): 795-802, 2018 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-30191563

RESUMO

BACKGROUND: Bone marrow harvest (BMH) for haematopoietic stem cell transplantation is a well-established procedure. The guidelines of World Marrow Donor Association provide information on donor selection. However, some of the guidelines regarding donors with anaemia prior to harvest lack in supporting data from clinical studies. With this study, we aimed to provide such data. MATERIAL AND METHODS: In this retrospective, single-centre study, we analysed the interplay between haemoglobin levels and BMH and BMH impact on haemoglobin levels in a cohort of 149 unrelated BM donors, including 13 subjects with mild anaemia. RESULTS: The BMH led to significantly lower decrease in haemoglobin levels in donors with anaemia than in control group (1·79 g/dl vs. 2·56 g/dl, P < 0·0001). The following parameters: BMH volume (ml), BMH volume/donor body weight (ml/kg), total nucleated cells (TNC) in product (×108 ) and TNC/kg recipient body weight in product (×108 /kg) did not differ significantly between those two analysed groups (P > 0·05). Median BM volume harvested from anaemic donors was 16·34 ml/kg; none of them required blood transfusion after BMH. CONCLUSION: Mild anaemia prior to BMH does not significantly impact the collection results. The BMH is safe and feasible in donors with mild anaemia.


Assuntos
Anemia/sangue , Doadores de Sangue , Transplante de Medula Óssea/métodos , Seleção do Doador/métodos , Adulto , Células da Medula Óssea/química , Transplante de Medula Óssea/normas , Seleção do Doador/normas , Feminino , Hemoglobinas/análise , Humanos , Pessoa de Meia-Idade
8.
Curr Res Transl Med ; 66(2): 57-58, 2018 05.
Artigo em Inglês | MEDLINE | ID: mdl-29685843

RESUMO

The Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC) hold its eighth practice harmonization workshops on September 2017. In a workshop dedicated to chimeric antigen receptor T-cell therapy (CAR T-cells), the society issued recommendations regarding the prerequisite for hematopoietic cellular therapy programs to set up CAR T-cell therapy. In this article we focused on the prerequisite needed, in France, for a hematopoietic transplantation unit to start a CAR T-cell program with industrial manufactured cells within investigational products or after market access authorization.


Assuntos
Transplante de Medula Óssea/normas , Terapia Baseada em Transplante de Células e Tecidos/normas , Imunoterapia Adotiva/normas , Receptores de Antígenos Quiméricos/uso terapêutico , Linfócitos T/transplante , Transplante de Medula Óssea/métodos , Terapia Baseada em Transplante de Células e Tecidos/métodos , França , Transplante de Células-Tronco Hematopoéticas/normas , Unidades Hospitalares/organização & administração , Unidades Hospitalares/normas , Humanos , Imunoterapia Adotiva/métodos , Receptores de Antígenos Quiméricos/genética , Sociedades Médicas , Linfócitos T/fisiologia , Transplante Homólogo
9.
Circ J ; 82(6): 1688-1698, 2018 05 25.
Artigo em Inglês | MEDLINE | ID: mdl-29576595

RESUMO

BACKGROUND: The therapeutic efficacy of bone marrow mononuclear cells (BM-MNC) autotransplantation in critical limb ischemia (CLI) has been reported. Variable proportions of circulating monocytes express low levels of CD34 (CD14+CD34lowcells) and behave in vitro as endothelial progenitor cells (EPCs). The aim of the present randomized clinical trial was to compare the safety and therapeutic effects of enriched circulating EPCs (ECEPCs) with BM-MNC administration.Methods and Results:ECEPCs (obtained from non-mobilized peripheral blood by immunomagnetic selection of CD14+and CD34+cells) or BM-MNC were injected into the gastrocnemius of the affected limb in 23 and 17 patients, respectively. After a mean of 25.2±18.6-month follow-up, both groups showed significant and progressive improvement in muscle perfusion (primary endpoint), rest pain, consumption of analgesics, pain-free walking distance, wound healing, quality of life, ankle-brachial index, toe-brachial index, and transcutaneous PO2. In ECEPC-treated patients, there was a positive correlation between injected CD14+CD34lowcell counts and the increase in muscle perfusion. The safety profile was comparable between the ECEPC and BM-MNC treatment arms. In both groups, the number of deaths and major amputations was lower compared with eligible untreated patients and historical reference patients. CONCLUSIONS: This study supports previous trials showing the efficacy of BM-MNC autotransplantation in CLI patients and demonstrates comparable therapeutic efficacy between BM-MNC and EPEPCs.


