Affiliation: University of Minnesota
- Transplantation of unrelated donor umbilical cord blood in 102 patients with malignant and nonmalignant diseases: influence of CD34 cell dose and HLA disparity on treatment-related mortality and survivalJohn E Wagner
Blood and Marrow Transplant Program of the Department of Pediatrics, University of Minnesota Cancer Center and School of Medicine, Minneapolis 55455, USA
Blood 100:1611-8. 2002..7 x 10(5) CD34(+) cells per kilogram of recipient's body weight. Therefore, graft selection should be based principally on CD34 cell dose when multiple UCB units exist with an HLA disparity of 2 or less...
- Cerebral X-linked adrenoleukodystrophy: the international hematopoietic cell transplantation experience from 1982 to 1999Charles Peters
Department of Pediatrics, University of Minnesota, Minneapolis, USA
Blood 104:881-8. 2004..In this first comprehensive report of the international HCT experience for X-ALD, we conclude that boys with early-stage disease benefit from HCT, whereas boys with advanced disease may be candidates for experimental therapies...
- Long-term outcomes of adaptive functions for children with mucopolysaccharidosis I (Hurler syndrome) treated with hematopoietic stem cell transplantationKendra J Bjoraker
Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA
J Dev Behav Pediatr 27:290-6. 2006..Although cognitive and orthopedic problems as well as medical complications limit adaptive ability, identifying these problems early allow beneficial targeted interventions...
- Inflammatory cytokines and the development of pulmonary complications after allogeneic hematopoietic cell transplantation in patients with inherited metabolic storage disordersSandhya Kharbanda
University of Minnesota Cancer Center and Department of Pediatrics, Division of Pediatric Hematology Oncology and Blood and Marrow Transplant, Minneapolis, 55455, USA
Biol Blood Marrow Transplant 12:430-7. 2006..Larger studies will be required to further elucidate etiologic mechanisms and predictive factors...
- Functional abnormalities of heparan sulfate in mucopolysaccharidosis-I are associated with defective biologic activity of FGF-2 on human multipotent progenitor cellsChendong Pan
Associate Professor of Medicine, University of Minnesota Medical School, Hematology Oncology Section 111E, VA Medical Center, One Veterans Dr, Minneapolis, MN 55417
Blood 106:1956-64. 2005..Similar mechanisms may operate in the pathogenesis of other diseases where structurally abnormal GAGs accumulate...
- Autoimmune haemolytic anaemia complicating haematopoietic cell transplantation in paediatric patients: high incidence and significant mortality in unrelated donor transplants for non-malignant diseasesTracey A O'Brien
Pediatric Hematology Oncology and Blood and Marrow Transplant Program, Medical School, University of Minnesota, Minneapolis, MN, USA
Br J Haematol 127:67-75. 2004..Alternative treatment strategies were employed, with the majority of patients demonstrating disease refractory to traditional steroid therapy...
- Predictive factors for vision loss after hematopoietic cell transplant for X-linked adrenoleukodystrophyAdam Gess
Department of Ophthalmology, University of Minnesota, Minneapolis, Minnesota 55455, USA
J AAPOS 12:273-6. 2008..Visual dysfunction is a common component of the disease and may continue to progress in patients after HCT. This study was designed to determine prognostic factors predictive of vision loss after HCT...
- Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndromeSatkiran S Grewal
Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota 55455, USA
Genet Med 7:143-6. 2005..However, morbidity and mortality associated with HSCT can limit its success. We describe the initial experience with combined use of enzyme replacement therapy (ERT, laronidase) and HSCT in Hurler syndrome...
- Mucopolysaccharidosis I: management and treatment guidelinesJoseph Muenzer
Department of Pediatrics, University of North Carolina at Chapel Hill, Chapel Hill, NC 27514, USA
Pediatrics 123:19-29. 2009..The availability of hematopoietic stem cell transplantation and the recent introduction of enzyme replacement therapy for mucopolysaccharidosis I necessitate the establishment of system-specific management guidelines for this condition...
- Effective treatment of alpha-mannosidosis by allogeneic hematopoietic stem cell transplantationSatkiran S Grewal
Division of Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN 55455, USA
J Pediatr 144:569-73. 2004..To study the efficacy of hematopoietic stem cell transplantation (HCT) for ameliorating the clinical manifestations of alpha-mannosidosis...
- Azacitidine pharmacokinetics in an adolescent patient with renal compromiseCourtney F Tsao
Department of Pharmacy, Division of Hematology Oncology, The Children s Mercy Hospital and Clinics, University of Missouri, Kansas City, MO 64108, USA
J Pediatr Hematol Oncol 29:330-3. 2007....
- Survival analysis of haematopoietic cell transplantation for childhood cerebral X-linked adrenoleukodystrophy: a comparison studyAsif Mahmood
Kennedy Krieger Institute and Department of Neurology, Johns Hopkins University, Baltimore, MD, USA
Lancet Neurol 6:687-92. 2007..We analysed survival of CCALD patients who had not received HCT and, in a subgroup with early cerebral disease, compared survival in those who underwent HCT with those who did not...
- Reconstitution of lymphocyte subpopulations in children with inherited metabolic storage diseases after haematopoietic cell transplantationPaola Corti
Clinica Pediatrica, Universita di Milano Bicocca, Ospedale San Gerardo, Monza, Italy
Br J Haematol 130:249-55. 2005..Because of the unique graft manipulation, the pace of lymphocyte reconstitution was particularly slow, suggesting that these patients are at a significantly increased risk of infections for up to 2 years after HCT...