Research Topics
| P HarmatzSummaryAffiliation: Children's Hospital and Research Center Country: USA Publications
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Detail Information
Publications
Oxidative stress and inflammation in iron-overloaded patients with beta-thalassaemia or sickle cell diseasePatrick B Walter
Children's Hospital Oakland Research Institute, Oakland, CA 94609, USA
Br J Haematol 135:254-63. 2006..This supports the hypothesis that the biology of SCD may show increased inflammation and increased levels of protective antioxidants compared with thalassaemia...- Mucopolysaccharidosis VIVassili Valayannopoulos
Reference Center for Inherited Metabolic Diseases, Necker Enfants Malades Hospital, Paris, France
Orphanet J Rare Dis 5:5. 2010..Prognosis is variable depending on the age of onset, rate of disease progression, age at initiation of ERT and on the quality of the medical care provided... - Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfatasePaul Harmatz
Children s Hospital and Research Center Oakland, CA 94609, USA
J Inherit Metab Dis 33:51-60. 2010..Thus, analysis of mean percent change data and longitudinal modeling both indicate that long-term ERT resulted in improvement in pulmonary function in MPS VI patients... 
Safety and efficacy of pegylated interferon alpha-2a and ribavirin for the treatment of hepatitis C in patients with thalassemiaPaul Harmatz
Children s Hospital and Research Center Oakland, 747 52 Street, Oakland, CA 94609, USA
Haematologica 93:1247-51. 2008..The molar efficacy of deferoxamine improved with reduction in liver inflammation on biopsy (p=0.001). In conclusion, antiviral treatment is safe if transfusion requirement, iron toxicities and neutropenia are monitored...- Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatasePaul Harmatz
Children s Hospital and Research Center Oakland, 747 52nd Street, Oakland, CA 94609, USA
Mol Genet Metab 94:469-75. 2008..Only 560 of 4121 reported AEs (14%) were related to treatment with only 10 of 560 (2%) described as severe. CONCLUSION: rhASB treatment up to 5 years results in sustained improvements in endurance and has an acceptable safety profile... 
Phase Ib clinical trial of starch-conjugated deferoxamine (40SD02): a novel long-acting iron chelatorPaul Harmatz
Department of Gastroenterology, The Children s Hospital and Research Center Oakland, CA, USA
Br J Haematol 138:374-81. 2007..79-1.90 mg/kg) over 1 week, with residual iron-binding capacity present in the plasma for over 6 d. In summary, treatment with S-DFO, administered weekly, has the potential to achieve iron balance in the poorly compliant patient...- Severity of iron overload in patients with sickle cell disease receiving chronic red blood cell transfusion therapyP Harmatz
Departments of Gastroenterology, Hematology Oncology, and Pathology, Children s Hospital Oakland, Oakland, CA 94609, USA
Blood 96:76-9. 2000..Despite high levels of liver iron, the associated liver injury was not severe... - Enzyme replacement therapy for mucopolysaccharidosis VI: a phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-lPaul Harmatz
Children s Hospital and Research Center at Oakland, California 94609, USA, and Children s Hospital, University of Mainz, Germany
J Pediatr 148:533-539. 2006.... - Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)Paul Harmatz
Pediatric Clinical Research Center, Children s Hospital and Research Center at Oakland, California 94606, USA
J Pediatr 144:574-80. 2004..To evaluate the safety and efficacy of weekly treatment with human recombinant N-acetylgalactosamine 4-sulfatase (rhASB) in humans with mucopolysaccharidosis type VI (MPS VI)... - Pharmacokinetic profile of recombinant human N-acetylgalactosamine 4-sulphatase enzyme replacement therapy in patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): a phase I/II studyP Harmatz
Pediatric Clinical Research Center, Hospital and Research Center at Oakland, Oakland, CA 94609, USA
