Greffe de cellules souches hématopoïétiques

Greffe de cellules souches
  • 文章类型: English Abstract
    造血干细胞移植前的调理方案通过使用性腺照射和/或双功能烷化剂对患者生育能力有影响。它们对生育的影响主要取决于使用的剂量,在女性中,治疗时的年龄。所有患者应在治疗前受益于咨询,告知他们对生育能力和生育能力保护技术的潜在影响。在没有禁忌症的情况下,清髓性预处理方案的主要毒性证明保留生育力是合理的。强度降低后,关于生育力的数据很少。尽管理论性腺毒性较低,我们还建议保留生育能力,如果可能的话,在移植前。使用的生育力保存技术取决于患者的年龄,病理学和调理。如果随后在急性白血病或侵袭性淋巴瘤的情况下使用收获的性腺组织,建议评估肿瘤细胞重新引入的风险。最后,建议在移植后评估性腺功能,特别是在减少调理后。如果有持续性的残存性腺功能,应讨论治疗后的生育力保护。
    Conditioning regimen prior to hematopoietic stem cell transplantation have an impact on patient fertility through the use of gonadal irradiation and/or bifunctional alkylating agents. Their impact on fertility depends mainly on the dose used and, in women, on age at the time of treatment. All patients should benefit before treatment from a consultation informing them of the potential impact on fertility and of fertility preservation techniques. In the absence of contraindications, the major toxicity of myeloablative conditioning regimen justifies fertility preservation. There are few data concerning fertility after reduced-intensity conditioning. Despite lower theoretical gonadotoxicity, we also recommend fertility preservation, if possible before transplantation. The fertility preservation techniques used depend on the patient\'s age, pathology and conditioning. In the event of subsequent use of harvested gonadal tissue in the context of acute leukemia or aggressive lymphoma, it is advisable to assess the risk of reintroduction of tumor cells. Finally, it is recommended to assess gonadal function after transplant, especially after reduced conditioning. If there is persistent residual gonadal function, post-treatment fertility preservation should be discuss.
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  • 文章类型: English Abstract
    从儿科捐献者中收集造血干细胞是一种常见的挽救生命的做法,在过去的十年中,SFGM-TC中心的未成年人捐赠者移植病例的数量每年都在增加。尽管如此,从健康和未成年捐赠者的人体组织的医疗使用需要适当的法规和医疗管理。以下准则旨在强调思考法律的重要性,使用健康儿科供体干细胞的医学和伦理方面,并强调在评估HLA相容性时获得知情同意的重要性。捐赠前需要进行综合医疗和心理评估,以及一个月后和一年后,以确保孩子的身心健康。全身麻醉下的骨髓收获仍然是儿童的首选收集方法。外周血干细胞收集只应考虑不需要中心静脉途径收集的儿童。我们的目标是提供以健康儿童捐赠干细胞和他/她的健康为中心的指南,这些应该随着医疗实践的发展定期审查。
    Haematopoietic stem cell collection from paediatric donors is a common and life-saving practice, as evidenced by the fact that there is a growing annual number of cases of transplants from minor donors among SFGM-TC centers over the last decade. Still, medical use of human tissue from a healthy and underage donor requires proper regulations and medical management. The guidelines below aim at underlining the importance of pondering the legal, medical and ethical aspects of using stem cells from healthy paediatric donors and stress out the importance of obtaining informed consent at the time of assessing HLA compatibility. Combined medical and psychological assessments are required before the donation, as well as one month later and one year later to ensure of the child\'s physical and mental wellbeing. Bone marrow harvest under general anaesthetics remains the preferred method of collection for children. Peripheral blood stem cell collection should only be considered for children who will not require a central venous access for collection. We aim at offering guidelines centered on the healthy child donating stem cells and his/her wellbeing, and these should be regularly reviewed as medical practices evolve.
