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  • 1
    In: Bone Marrow Transplantation, Springer Science and Business Media LLC, Vol. 56, No. 3 ( 2021-03), p. 536-543
    Abstract: Severe blood disorders and cancer are the leading cause of death and disability from noncommunicable diseases in the global pediatric population and a major financial burden. The most frequent of these conditions, namely sickle cell disease and severe thalassemia, are highly curable by blood or bone marrow transplantation (BMT) which can restore a normal health-related quality of life and be cost-effective. This position paper summarizes critical issues in extending global access to BMT based on ground experience in the start-up of several BMT units in middle-income countries (MICs) across South-East Asia and the Middle East where close to 700 allogeneic BMTs have been performed over a 10-year period. Basic requirements in terms of support systems, equipment, and consumables are summarized keeping in mind WHO’s model essential lists and recommendations. BMT unit setup and maintenance costs are summarized as well as those per transplant. Low-risk BMT is feasible and safe in MICs with outcomes comparable to high-income countries but at a fraction of the cost. This report might be of assistance to health care institutions in MICs interested in developing hematopoietic stem cell transplantation services and strengthening context appropriate tertiary care and higher medical education.
    Type of Medium: Online Resource
    ISSN: 0268-3369 , 1476-5365
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    Language: English
    Publisher: Springer Science and Business Media LLC
    Publication Date: 2021
    detail.hit.zdb_id: 2004030-1
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  • 2
    In: British Journal of Haematology, Wiley, Vol. 164, No. 3 ( 2014-02), p. 396-408
    Abstract: Eighty‐two children and adolescents who underwent allogeneic transplantation for acute lymphoblastic leukaemia in remission (period 2001–2011, median follow‐up 4·9 years) had been assessed for minimal residual disease ( MRD ) by real‐time quantitative polymerase chain reaction before and at 1, 3, 6, 9 and 12 months after transplantation. Five‐year event‐free survival ( EFS ) and cumulative incidence of relapse were 77·7% [standard error ( SE ) 5·7] and 11·4% ( SE 4·4), respectively, for patients with pre‐transplant MRD 〈 1 × 10 −4 (68%), versus 30·8% ( SE 9·1; P   〈  0·001) and 61·5% ( SE 9·5; P   〈  0·001), respectively, for those with MRD ≥1 × 10 −4 (32%). Pre‐transplant MRD ≥1 × 10 −4 was associated with a 9·2‐fold risk of relapse [95% confidence interval ( CI ) 3·54–23·88; P   〈  0·001] compared with patients with MRD 〈 1 × 10 −4 . Patients who received additional chemotherapy pre‐transplant to reduce MRD had a fivefold reduction of risk of failure (hazard ratio 0·19, CI 0·05–0·70, P  = 0·01). Patients who experienced MRD positivity post‐transplant did not necessarily relapse (5‐year EFS 40·3%, SE 9·3), but had a 2·5‐fold risk of failure ( CI 1·05–5·75; P  = 0·04) if any MRD was detected in the first 100 d, which increased to 7·8‐fold ( CI 2·2–27·78; P  = 0·002) if detected after 6 months. Anticipated immunosuppression‐tapering according to MRD may have improved outcome, nevertheless all patients with post‐transplant MRD ≥1 × 10 −3 ultimately relapsed, regardless of immunosuppression discontinuation or donor‐lymphocyte‐infusion. In conclusion, MRD before transplantation had the strongest impact on relapse and MRD positivity after transplantation, mostly if detected early and at low levels, did not necessarily imply relapse. Additional intensified chemotherapy and modulation of immunosuppression may reduce relapse risk and improve ultimate outcome.
