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Bone Marrow Transplantation Aug 2019Based on preclinical studies, combined kidney and hematopoietic cell transplantation was performed on fully HLA matched and haplotype matched patients at the Stanford... (Review)
Review
Based on preclinical studies, combined kidney and hematopoietic cell transplantation was performed on fully HLA matched and haplotype matched patients at the Stanford University Medical Center. The object of the studies was to induce mixed chimerism, immune tolerance, and complete immunosuppressive drug withdrawal. Tolerance, persistent mixed chimerism, and complete withdrawal was achieved in the majority of fully matched patients. Persistent mixed chimerism and partial withdrawal has been achieved in the haplotype matched patients at present.
Topics: Chimerism; Hematopoietic Stem Cell Transplantation; Humans; Kidney Transplantation; Transplantation Conditioning
PubMed: 31431706
DOI: 10.1038/s41409-019-0603-4 -
Veterinary and Comparative Oncology Dec 2020The development of safe and reliable haematopoietic cell transplantation (HCT) protocols to treat human patients with malignant and non-malignant blood disorders was... (Review)
Review
The development of safe and reliable haematopoietic cell transplantation (HCT) protocols to treat human patients with malignant and non-malignant blood disorders was highly influenced by preclinical studies obtained in random-bred canines. The surmounted barriers included recognizing the crucial importance of histocompatibility matching, establishing long-term donor haematopoietic cell engraftment, preventing graft-vs-host disease and advancing effective conditioning and post-grafting immunosuppression protocols, all of which were evaluated in canines. Recent studies have applied the tolerance inducing potential of HCT to solid organ and vascularized composite tissue transplantation. Several advances in HCT and tolerance induction that were first developed in the canine preclinical model and subsequently applied to human patients are now being recruited into veterinary practice for the treatment of malignant and non-malignant disorders in companion dogs. Here, we review recent HCT advancements attained in the canine model during the past 15 years.
Topics: Animals; Bone Marrow Transplantation; Dog Diseases; Dogs; Graft vs Host Disease; Hematopoietic Stem Cell Transplantation; Neoplasms; Transplantation Conditioning
PubMed: 32385957
DOI: 10.1111/vco.12608 -
Comptes Rendus Biologies Mar 2013Hematopoietic stem cell transplantation (HSCT) is the one and only curative therapy available for patient with severe sickle cell disease (SCD). Until today, several... (Review)
Review
Hematopoietic stem cell transplantation (HSCT) is the one and only curative therapy available for patient with severe sickle cell disease (SCD). Until today, several hundreds of patients have undergone geno-identical HSCT. More than 200 patients were transplanted in France. The first indication was cerebral vasculopathy. Among both malignant and non-malignant diseases treated with HSCT, the success rate obtained in SCD patients appears as the best one. From the year 2000, more than 95% of transplanted patients survived the HSCT procedure and more than 90% are completely cured and experience a very satisfying health condition post-transplantation. However, the current standard procedure includes a myeloablative conditioning regimen for warranting engraftment. Such regime is linked to severe long-term side effects such as hypofertility. Due to the excellent obtained results, we have to think about a possible widening of indications, a decrease of conditioning intensity and toxicity, and about HSCT from alternative stem cell sources, such as mismatch family donor, unrelated volunteer donor or unrelated cord blood.
Topics: Adolescent; Anemia, Sickle Cell; Bone Marrow Transplantation; Child; Child, Preschool; Cord Blood Stem Cell Transplantation; Fertility Preservation; France; Hematopoietic Stem Cell Transplantation; Histocompatibility; Humans; Immunosuppressive Agents; Infant; Living Donors; Myeloablative Agonists; Quality of Life; Siblings; Transplantation Conditioning; Treatment Outcome
PubMed: 23643397
DOI: 10.1016/j.crvi.2012.09.004 -
Chinese Clinical Oncology Apr 2024
Topics: Humans; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Transplantation Conditioning
PubMed: 38372061
DOI: 10.21037/cco-23-115 -
Blood Reviews Nov 2014Blood and marrow transplantation is a curative therapy for patients with sickle cell disease yet this option is seldom used. Clinical studies have shown however that... (Review)
Review
Blood and marrow transplantation is a curative therapy for patients with sickle cell disease yet this option is seldom used. Clinical studies have shown however that children transplanted for this condition can achieve excellent results. In children with sickle cell disease transplanted following conditioning with busulfan, cyclophosphamide, and anti-thymocyte globulin, cure rates in excess of 80% can be obtained when an HLA-matched sibling is used as the donor. However, the large majority of patients with sickle cell disease will not have such a donor, or will not be able to tolerate high dose conditioning regimens. Therefore novel approaches such as non-myeloablative regimes, and alternative donors such as haploidentical, unrelated, or cord blood grafts are currently being explored in clinical trials. Recent reports on non-myeloablative conditioning (HLA-matched or haploidentical donors) highlight the safety and efficacy of these approaches with low mortality and high efficacy suggesting that in the near future non-myeloablation could be the preferred type of conditioning and donor availability will not be a barrier anymore to proceed to transplant. This review will focus on the results obtained when bone marrow transplants are used to treat sickle cell disease and will discuss the results obtained with these novel approaches.