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  • 文章类型: English Abstract
    儿科的做法-特别是在照顾低体重儿童时-不同于成人的做法,由于儿科患者的手术数量很少,这对相关医护人员的实践培训和再培训具有实际意义,根据适应训练和验证持续能力的需要。必须进行专门的培训,以确保儿童和工作人员在收集期间和之后的安全,以及确保收集的细胞产品的高质量,并且其符合取决于其预期用途的预定义规格。低体重和极低体重儿童在许多程序和临床方面值得特别注意:静脉通路的性质和质量,以确保细胞分离器的正常运行,与儿童的总血液体积相比,与体外血液体积的相对重要性相关的血液动力学波动的管理,柠檬酸盐毒性的风险和临床表现,在手术过程中压力的最小化,可能包括但不限于药物镇静。处理这些方面所需的全部能力很少出现在一个单一的医疗保健专业人员团队中;它通常需要从收集设施中汲取的专业知识的紧密结合,儿科,可能还有儿科重症监护病房,无论是来自同一机构还是来自不同机构。互动必须在一份文件中形式化,准确描述哪一类行为者对每一类行为负责(处方、decisions),根据他们最初的资格,特定能力,和从属关系。
    Practice of pediatric aphereses - in particular when caring for low-weight children - differs from the practice of adult aphereses, since pediatric aphereses represent low numbers of procedures, which has practical implications in terms of practical training and retraining for involved healthcare personnel, as needed for habilitation and validation of ongoing competencies. A specific training is mandatory in order to ensure both the child and the staff safety during and after collection, as well as ensure high quality of the collected cell product and that its meets predefined specifications that depend on its intended use. Low and very low-weight children deserve a particular attention for a number of procedural and clinical aspects: the nature and quality of venous accesses to ensure proper operation of the cell separator, management of hemodynamic fluctuations in relation with the relative importance of the extracorporeal blood volume as compared to the total blood volume of the child, risks and clinical manifestations of citrate toxicity, minimization of stress during the procedure that may include but is not limited to pharmacological sedation. The full spectrum of competencies needed to deal with these aspects is rarely present within a single team of healthcare professionals; it most often requires the tight combination of expertise drawing from the collection facility, the pediatric department and possibly the pediatric intensive care unit ward, whether from the same or from different institutions. Interactions must be formalized in a document that accurately describes which category of actors is responsible for each category of acts (prescriptions, decisions), depending on their initial qualifications, specific competencies, and affiliations.
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  • 文章类型: English Abstract
    供体的选择是异基因造血干细胞移植的基本要素。在没有HLA匹配相关供体的情况下,考虑选择不相关的捐赠者,并且是目前在实践中使用的最常见的同种异体供体类型。当多个供体可用时,选择考虑了许多标准,特别是在部分匹配的情况下。该讲习班的目的是协助选择无关的捐助者,与文献中的最新数据保持一致。
    The selection of a donor is an essential element in allogeneic hematopoietic stem cell transplantation. In the absence of an HLA-matched related donor, the selection of an unrelated donor is considered, and is currently the most common type of allogenic donor used in practice. Many criteria are considered for the selection when multiple donors are available, particularly in case of partial match. The aim of this workshop is to assist in the selection of an unrelated donor, in keeping with recent data from the literature.
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  • 文章类型: English Abstract
    异基因造血细胞移植(allo-HCT)后的复发仍然是一个主要问题,因为它与不良生存率有关。在这种情况下,第二allo-HCT是有效选项。在法语国家骨髓移植和细胞治疗协会(SFGM-TC)的第13届年度协调研讨会上,一个指定的工作组审查了文献,以更新上一次研讨会(2016年)期间提出的第二项allo-HCT建议.第二次allo-HCT的主要适应症仍然是最初的血液系统恶性肿瘤的复发。疾病状态;完全缓解(CR),首次allo-HCT>6个月后的复发时间对第二次allo-HCT后患者的总生存率有积极影响。捐赠者变更是一个有效的选择,特别是如果在首次单倍体相合或错配的allo-HCT后白血病细胞上存在HLA丢失。推荐降低强度的调理,而对于增殖性疾病患者,序贯方案是一个合理的选择。即使尚未停止免疫抑制治疗,也建议在第60天进行血液学恢复后的移植后维持策略。低甲基化剂,和靶向治疗如抗FLT3,抗BCL2,抗IDH1/2,TKI,抗TP53,抗CD33,抗CD19,抗CD22,抗CD30,检查点抑制剂,和CAR-T细胞可以用作移植的桥梁或作为第二个allo-HCT的替代治疗。
    Relapse after allogeneic hematopoietic cell transplantation (allo-HCT) remains a major concern because it is associated with poor survival. A second allo-HCT is a valid option in this situation. During the 13th annual harmonization workshops of the francophone Society of bone marrow transplantation and cellular therapy (SFGM-TC), a designated working group reviewed the literature in order to update the second allo-HCT recommendations elaborated during the previous workshop (2016). The main indication for a second allo-HCT remains relapse of initial hematologic malignancy. Disease status; complete remission (CR), and relapse time after the first allo-HCT>6 months impact positively the overall survival of patients after the second allo-HCT. Donor change is a valid option, particularly if there is HLA loss on leukemic cells after a first haploidentical or following a mismatched allo-HCT is documented. Reduced intensity conditioning is recommended, while a sequential protocol is a reasonable option in patients with proliferative disease. A post-transplant maintenance strategy after hematological recovery is recommended as soon as day 60, even if the immunosuppressive treatment has not yet been stopped. Hypomethylating agents, and targeted therapies such as anti FLT3, anti BCL2, anti-IDH1/2, TKI, anti-TP53, anti-CD33, anti-CD19, anti-CD22, anti-CD30, check point inhibitors, and CAR-T cells can be used as a bridge to transplant or as an alternative treatment to the second allo-HCT.