    Type of Medium: Online Resource
    ISSN: 0007-1048 , 1365-2141
    URL: Issue
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    Language: English
    Publisher: Wiley
    Publication Date: 2014
    detail.hit.zdb_id: 1475751-5
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  • 3
    In: Blood, American Society of Hematology, Vol. 134, No. Supplement_1 ( 2019-11-13), p. 2044-2044
    Abstract: Introduction: Blood or marrow transplantation (BMT) programs are largely insufficient to meet the growing need in MICs. Where BMT is not available families, charities or governments often seek for alternative programs, usually to send children abroad for BMT; this policy is associated with many drawbacks, including costs, and difficulties to manage transplant related complications after the patient return home. Evaluation of feasibility and sustainability plays a crucial role in the success of all cooperative projects but especially of high specialization ones. Materials and methods: In 2016 the first HSCTU inclusive of a pediatric section was developed in Iraqi Kurdistan at Hiwa Cancer Hospital of Sulaymaniah, thanks to a cooperative project funded by the Italian Agency for Development Cooperation and to the availability of professionals from specialized centers to train local personnel. After verifying with an appropriate grid that essential requirements were in place, a capacity building project was started. An intensive educational course was carried on initially and then, using a training on the job approach, all specific skills were set-up and/or implemented. A complete protocol handbook was created with involvement of the local staff; a staggered approach to the disease was chosen, starting from low risk diseases and moving later to high risk ones. After 3 years project the center is now completely autonomous; up to now they have been performed 158 transplants (autologous and allogeneic, both in adult and children) with results comparable with those of high-income countries (HICs). In a cohort of 26 pediatric patients with thalassemia the event free survival (EFS) is over 90% with a median follow up of 492 days. Next to this experience, in 2018 a new project has been activated in Paraguay, following a specific request from the pediatric hospital Ninos de Acosta Nu in Asuncion. The similar methodology has been applied. After documenting the feasibility, a 3 years project has been set-up, with an initial intensive educational course, followed by training on the job by experts in the field and also limited training of the local staff abroad. A six months continuously oversight by experienced nurses and periodically by expert physicians has been planned along with the continuous support through on line contacts; focused training in Europe on minimal residual disease, chimerism, apheresis and HLA typing has been scheduled. The first allogeneic transplant from matched sibling donor is planned in autumn 2019. Discussion: Based on our experience new centers should start with patients highly curable, low-risk matched sibling transplants, well-established context-appropriate protocols with minimal toxicity, proven high success rates and contained costs. If socio-economical context of the country and needs of the population allow to move to oncological patients, a staggered approach is suggested: from matched sibling donor transplant to mismatched ones. Minimal essential requirements for a pediatric BMT start-up activity may be affordable in many MICs, if health authorities and/or local charities/foundations are committed. This shall include good quality blood products (leuco-reduced or irradiated); drugs for conditioning regimens (busulphan, tiothepa, fludarabine, cyclophosphamide, melphalan), GVHD prophylaxis and treatment (methotrexate, thymoglobuline, cyclosporine, mycophenolate mofetil), infections control (broad spectrum antibiotics, one anti-mold drug, ganciclovir) and essential laboratory tests (at least cyclosporin levels and citomegalovirus copies count). Conclusion: The strategy described above requires the availability of expert BMT physicians and nurses willing to rotate periodically in MICs Units for training on the job and supervision. This model is replicable in different contexts, allows a fast development of BMT units in MICs, and in our opinion compares favorably with that of offering training to multiple professionals in specialized centers. Figure Disclosures No relevant conflicts of interest to declare.
    Type of Medium: Online Resource
    ISSN: 0006-4971 , 1528-0020
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    Language: English
    Publisher: American Society of Hematology
    Publication Date: 2019
    detail.hit.zdb_id: 1468538-3
    detail.hit.zdb_id: 80069-7
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  • 4
    In: Bone Marrow Transplantation, Springer Science and Business Media LLC, Vol. 55, No. 10 ( 2020-10), p. 1900-1905
    Type of Medium: Online Resource
    ISSN: 0268-3369 , 1476-5365
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    Language: English
    Publisher: Springer Science and Business Media LLC
    Publication Date: 2020
    detail.hit.zdb_id: 2004030-1
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