Topics: Anemia, Sickle Cell; Bone Marrow Transplantation; Humans; Transplantation Conditioning
PubMed: 25217413
DOI: 10.1016/j.blre.2014.08.001 -
Nephron 2015Chimerism is a state in which bone marrow hematopoietic stem cells from two genetically different animals coexist. To date, the approach has been used successfully to... (Review)
Review
Chimerism is a state in which bone marrow hematopoietic stem cells from two genetically different animals coexist. To date, the approach has been used successfully to induce the state of immunologic tolerance in the animal models and is now being evaluated in clinical trials of both HLA-identical and HLA-mismatched living-donor kidney transplant recipients in some transplant centers with varying degrees of success. Although the results are promising, the current conditioning regimens are not optimal and longer-term follow up and multicenter studies are needed to ensure the efficacy and safety of the procedures.
Topics: Animals; HLA Antigens; Hematopoietic Stem Cells; Humans; Immunosuppressive Agents; Kidney Transplantation; Living Donors; Transplantation Chimera; Transplantation Conditioning; Transplantation Tolerance
PubMed: 25531026
DOI: 10.1159/000369311 -
Current Opinion in Oncology Jun 2009The development of non-myeloablative and reduced-intensity conditioning regimens has enabled older or medically infirm patients with myeloid malignancies to be treated... (Review)
Review
The development of non-myeloablative and reduced-intensity conditioning regimens has enabled older or medically infirm patients with myeloid malignancies to be treated with allogeneic hematopoietic cell transplantation (HCT). The regimens are sufficiently immunosuppressive to allow engraftment of allogeneic cells and they rely largely on graft-versus-leukemia effects rather than high-dose cytotoxic therapy to eliminate malignant cells. Overall 2-5-year survivals after allogeneic HCT in older patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) have ranged from 25% to 64%. Outcomes were comparable for human leukocyte antigen-matched related and unrelated grafts. The best results were seen in patients transplanted in the first or second remission. Relapse and progressive disease continue to be problems, particularly in patients with large tumor burdens at the time of HCT. Reduction of the tumor burden before HCT with targeted therapy such as radiolabelled anti-CD45 antibody may improve the outcome. Despite still existing problems, early results in elderly patients with AML/MDS have been encouraging.
Topics: Aged; Graft vs Leukemia Effect; Hematopoietic Stem Cell Transplantation; Humans; Leukemia, Myeloid, Acute; Myelodysplastic Syndromes; Transplantation Conditioning; Treatment Outcome
PubMed: 19561410
DOI: 10.1097/01.cco.0000357467.45843.ba -
Cells Feb 2022Moving from indication to transplantation is a critical process in myelofibrosis. Most of guidelines specifically focus on either myelofibrosis disease or transplant... (Review)
Review
Moving from indication to transplantation is a critical process in myelofibrosis. Most of guidelines specifically focus on either myelofibrosis disease or transplant procedure, and, currently, no distinct indication for the management of MF candidates to transplant is available. Nevertheless, this period of time is crucial for the transplant outcome because engraftment, non-relapse mortality, and relapse incidence are greatly dependent upon the pre-transplant management. Based on these premises, in this review, we will go through the path of identification of the MF patients suitable for a transplant, by using disease-specific prognostic scores, and the evaluation of eligibility for a transplant, based on performance, comorbidity, and other combined tools. Then, we will focus on the process of donor and conditioning regimens' choice. The pre-transplant management of splenomegaly and constitutional symptoms, cytopenias, iron overload and transplant timing will be comprehensively discussed. The principal aim of this review is, therefore, to give a practical guidance for managing MF patients who are potential candidates for allo-HCT.
