Reduced intensity and non-myeloablative allogeneic stem cell transplantation in children and adolescents with malignant and non-malignant diseases

Department of Pediatrics, Columbia University, New York, New York, USA.
Pediatric Blood & Cancer (Impact Factor: 2.39). 01/2008; 50(1):1-8. DOI: 10.1002/pbc.21303
Source: PubMed


Allogeneic hematopoietic stem cell transplant (AlloSCT) from related or unrelated histocompatible donors has been well established as potentially curative therapy for children and adolescents with selected malignant and non-malignant diseases. In the malignant setting non-myeloablative (NMA)/reduced intensity (RI)-AlloSCT eradicates malignant cells through a graft versus malignancy effect provided by alloreactive donor T-lymphocytes and/or natural killer cells. In patients with non-malignant diseases NMA/RI AlloSCT provides enough immunosuppression to promote engraftment and correct underlying genetic defects. In children, myeloablative AlloSCT is not only associated with acute short-term toxicities but also long-term late complications such as growth retardation, infertility, and secondary malignancies. NMA/RI-AlloSCT in children may be associated with reduction in use of blood products, risk of infections, transplant-related mortality, and length of hospitalization. Despite the success of RI-AlloSCT in adults, large prospective and/or randomized multicenter studies are necessary in children and adolescent recipients to define the appropriate patient population, optimal conditioning regimens, cost-benefits, survival and differences in short-term and long-term effects compared to conventional myeloablative conditioning.

Full-text preview

Available from:
  • [Show abstract] [Hide abstract]
    ABSTRACT: Public health care has to make use of the potentials of IT to meet the enormous demands on patient management in the future. Embedding artificial intelligence in medicine may lead to an increase in quality and safety. One possibility in this respect is an expert system. Conditions for an expert system are structured data sources to extract relevant data for the proposed decision. Therefore the demonstrator ‘allo-tool’ was designed. The concept of introducing a ‘Medical decision support system based on the model of Stem Cell Transplantation’ was developed afterwards. The objectives of the system are (1) to improve patient safety (2) to support patient autonomy and (3) to optimize the workflow of medical personnel.
    No preview · Conference Paper · Jan 2007
  • [Show abstract] [Hide abstract]
    ABSTRACT: Hatakeyama N, Hori T, Yamamoto M, Inazawa N, Hirako Y, Tsutsumi H, Suzuki N. Successful treatment of refractory Langerhans cell histiocytosis with pulmonary aspergillosis by reduced-intensity conditioning cord blood transplantation. Pediatr Transplantation 2010: 14: E4–E10. © 2009 Wiley Periodicals, Inc. Abstract: The prognosis of multisystem LCH in children with risk organ involvement is extremely poor when they fail to respond to conventional chemotherapy. In such patients, allogeneic SCT may produce complete and sustained remission; however, high-dose myeloablative regimens are frequently associated with treatment-related morbidity and mortality. More recently, allogeneic SCT following an RIC regimen has been performed as an alternative salvage approach. We describe a nine-month-old boy with refractory multisystem LCH with pulmonary aspergillosis who was successfully treated with reduced-intensity cord blood transplantation.
    No preview · Article · Feb 2009 · Pediatric Transplantation
  • [Show abstract] [Hide abstract]
    ABSTRACT: Tremendous progress has been made over the past two decades in understanding the molecular genetics of heritable skin diseases. The paradigm for such conditions is epidermolysis bullosa (EB), which comprises a group of heritable blistering disorders caused by mutations in ten genes expressed in the cutaneous basement membrane zone and has high morbidity and mortality. Identification of distinct mutations has improved the diagnosis and subclassification of EB, leading to improvements in disease prognosis, and has provided a basis for prenatal and pre-implantation genetic diagnosis for this disorder. Nevertheless, there is no cure or effective treatment for EB. Here, we review recent exciting developments in the areas of molecular therapies, including gene therapy, protein replacement therapy and bone-marrow-derived stem cell transfer, as potential new avenues to treat EB and other currently intractable heritable skin diseases.
    No preview · Article · Aug 2009 · Trends in Molecular Medicine
Show more