Acta Paediatr Suppl 94:61-8; discussion 57. 2005..2 and 1.0 mg/kg/week) phase I/II study (n = 7) followed by an open-label single dose (1.0 mg/kg/week) extension study. We report the pharmacokinetic profile of rhASB and the impact of antibody development... - Direct comparison of measures of endurance, mobility, and joint function during enzyme-replacement therapy of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): results after 48 weeks in a phase 2 open-label clinical study of recombinant human N-acetylgaPaul Harmatz
Children s Hospital and Research Center at Oakland, 747 52nd St, Oakland, CA 94609, USA
Pediatrics 115:e681-9. 2005.... - Treatment of hepatitis C virus infection in thalassemiaEllen Butensky
Department of Gastroenterology and Nutrition, Children's Hospital and Research Center at Oakland, 747 52nd St, Oakland, CA 94609, USA
Ann N Y Acad Sci 1054:290-9. 2005..Although transfusion requirements increased in most patients, iron burden was not necessarily increased... - Elevated exhaled carbon monoxide concentration in hemoglobinopathies and its relation to red blood cell transfusion therapyEllen Butensky James
Department of Gastroenterology, Children s Hospital and Research Center Oakland, Oakland, California 94609, USA
Pediatr Hematol Oncol 27:112-21. 2010.... - Iron homeostasis during transfusional iron overload in beta-thalassemia and sickle cell disease: changes in iron regulatory protein, hepcidin, and ferritin expressionZandra A Jenkins
Council for BioIron at CHORI, Children s Hospital Oakland Research Institute, Oakland, California 94609, USA
Pediatr Hematol Oncol 24:237-43. 2007..Finally, the absence of a detectable change in ferritin mRNA indicates insufficient oxidative stress to significantly activate MARE/ARE promoters... - Inflammation and oxidant-stress in beta-thalassemia patients treated with iron chelators deferasirox (ICL670) or deferoxamine: an ancillary study of the Novartis CICL670A0107 trialPatrick B Walter
Children s Hospital and Research Center Oakland, 747 52nd Street, Oakland, CA 94609, USA
Haematologica 93:817-25. 2008..We assessed whether oxidant-stress and inflammation in beta-thalassemia could be controlled by the novel oral iron chelator deferasirox as effectively as by deferoxamine... - Potential mechanisms for altered iron metabolism in human immunodeficiency virus diseaseEllen Butensky
Department of Gastroenterology and Nutrition, Children's Hospital and Research Center, Oakland, California, USA
J Assoc Nurses AIDS Care 15:31-45. 2004..Implications for clinical practice and recommendations for future research are discussed... - Altered iron metabolism in children with human immunodeficiency virus diseaseEllen Butensky James
Department of Gastroenterology and Nutrition, Children s Hospital and Research Center Oakland, 747 52nd Street, Oakland, CA 94609, USA
Pediatr Hematol Oncol 26:69-84. 2009..Despite the high prevalence of altered iron metabolism in children with human immunodeficiency virus (HIV) disease, these alterations have not been well studied... - Morbidity and mortality in chronically transfused subjects with thalassemia and sickle cell disease: A report from the multi-center study of iron overloadEllen B Fung
Department of Hematology, Children s Hospital and Research Center, Oakland, California, USA
Am J Hematol 82:255-65. 2007..Long term follow-up of this unique cohort of subjects will be helpful in further defining the relationship of chronic, heavy iron overload to morbidity and mortality... - A simple model to assess and improve adherence to iron chelation therapy with deferoxamine in patients with thalassemiaZahra Pakbaz
Children s Hospital and Research Center at Oakland, 747 52nd St, Oakland, CA 94609, USA
Ann N Y Acad Sci 1054:486-91. 2005..Education and frequent noninvasive LIC assessments can improve adherence and iron burden. Elevated LIC does not necessarily reflect concurrent noncompliance; however, it can be an indication of nonadherence in the past... - Monitoring long-term efficacy of iron chelation treatment with biomagnetic liver susceptometryRoland Fischer
University Medical Center Hamburg Eppendorf, Department of Molecular Cell Biology, Hamburg, Germany