Topics: Aged; Aged, 80 and over; Hematopoietic Stem Cell Transplantation; Humans; Primary Myelofibrosis; Transplantation Conditioning; Transplantation, Homologous
PubMed: 35159362
DOI: 10.3390/cells11030553 -
Current Opinion in Hematology Nov 2013The only current treatment capable of curing patients with myelodysplastic syndromes (MDS) is allogeneic haematopoietic stem cell transplantation (HCT). However, many... (Review)
Review
PURPOSE OF REVIEW
The only current treatment capable of curing patients with myelodysplastic syndromes (MDS) is allogeneic haematopoietic stem cell transplantation (HCT). However, many MDS patients are older, often with substantial comorbid conditions, and the disease is heterogeneous. As a consequence, results of HCT vary considerably, and the practices of HCT for MDS are evolving.
RECENT FINDINGS
The newly published modified International Prognostic Scoring System (IPSS-R), developed for nontransplanted patients, also correlates with post-HCT outcome, with the patient's karyotype having the strongest impact. The presence of monosomal karyotype and various genetic and molecular markers have also been shown to have a prognostic value. The use of hypomethylating agents, before or after HCT, may reduce the post-HCT relapse risk or delay relapse. Low and reduced-intensity conditioning regimens have allowed to transplant growing numbers of older patients with MDS, and the development of novel regimens may lead to improved relapse-free survival even in patients with high-risk cytogenetics. The optimal stem cell source may differ for different patient populations and different disease risk categories.
SUMMARY
Transplant results for MDS have improved in recent years. Some patients even in the eighth decade of life have been transplanted successfully. Ongoing studies are aimed at further reducing transplant-related toxicity, graft-versus-host disease and post-HCT relapse.
Topics: Age Factors; Comorbidity; Graft vs Host Disease; Hematopoietic Stem Cell Transplantation; Humans; Karyotype; Myelodysplastic Syndromes; Prognosis; Secondary Prevention; Transplantation Conditioning
PubMed: 24104409
DOI: 10.1097/MOH.0b013e328364f547 -
Pediatric Rheumatology Online Journal Jul 2022Mevalonate kinase deficiency (MKD) is a rare autoinflammatory syndrome. Several reports have described allogeneic hematopoietic stem cell transplantation in severely...
OBJECTIVES
Mevalonate kinase deficiency (MKD) is a rare autoinflammatory syndrome. Several reports have described allogeneic hematopoietic stem cell transplantation in severely affected patients, sometimes with promising results. In view of the scarcity of data, this study aims to analyse the efficacy and safety of allogeneic hematopoietic stem cell transplantation (HSCT) to give a more complete overview of this treatment.
METHODS
This multicentre retrospective study on behalf of the European Society for Blood and Marrow Transplantation aimed to include all MKD patients who had undergone allogeneic HSCT. All centres related to EMBT and centres that have reported cases of allogeneic HSCT in the literature were contacted via the EBMT data office.
RESULTS
We analyzed 9 patients (5 male). Treosulfan based conditioning was the most frequently used conditioning regimen. Engraftment occurred in all but one patient. Source of stem cells was cord blood (n = 2), peripheral blood stem cells (n = 4) and bone marrow (n = 5). Two patients needed a second transplantation due to an incomplete response or primary graft failure. Seven patients went into complete remission after stem cell transplantation. At final follow-up these patients reported no symptoms of MKD. Four patients suffered from grade II-IV acute graft-versus-host disease (GvHD). During follow-up two patients died due to transplantation related complications.
CONCLUSION
In conclusion, allogeneic stem cell transplantation represents an effective treatment for the most severely affected MKD patients. However, treatment-related morbidity and mortality are significant. Transplantation may be justified in patients with a severe disease course on conservative therapy.
Topics: Graft vs Host Disease; Hematopoietic Stem Cell Transplantation; Humans; Male; Mevalonate Kinase Deficiency; Retrospective Studies; Transplantation Conditioning
PubMed: 35906690
DOI: 10.1186/s12969-022-00716-4