Assuntos
Transplante de Medula Óssea/métodos , Células Progenitoras Endoteliais/transplante , Isquemia/terapia , Transplante Autólogo/métodos , Idoso , Amputação , Células da Medula Óssea , Transplante de Medula Óssea/normas , Extremidades/patologia , Feminino , Humanos , Leucócitos Mononucleares/transplante , Masculino , Pessoa de Meia-Idade , Análise de Sobrevida , Transplante Autólogo/normas
10.
Biol Blood Marrow Transplant ; 24(5): 909-913, 2018 05.
Artigo em Inglês | MEDLINE | ID: mdl-29360515

RESUMO

Choosing Wisely encourages dialogue about reducing unnecessary procedures, tests, or treatments in healthcare. The American Society for Blood and Marrow Transplantation (ASBMT) and Canadian Blood and Marrow Transplant Group (CBMTG) established a Choosing Wisely BMT Task Force whose objective was to create a list of top 5 practices in blood and marrow transplantation to be questioned. The Task Force consisted of representatives from ASBMT's Quality Outcomes, Education, and Practice Guidelines committees; ASBMT's Pharmacy Special Interest Group; CBMTG Program Directors; and Center for International Blood and Marrow Transplant Research (CIBMTR). Suggestions for current transplantation practices to question were elicited from the CBMTG Program Directors; members of ASBMT's Quality Outcomes, Practice Guidelines, and Education committees; and chairs of the CIBMTR scientific working committees. We received 119 unique suggestions that were ranked based on their potential impact on harm reduction, cost reduction, necessity of the test or practice, and the strength of available evidence. Through a modified Delphi process, suggestions were narrowed down to 6, which were then subjected to systematic reviews. The final 5 recommendations focus on graft source for patients with aplastic anemia, corticosteroid dose for initial treatment of graft-versus-host-disease, optimal number of umbilical cord blood units for transplantation, graft source in matched unrelated donor transplantation, and use of prophylactic intravenous immunoglobulin in transplant recipients. These Choosing Wisely BMT recommendations are relevant to the current clinical practice of blood and marrow transplantation and focus on tests, treatments, or procedures that may be harmful, wasteful, or for which there is no apparent clinical benefit.


Assuntos
Transplante de Medula Óssea/normas , Transplante de Células-Tronco/normas , Comitês Consultivos , Transplante de Medula Óssea/métodos , Canadá , Assistência à Saúde/economia , Assistência à Saúde/normas , Humanos , Transplante de Células-Tronco/métodos , Terapêutica/economia , Terapêutica/normas , Estados Unidos
12.
Bull Cancer ; 104(12S): S65-S70, 2017 Dec.
Artigo em Francês | MEDLINE | ID: mdl-29128085

RESUMO

As part of the 7th Annual francophone workshop series on the harmonization of clinical practices in allogeneic stem cell transplantation held in Lille in September 2016, our workgroup discussed how transplant centers might follow a collective approach to coding data. This was done mainly by analyzing the study results found in the literature that do not provide clear answers. In addition, we discuss practical ways of coding for both donor and recipient HLA typing in the European bone marrow transplantation database called ProMISe which is managed by the European Society for Blood and Marrow Transplantation (EBMT).


Assuntos
Transplante de Medula Óssea/normas , Bases de Dados Factuais , Antígenos HLA/classificação , Teste de Histocompatibilidade/normas , Alelos , Terapia Baseada em Transplante de Células e Tecidos/normas , França , Antígenos HLA/imunologia , Teste de Histocompatibilidade/métodos , Humanos , Sociedades Médicas
14.
Biol Blood Marrow Transplant ; 23(5): 717-725, 2017 May.
Artigo em Inglês | MEDLINE | ID: mdl-27713091

RESUMO

Hematopoietic cell transplantation (HCT) survivors are at risk for development of late complications and require lifelong monitoring for screening and prevention of late effects. There is an increasing appreciation of the issues related to healthcare delivery and coverage faced by HCT survivors. The 2016 National Institutes of Health Blood and Marrow Transplant Late Effects Initiative included an international and broadly representative Healthcare Delivery Working Group that was tasked with identifying research gaps pertaining to healthcare delivery and to identify initiatives that may yield a better understanding of the long-term value and costs of care for HCT survivors. There is a paucity of literature in this area. Critical areas in need of research include pilot studies of novel and information technology supported models of care delivery and coverage for HCT survivors along with development and validation of instruments that capture patient-reported outcomes. Investment in infrastructure to support this research, such as linkage of databases including electronic health records and routine inclusion of endpoints that will inform analyses focused around care delivery and coverage, is required.


