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- Cohen, A, et al.
(author)
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Endocrinological late complications after hematopoietic SCT in children
- 2008
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In: Bone Marrow Transplantation. - : Springer Science and Business Media LLC. - 1476-5365 .- 0268-3369. ; 41, s. 43-48
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Journal article (peer-reviewed)abstract
- The main challenge for a pediatric hemato-oncologist today is to obtain a cure for the sick child with the minimum of treatment-related complications. Children on their way to achieving adulthood face many risks after hematopoietic SCT (HSCT). Continuous follow-up includes assessment of organ function, focus on vaccinations and screening for secondary malignancies. Updated treatment protocols are already adjusted according to the knowledge obtained on late effects, and the potential risks for complications are well balanced with expected benefits hopefully resulting in decreased potential risk for organ damage but still maintaining an unchanged or improved survival rate. Recent developments on pre-HSCT regimens, such as the introduction of new anticancer regimens and immunosuppressive agents will hopefully contribute to minimize the frequency and the severity of late complications. Knowledge about increased risk for long-term complications due to cancer therapy and pre-HSCT preparative regimens should encourage each caring physician to stick to follow-up protocols and treatment guidelines not only to improve the survival rate of transplanted children but also to improve their quality of life. To achieve adulthood by maintaining cognitive ability and psychosocial skills is the highest goal for an individual to become a competent member of a society. This review of late endocrine complications after HSCT focuses on growth, pubertal development, thyroid disorders and glucose metabolism in long-term survivors.
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- Dufour, C, et al.
(author)
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TNF-alpha and IFN-gamma are overexpressed in the bone marrow of Fanconi anemia patients and TNF-alpha suppresses erythropoiesis in vitro
- 2003
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In: Blood. - : American Society of Hematology. - 1528-0020 .- 0006-4971. ; 102:6, s. 2053-2059
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Journal article (peer-reviewed)abstract
- In Fanconi anemia (FA) C mice tumor necrosis factor alpha (TNF-alpha) and interferon gamma (IFN-gamma) have key roles in the pathogenesis of bone marrow failure. In FA subjects TNF-alpha was found to be increased in the serum and overproduced by patient-derived B-cell lines. In acquired aplastic anemia, a disease in which, similarly to FA, marrow failure occurs, TNF-alpha and IFN-gamma act as late mediators of the stem cell damage and are overexpressed in patient marrow lymphocytes. This study evaluated in marrow mononuclear cells (MNCs) of patients with FA, the expression of negative modulators of the hematopoiesis, such as TNF-alpha, IFN-gamma, macrophage inflammatory protein 1alpha (MIP-1alpha), and surface Fas ligand, and the role of TNF-alpha on FA erythropoiesis in vitro. TNF-alpha and IFN-gamma were significantly overexpressed in stimulated marrow MNCs of FA patients as compared to healthy controls. MIP-1alpha and Fas ligand were undetectable in patients and controls. In bone marrow cultures, the addition of anti-TNF-alpha increased the size and significantly increased the number of erythroid colony-forming units and erythroid burst-forming units grown from FA patients but not from healthy controls. This indicates that FA subjects have a marrow TNF-alpha activity that inhibits erythropoiesis in vitro. TNF-alpha has a relevant role in the pathogenesis of erythroid failure in FA patients.
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| 9. |
- Korthof, E. T., et al.
(author)
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Management of acquired aplastic anemia in children
- 2013
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In: Bone Marrow Transplantation. - : Springer Science and Business Media LLC. - 1476-5365 .- 0268-3369. ; 48:2, s. 191-195
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Research review (peer-reviewed)abstract
- The diagnosis of aplastic anemia in children requires exclusion of a variety of inherited or acquired BM failure syndromes with similar phenotypes. An efficient diagnostic plan is important because time from diagnosis to 'final' treatment is directly related to outcome regardless of the therapeutic option chosen. The gold standard of therapy remains hematopoietic SCT with a graft of BM cells for those children with matched sibling donors. Conversely for children without a sibling donor the high response and markedly improved overall survival rates of combined immunosuppressive therapy have proven robust, especially when horse derived anti-thymocyte globuline plus ciclosporine A are used. Incomplete response, relapse and progression to myelodysplasia/leukemia however have emerged as significant long-term issues. Improvements in outcome of alternative donor transplantation and the use of established and novel immunosuppressive agents provide multiple alternatives for treating refractory or relapsed patients. Regardless of the type of therapeutic approach, patients require centralized treatment in a center of excellence, ongoing monitoring for recurrence of disease and/or therapy-related immediate side effects and long-ternn effects. Bone Marrow Transplantation (2013) 48, 191-195; doi:10.1038/bmt.2012.235; published online 7 January 2013
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| 10. |
- Ljungman, P., et al.
(author)
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Long-term follow-up of HCV-infected hematopoietic SCT patients and effects of antiviral therapy
- 2012
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In: Bone Marrow Transplantation. - : Springer Science and Business Media LLC. - 1476-5365 .- 0268-3369. ; 47:9, s. 1217-1221
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Journal article (peer-reviewed)abstract
- This prospective study was initiated in 1993 with the aim to study late effects and responses to antiviral therapy in a cohort of hepatitis C virus (HCV)-infected patients. A total of 195 patients were included from 12 centers. In all, 134 patients had undergone allogeneic and 61 autologous hematopoietic SCT (HSCT). The median follow-up from HSCT is currently 16.8 years and the maximum 27.2 years. Overall 33 of 195 patients have died of which 6 died from liver complications. The survival probability was 81.6% and the cumulative incidence for death in liver complications was 6.1% at 20 years after HSCT. The cumulative incidence of severe liver complications (death from liver failure, cirrhosis and liver transplantation) was 11.7% at 20 years after HSCT. In all, 85 patients have been treated with IFN; 42 in combination with ribavirin. The sustained response rate was 40%. The rates of severe side effects were comparable to other patient populations and no patient developed significant exacerbations of GVHD. Patients receiving antiviral therapy had a trend toward a decreased risk of severe liver complications (odds ratio=0.33; P=0.058). HCV infection is associated with morbidity and mortality in long-term survivors after HSCT. Antiviral therapy can be given safely and might reduce the risk for severe complications.
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