Individualized conditioning regimes in cord blood transplantation: Towards improved and predictable safety and efficacy

Admiraal, Rick; Boelens, Jaap Jan

doi:10.1517/14712598.2016.1164688

Cited by 12 publications

(6 citation statements)

References 102 publications

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“…This method has been applied to study the population PKs of fludarabine and clofarabine [ 33 , 34 , 35 ]. The combination of busulfan and fludarabine, with or without clofarabine, is currently emerging as an HCT conditioning regimen [ 36 ]. In this study, we also performed HCT conditioning by combing busulfan with fludarabine; therefore, we decided to adopt Punt’s method [ 28 ] and focus on busulfan TDM.…”

Section: Resultsmentioning

confidence: 99%

Therapeutic Drug Monitoring of Busulfan in Patients Undergoing Hematopoietic Cell Transplantation: A Pilot Single-Center Study in Taiwan

et al. 2021

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(1) Background: Busulfan has been used as a conditioning regimen in allogeneic hematopoietic cell stem transplantation (HSCT). Owing to a large inter-individual variation in pharmacokinetics, therapeutic drug monitoring (TDM)-guided busulfan dosing is necessary to reduce graft failure and relapse rate. As there exists no TDM of busulfan administration for HCT in Taiwan, we conducted a pilot study to assess the TDM-dosing of busulfan in the Taiwanese population; (2) Methods: Seven patients with HCT from The Koo Foundation Sun Yat-Sen Cancer Center, Taipei, Taiwan, received conditioning regimens consisting of intravenous busulfan and other chemotherapies. After the initial busulfan dose, blood samples were collected for busulfan TDM at 5 min, 1 h, 2 h, and 3 h. Busulfan was extracted and detected by performing stable-isotope dilution LC–MS/MS. Plasma busulfan concentration was quantified and used for dose adjustment. Potential adverse effects of busulfan, such as mucositis and hepatic veno-occlusive disease (VOD), were also evaluated; (3) Results: The LC–MS/MS method was validated with an analyte recovery of 88–99%, within-run and between-run precision of <15%, and linearity ranging from 10 to 10,000 ng/mL. Using TDM-guided busulfan dosing, dose adjustment was necessary and performed in six out of seven patients (86%) with successful engraftments in all patients (100%). Mild mucositis was observed, and VOD was diagnosed in only one patient; (4) Conclusions: This single-center study in Taiwan demonstrated the importance of busulfan TDM in increasing the success rate of HCT transplantation. It is also necessary to further investigate the optimal busulfan target value in the Taiwanese population in the future.

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Section: Resultsmentioning

confidence: 99%

Therapeutic Drug Monitoring of Busulfan in Patients Undergoing Hematopoietic Cell Transplantation: A Pilot Single-Center Study in Taiwan

et al. 2021

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“…The impact of ATG on immunity is also influenced by the total dose, timing of administration in relation to transplantation, and peritransplant host lymphocyte count. Higher doses of ATG, timing close to transplantation, and lower host total lymphocyte count can all lead to persistent ATG exposure after the infusion of donor T cells and subsequently to the downstream effects of donor T cell depletion, increasing the potential for relapse, infections, and post-transplant lymphoproliferative disorders (61)(62)(63)(64)(65)(66). Thus, these factors must be considered when evaluating outcomes after ATG.…”

Section: Anti-thymocyte Globulinmentioning

confidence: 99%

Controversies and expectations for the prevention of GVHD: A biological and clinical perspective

Watkins

Williams

2022

Front. Immunol.

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Severe acute and chronic graft versus host disease (GVHD) remains a major cause of morbidity and mortality after allogeneic hematopoietic cell transplantation. Historically, cord blood and matched sibling transplantation has been associated with the lowest rates of GVHD. Newer methods have modified the lymphocyte components to minimize alloimmunity, including: anti-thymocyte globulin, post-transplant cyclophosphamide, alpha/beta T cell depletion, and abatacept. These agents have shown promise in reducing severe GVHD, however, can be associated with increased risks of relapse, graft failure, infections, and delayed immune reconstitution. Nonetheless, these GVHD prophylaxis strategies have permitted expansion of donor sources, especially critical for those of non-Caucasian decent who previously lacked transplant options. This review will focus on the biologic mechanisms driving GVHD, the method by which each agent impacts these activated pathways, and the clinical consequences of these modern prophylaxis approaches. In addition, emerging novel targeted strategies will be described. These GVHD prophylaxis approaches have revolutionized our ability to increase access to transplant and have provided important insights into the biology of GVHD and immune reconstitution.

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“…Recent expert guidelines, 115 opinions, 36 and pharmacokinetic studies on conditioning drugs as well as accurate analysis of immune reconstitution post transplant support the development of more and more individualised therapy regimens, thereby promising further reduction of the risk of severe transplant-related morbidity and mortality. 8,[130][131][132][133][134][135][136]…”

Section: Transplant Technique and Supportive Carementioning

confidence: 99%

Haematopoietic stem cell transplantation for severe autoimmune diseases in children: A review of current literature, registry activity and future directions on behalf of the autoimmune diseases and paediatric diseases working parties of the European Society for Blood and Marrow Transplantation

et al. 2022

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Although modern clinical management strategies have improved the outcome of paediatric patients with severe autoimmune and inflammatory diseases over recent decades, a proportion will experience ongoing or recurrent/relapsing disease activity despite multiple therapies often leading to irreversible organ damage, and compromised quality of life, growth/development and long-term survival. Autologous and allogeneic haematopoietic stem cell transplantation (HSCT) have been used successfully to induce disease control and often apparent cure of severe treatment-refractory autoimmune diseases (ADs) in children. However, transplant-related outcomes are disease-dependent and long-term outcome data are limited in respect to efficacy and safety. Moreover, balancing risks of HSCT against AD prognosis with continually evolving non-transplant options is challenging. This review appraises published literature on HSCT strategies and outcomes in individual paediatric ADs. We also provide a summary of the European Society for Blood and Marrow Transplantation (EBMT) Registry, where 343 HSCT procedures (176 autologous and 167 allogeneic) have been reported in 326 children (<18 years) for a range of AD indications. HSCT is a promising treatment modality, with potential long-term disease control or cure, but therapy-related morbidity and mortality need to be reduced. Further research is warranted to establish the position of HSCT in paediatric ADs via registries and prospective clinical studies to support evidence-based interspeciality guidelines and recommendations. K E Y W O R D S autoimmune diseases, haematopoietic stem cell transplantation, paediatric Selim Corbacioglu and Raffaella Greco contributed equally.

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Individualized conditioning regimes in cord blood transplantation: Towards improved and predictable safety and efficacy

Cited by 12 publications

References 102 publications

Therapeutic Drug Monitoring of Busulfan in Patients Undergoing Hematopoietic Cell Transplantation: A Pilot Single-Center Study in Taiwan

Therapeutic Drug Monitoring of Busulfan in Patients Undergoing Hematopoietic Cell Transplantation: A Pilot Single-Center Study in Taiwan

Controversies and expectations for the prevention of GVHD: A biological and clinical perspective

Haematopoietic stem cell transplantation for severe autoimmune diseases in children: A review of current literature, registry activity and future directions on behalf of the autoimmune diseases and paediatric diseases working parties of the European Society for Blood and Marrow Transplantation

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