Assuntos
Transplante de Medula Óssea/métodos , Bases de Dados Factuais , Assistência à Saúde/métodos , Transplante de Células-Tronco Hematopoéticas/métodos , National Institutes of Health (U.S.) , Projetos de Pesquisa , Transplante de Medula Óssea/efeitos adversos , Transplante de Medula Óssea/normas , Assistência à Saúde/tendências , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Transplante de Células-Tronco Hematopoéticas/normas , Humanos , Efeitos Adversos de Longa Duração , Sobreviventes , Estados Unidos
15.
Bull Cancer ; 103(11S): S229-S242, 2016 Nov.
Artigo em Francês | MEDLINE | ID: mdl-27842860

RESUMO

Haploidentical hematopoietic stem cell transplantation (HSCT) is being increasingly used due to improvement of the transplantation procedures allowing a reduction of graft-versus-host-disease (GVHD) and of transplant-related mortality (TRM). Such improvements have been particularly observed after administration of T-replete HSCT graft associated to an in vivo T cell depletion by the administration of high-doses of cyclophosphamide (HD-Cy) after transplantation. Here, we have analyzed the results of haplo-identical T replete HSC transplants, in particular, when performed with post-transplant HD-Cy in order to provide recommendations for the clinical practice. Criteria of choice for a haploidentical donor by priority order are absence of donor-specific antibodies (DSA) and to prioritize: CMV seronegative recipient/donor couples, ABO matching in case of deserythrocytation, male donor for a male recipient, the youngest donor. There is no clear argument in favor of the use of bone marrow versus peripheral blood stem cells (PBSC) after non myeloablative conditioning regimen, while after ablative conditioning PBSC seem to be associated with higher risks of GVHD without obvious impact on survival. Results of haploidentical HSCT, confirmed by several groups, are interesting in lymphomas (in particular Hodgkin disease) and for acute leukemia. Outcomes of patients rely on age, disease status at transplant and conditioning intensity. At equivalent disease risk, results of haploidentical HSCT seem comparable to those of HLA matched HSCT, raising the question of the classification of such transplants as alternatives. In all cases, we recommend to include patients in prospective clinical trials.


Assuntos
Seleção do Doador/normas , Haploidia , Transplante de Células-Tronco Hematopoéticas , Histocompatibilidade/genética , Sistema ABO de Grupos Sanguíneos , Fatores Etários , Transplante de Medula Óssea/normas , Doença Enxerto-Hospedeiro/imunologia , Doença Enxerto-Hospedeiro/prevenção & controle , Teste de Histocompatibilidade , Doença de Hodgkin/terapia , Humanos , Leucemia/terapia , Linfoma não Hodgkin/terapia , Fatores Sexuais , Sociedades Médicas , Linfócitos T/imunologia , Condicionamento Pré-Transplante/métodos
16.
Int J Hematol ; 104(2): 168-74, 2016 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-27278666

RESUMO

Standard front-line treatment for acquired aplastic anemia (AA) for patients is either immunosuppressive therapy (IST) or bone marrow transplantation (BMT), usually from an HLA identical sibling. Whereas long-term survival is comparable with either treatment, important differences remain: IST patients may have incomplete or no recovery, are exposed to late clonal disorders and relapse of the original disease. Transplantation is a curative treatment, but patients are exposed to transplant-related complications both acute and chronic, such as chronic graft versus host disease (cGvHD). In the year 2000, a study by the European Group for Blood and Marrow Transplantation (EBMT), looked at failure free survival (FFS), in patients receiving first-line BMT from an HLA identical sibling, or the first-line IST. Young patients with low neutrophil counts benefited of the first-line BMT; the opposite was true for older patients with higher neutrophil counts; and a third intermediate group of patients had comparable survival irrespective of the first-line therapy. We have now studied a more recent cohort of patients to assess whether things have changed over the years. We have found similar results, although overall survival has improved, as a consequence of changes in the IST and BMT protocols.