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  • 文章类型: English Abstract
    在资源有限的国家,急性淋巴细胞白血病(ALL)患者的管理取决于预后分层的方法,可用的治疗和后勤。在法语国家骨髓移植和细胞治疗协会(SFGM-TC)的第12届年度协调研讨会上,一个指定的工作组回顾了文献,以便详细阐述针对该疾病的异基因造血细胞移植(Allo-HCT)的统一指南.常规不良预后因素可用于确定首次缓解时allo-HCT的指征。如果有的话,缺乏HLA匹配的相关供体的患者可以用单倍体相同的供体allo-HCT进行同种异体移植。如果TBI不可用,可以使用基于化疗的预处理方案。因为找到具有全身照射能力的放射治疗部门的可能性很低。对于费城染色体阳性(Phi+)ALL的患者,建议将移植后酪氨酸激酶抑制剂作为系统的维持策略。自体HCT对于微小残留病阴性的Phi+ALL患者是可选的,没有资格获得allo-HCT的人。患有难治性/复发性疾病的患者预后不良,这凸显了在未来获得新疗法的重要性,例如:blinatumumab,伊托珠单抗,和CAR-T细胞。
    Management of acute lymphoblastic leukemia (ALL) patients in countries with limited resources depends on the means of prognostic stratification, available treatment and logistics. During the 12th annual harmonization workshops of the francophone Society of bone marrow transplantation and cellular therapy (SFGM-TC), a designated working group reviewed the literature in order to elaborate unified guidelines for allogeneic hematopoietic cell transplantation (Allo-HCT) in this disease. Conventional poor prognostic factors can be used to determine the indication of allo-HCT in first remission. Patients lacking a HLA-matched related donor can be allografted with a haploidentical donor allo-HCT if available. Chemotherapy based conditioning regimen can be used if TBI is not available, because the probability to find a radiotherapy department with the capacity for total body irradiation is low. For patients with Philadelphia chromosome positive (Phi+) ALL, post-transplantation tyrosine kinase inhibitors as a systematic maintenance strategy is recommended. Autologous HCT is optional for Phi+ ALL patients with negative minimal residual disease, who not eligible for allo-HCT. Patients with refractory/relapsed disease have a poor prognosis which highlights the importance of acquiring in the future new therapies such as: blinatumumab, inotuzumab, and CAR-T cells.
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  • 文章类型: English Abstract
    In an attempt to harmonize clinical practices among francophone hematopoietic stem cell transplantation centers, the Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC) held its eleventh annual workshop series in September 2020 in Lille. This event brought together practitioners from across Europe. Our article discusses the updates and modifications for the 2021 version of the national patient follow-up care logbook.
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  • 文章类型: English Abstract
    Hematopoietic cell transplantation (HCT) is the curative treatment for many malignant and non-malignant blood disorders and some solid cancers. However, transplant procedures are considered tertiary level care requiring a high degree of technicality and expertise and generating very high costs for hospital structures in developing countries as well as for patients without health insurance. During the 11th annual harmonization workshops of the francophone Society of bone marrow transplantation and cellular therapy (SFGM-TC), a designated working group reviewed the literature in order to elaborate unified guidelines, for developing the transplant activity in emerging countries. Access to infrastructure must comply with international standards and therefore requires a hospital system already in place, capable of accommodating and supporting the HCT activity. In addition, the commitment of the state and the establishment for the financing of the project seems essential.
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  • 文章类型: Journal Article
    Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is a dysimmune neuropathy with sensory and/or motor symptoms due to destruction of the myelin sheat secondary to an auto-immune attack. A quarter to a third of patients do not respond to immunomodulatory first line recommended therapies. No second line treatment has shown its effectiveness with a sufficient level of evidence. Autologous hematopoietic stem cell transplantation (AHSCT) is a promising therapy for autoimmune disease, especially for CIDP in recent works. We present in this article an update on the diagnosis of CIDP, its conventional treatments as well as the results of AHSCT in this indication, which was the subject of French recommendations under the aegis of the SFGMTC and neuromuscular disease french faculty (FILNEMUS) as a third line therapy after failure of two first-line and one second-line treatments.
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  • 文章类型: Journal Article
    Thalassemia and sickle cell disease (SCD) are among the most common inherited diseases worldwide. Red blood cell transfusion is a cornerstone of their treatment, but its indications have significantly changed over the past years. New therapies are emerging in both syndromes: among them, hematopoietic stem cell transplantation is now routinely proposed, and gene therapy has shown promising preliminary results.
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