Edwin M Horwitz

..These results suggest that BMT from HLA-compatible donors may benefit children with severe OI. Further studies are needed to determine the full potential of this strategy...

..Thus, allogeneic mesenchymal cells offer feasible posttransplantation therapy for osteogenesis imperfecta and likely other disorders originating in mesenchymal precursors...

..Both animal models to carefully address outstanding issues and pilot clinical trials to explore the therapeutic potential will be key elements to advance science for the benefit of patients...

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..To improve the efficacy of BMT for bone disorders, we sought to gain insight into the cellular mechanism of engraftment of transplantable marrow osteoprogenitors...

..The decision to perform chest CT should be made judiciously based on a careful history and physical examination. Sinus imaging, which was frequently abnormal, may be justified in all patients to plan post-HSCT care...

..Rapid T-cell reconstitution, retention of NK-cells in the graft and induction of low grade GvHD may also enhance the potential anti-cancer immune effect...

..We report our experience with cidofovir (CDV) for treatment of ADV infection in 57 HSCT patients, median age 8 years (range 0.5-26)...

..She subsequently developed disseminated PTLPD involving multiple organ and nodal sites. Her neoplastic lymphoblasts were host-derived and refractory to rituximab treatment due to lack of CD20 expression...

..Thus, GD2 appears to be the first reported single surface marker that uniquely distinguishes MSCs from other marrow elements. GD2 may prove valuable to study MSC biology and for the preparation of MSCs for clinical applications...

..To investigate the use of quantitative computed tomography (QCT) and dual energy absorptiometry (DXA) for assessing bone mineral density (BMD) in the evaluation of children with malignant infantile osteopetrosis (MIOP)...

..Using a quantitative approach, we demonstrate that MIOP children have OSA and hypoxemia; thus, these children should have airway evaluations and treatments to potentially reduce the risk of life-threatening pulmonary complications...

..With a multiple regression analysis, the use of antithymocyte globulin (beta = .86; P = .04) and recipient cytomegalovirus seronegativity (beta = .93; P = .035) remained significant risk factors for the development of HUS...

..In contrast, donor T-cell incompatibility was a risk factor for acute GVHD, chronic GVHD and death. Thus, T-cell alloreactivity dominated that of NK cells in minimally T-cell-depleted grafts...

..Its isolation and characterization may suggest novel treatments for genetic bone diseases and bone injuries...

..These interesting results and their biological and clinical implications are the focus of this comment...

..The use of Dkk-1 in cell-processing laboratories might advance cell-expansion technology and greatly increase the clinical uses of MSCs...

..To determine the incidence, etiology and outcome of graft failure in pediatric allogeneic bone marrow transplant (BMT) recipients...

..We suggest that PPH may be linked to a specific variant of MIOP and recognizing the potential for pulmonary hypertension in children with MIOP may lead to a more rapid diagnosis and life-saving intervention...

..The lack of durable donor-derived osteopoiesis may reflect an intrinsic genetic program or exogenous environmental signaling that suppresses the differentiation capacity of the donor stem cells...

..The remaining five patients seem to be heterozygous for a ClCN7 mutation, and significant variations were observed in the clinical manifestations of their disease, even within the same family...

..Collectively, the data generated by this 5-year project will help to clarify several outstanding questions in stem cell biology and the therapeutic relevance of marrow stem cells with apparent multilineage potential. ..