Assuntos
Anemia Aplástica/terapia , Transplante de Medula Óssea/métodos , Imunossupressão/métodos , Anemia Aplástica/mortalidade , Transplante de Medula Óssea/efeitos adversos , Transplante de Medula Óssea/normas , Humanos , Imunossupressão/efeitos adversos , Imunossupressão/normas , Imunossupressores/uso terapêutico , Resultado do Tratamento
17.
Nat Rev Dis Primers ; 2: 16010, 2016 03 10.
Artigo em Inglês | MEDLINE | ID: mdl-27159408

RESUMO

Acute myeloid leukaemia (AML) is a disorder characterized by a clonal proliferation derived from primitive haematopoietic stem cells or progenitor cells. Abnormal differentiation of myeloid cells results in a high level of immature malignant cells and fewer differentiated red blood cells, platelets and white blood cells. The disease occurs at all ages, but predominantly occurs in older people (>60 years of age). AML typically presents with a rapid onset of symptoms that are attributable to bone marrow failure and may be fatal within weeks or months when left untreated. The genomic landscape of AML has been determined and genetic instability is infrequent with a relatively small number of driver mutations. Mutations in genes involved in epigenetic regulation are common and are early events in leukaemogenesis. The subclassification of AML has been dependent on the morphology and cytogenetics of blood and bone marrow cells, but specific mutational analysis is now being incorporated. Improvements in treatment in younger patients over the past 35 years has largely been due to dose escalation and better supportive care. Allogeneic haematopoietic stem cell transplantation may be used to consolidate remission in those patients who are deemed to be at high risk of relapse. A plethora of new agents - including those targeted at specific biochemical pathways and immunotherapeutic approaches - are now in trial based on improved understanding of disease pathophysiology. These advances provide good grounds for optimism, although mortality remains high especially in older patients.


Assuntos
Leucemia Mieloide Aguda/diagnóstico , Leucemia Mieloide Aguda/fisiopatologia , Mutação/genética , Transplante de Medula Óssea/mortalidade , Transplante de Medula Óssea/normas , Tratamento Farmacológico/métodos , Dispneia/etiologia , Fadiga/etiologia , Células-Tronco Hematopoéticas/patologia , Humanos , Leucemia Mieloide Aguda/epidemiologia , Síndromes Mielodisplásicas/complicações , Recidiva , Indução de Remissão/métodos , Fatores de Risco
18.
Biol Blood Marrow Transplant ; 22(6): 1056-1064, 2016 06.
Artigo em Inglês | MEDLINE | ID: mdl-26964698

RESUMO

Chronic myeloid leukemia (CML) in children and young adults is uncommon. Young patients have long life expectancies and low morbidity with hematopoietic cell transplantation (HCT). Prolonged tyrosine kinase inhibitor (TKI) use may cause significant morbidity. In addition, indication for HCT in patients in the first chronic phase is not established. We hence retrospectively evaluated outcomes in 449 CML patients with early disease receiving myeloablative HCT reported to the CIBMTR. We analyzed various factors affecting outcome, specifically the effect of age and pre-HCT TKI in pediatric patients (age < 18 years, n = 177) and young adults (age 18 to 29 years, n = 272) with the goal of identifying prognostic factors. Post-HCT probability rates of 5-year overall survival (OS) and leukemia-free survival (LFS) were 75% and 59%, respectively. Rates of OS and LFS were 76% and 57% in <18-year and 74% and 60% in 18- to 29-year group, respectively, by univariate analysis (P = .1 and = .6). Five-year rates of OS for HLA matched sibling donor (MSD) and bone marrow (BM) stem cell source were 83% and 80%, respectively. In multivariate analysis there was no effect of age (<18 versus 18 to 29) or pre-HCT TKI therapy on OS, LFS, transplant related mortality, or relapse. Favorable factors for OS were MSD (P < .001) and recent HCT (2003 to 2010; P = .04). LFS was superior with MSD (P < .001), BM as graft source (P = .001), and performance scores > 90 (P = .03) compared with unrelated or mismatched peripheral blood stem cells donors and recipients with lower performance scores. Older age was associated with increased incidence of chronic graft-versus-host disease (P = .0002). In the current era, HCT outcomes are similar in young patients and children with early CML, and best outcomes are achieved with BM grafts and MSD.


Assuntos
Transplante de Células-Tronco Hematopoéticas/normas , Leucemia Mielogênica Crônica BCR-ABL Positiva/terapia , Adulto , Fatores Etários , Transplante de Medula Óssea/normas , Criança , Doença Enxerto-Hospedeiro/etiologia , Transplante de Células-Tronco Hematopoéticas/métodos , Humanos , Masculino , Morbidade , Prognóstico , Estudos Retrospectivos , Irmãos , Análise de Sobrevida , Doadores de Tecidos , Transplante Homólogo , Resultado do Tratamento , Adulto Jovem
19.
Transfusion ; 56(5): 1052-7, 2016 05.
Artigo em Inglês | MEDLINE | ID: mdl-26898844

RESUMO

BACKGROUND: Previous studies have demonstrated the importance of bone marrow (BM) harvest yield in determining transplant outcomes, but little is known regarding donor and procedure variables associated with achievement of an optimal yield. We hypothesized that donor demographics and variables relating to the procedure were likely to impact the yield (total nucleated cells [TNCs]/kg recipient weight) and quality (TNCs/mL) of the harvest. STUDY DESIGN AND METHODS: To test our hypothesis, BM harvests of 110 consecutive unrelated donors were evaluated. The relationship between donor or procedure characteristics and the BM harvest yield was examined. RESULTS: The relationship between donor and recipient weight significantly influenced the harvest yield; only 14% of BM harvests from donors who weighed less than their recipient achieved a TNC count of more than 4 × 10(8) /kg compared to 56% of harvests from donors heavier than their recipient (p = 0.001). Higher-volume harvests were significantly less likely to achieve an optimal yield than lower-volume harvests (32% vs. 78%; p = 0.007), and higher-volume harvests contained significantly fewer TNCs/mL, indicating peripheral blood contamination. BM harvest quality also varied significantly between collection centers adding to recent concerns regarding maintenance of BM harvest expertise within the transplant community. CONCLUSION: Since the relationship between donor and recipient weight has a critical influence yield, we recommend prioritizing this secondary donor characteristic when selecting from multiple well-matched donors. Given the declining number of requests for BM harvests, it is crucial that systems are developed to train operators and ensure expertise in this procedure is retained.


Assuntos
Peso Corporal , Transplante de Medula Óssea/métodos , Doadores de Tecidos , Coleta de Tecidos e Órgãos/métodos , Adolescente , Adulto , Medula Óssea , Células da Medula Óssea/citologia , Transplante de Medula Óssea/normas , Contagem de Células , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Probabilidade , Coleta de Tecidos e Órgãos/normas , Adulto Jovem
20.
Biomed Res Int ; 2015: 473159, 2015.
Artigo em Inglês | MEDLINE | ID: mdl-26495296

RESUMO

According to the European Medicine Agency (EMA) regulatory frameworks, Advanced Therapy Medicinal Products (ATMP) represent a new category of drugs in which the active ingredient consists of cells, genes, or tissues. ATMP-CD133 has been widely investigated in controlled clinical trials for cardiovascular diseases, making CD133(+) cells one of the most well characterized cell-derived drugs in this field. To ensure high quality and safety standards for clinical use, the manufacturing process must be accomplished in certified facilities following standard operative procedures (SOPs). In the present work, we report the fully compliant GMP-grade production of ATMP-CD133 which aims to address the treatment of chronic refractory ischemic heart failure. Starting from bone marrow (BM), ATMP-CD133 manufacturing output yielded a median of 6.66 × 10(6) of CD133(+) cells (range 2.85 × 10(6)-30.84 × 10(6)), with a viability ranged between 96,03% and 99,97% (median 99,87%) and a median purity of CD133(+) cells of 90,60% (range 81,40%-96,20%). Based on these results we defined our final release criteria for ATMP-CD133: purity ≥ 70%, viability ≥ 80%, cellularity between 1 and 12 × 10(6) cells, sterile, and endotoxin-free. The abovementioned criteria are currently applied in our Phase I clinical trial (RECARDIO Trial).


Assuntos
Antígenos CD/metabolismo , Células da Medula Óssea/citologia , Transplante de Medula Óssea/normas , Cardiomiopatias/terapia , Glicoproteínas/metabolismo , Isquemia Miocárdica/terapia , Peptídeos/metabolismo , Transplante de Células-Tronco/normas , Antígeno AC133 , Animais , Cardiomiopatias/etiologia , Cardiomiopatias/patologia , Aprovação de Equipamentos/normas , Europa (Continente) , Fidelidade a Diretrizes , Humanos , Isquemia Miocárdica/complicações , Isquemia Miocárdica/patologia , Guias de Prática Clínica como Assunto , Células-Tronco
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