Ann N Y Acad Sci 1054:350-7. 2005..Because liver iron measurements by biosusceptometry are now regularly available in Europe and America, as well as quantitative MRI worldwide, these methods may be used to adjust chelation treatment regimens to body iron stores... - Comparison of organ dysfunction in transfused patients with SCD or beta thalassemiaElliott Vichinsky
Department of Hematology/Oncology, Children's Hospital and Research Center at Oakland, CA 94609, USA
Am J Hematol 80:70-4. 2005..A prospective study with a larger number of subjects is needed to confirm the relationships between specific diagnosis, liver iron concentration over time, and organ dysfunction... - Serum ferritin underestimates liver iron concentration in transfusion independent thalassemia patients as compared to regularly transfused thalassemia and sickle cell patientsZahra Pakbaz
Children s Hospital and Research Center Oakland, Oakland, California 94609, USA, and University Medical Center Hamburg Eppendorf, Hamburg, Germany
Pediatr Blood Cancer 49:329-32. 2007..32, 0.87, and 1.2, respectively: P<0.001). Due to underestimation of LIC by ferritin levels, chelation treatment may be delayed or misdirected in patients with thalassemia intermedia... - The new SQUID biosusceptometer at Oakland: first year of experienceE B Fung
Children's Hospital and Research Center, Oakland, CA 94609, USA
Neurol Clin Neurophysiol 2004:5. 2004..The new SQUID Ferritometer located on the US West coast will give more patients access to this non-invasive liver iron assessment... - Variability in hepatic iron concentration in percutaneous needle biopsy specimens from patients with transfusional hemosiderosisEllen Butensky
Department of Gastroenterology and Nutrition, Children's Hospital and Research Center at Oakland, CA 94609, USA
Am J Clin Pathol 123:146-52. 2005..0%. We found high agreement in LICs between liver biopsy specimens processed by the paraffin-embedding technique but overestimation of LICs in comparison with desiccated fresh tissue samples... - Iron metabolism and iron chelation in sickle cell diseasePatrick B Walter
Children s Hospital and Research Center Oakland, Oakland, Calif, USA
Acta Haematol 122:174-83. 2009..This makes the tissues that retain iron different in SCD, and thus the organs that fail in SCD are different from those of other hemoglobinopathies, such as the cardiomyopathy or endocrinopathies of thalassemia... - Reversed papilledema in an MPS VI patient with galsulfase (Naglazyme) therapySelim T Koseoglu
Children s Hospital Oakland, 5275 Claremont Avenue, Oakland, CA 94618, USA
Int Ophthalmol 29:267-9. 2009..J Pediatr 148(4), 533-539, 2006)... - Mutational analysis of 105 mucopolysaccharidosis type VI patientsLitsa Karageorgos
Lysosomal Diseases Research Unit, Department of Genetic Medicine, Children, Youth and Women s Health Service, North Adelaide, South Australia, Australia
Hum Mutat 28:897-903. 2007..This mutation analysis has identified a clear correlation between genotype and urinary GAG that can be used to predict clinical outcome... - Caregiving time in sickle cell disease: psychological effects in maternal caregiversJudith Tedlie Moskowitz
Department of Medicine, University of California San Francisco, Osher Center for Integrative Medicine, San Francisco, California, USA
Pediatr Blood Cancer 48:64-71. 2007..The purpose of this paper is to examine patterns of caregiving and the associated psychological impact on maternal caregivers of children with sickle cell disease (SCD)... - A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)Joseph Muenzer
University of North Carolina, Department of Pediatrics, Chapel Hill, NC 27599, USA
Genet Med 8:465-73. 2006..Idursulfase antibodies were detected in 46.9% of patients during the study. CONCLUSION: This study supports the use of weekly infusions of idursulfase in the treatment of mucopolysaccharidosis II... - Does liver biopsy overestimate liver iron concentration?Roland Fischer
Blood 108:1775-6; author reply 1776. 2006 - Threshold effect of urinary glycosaminoglycans and the walk test as indicators of disease progression in a survey of subjects with Mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)Stuart J Swiedler
BioMarin Pharmaceutical Inc, Novato, California, USA
Am J Med Genet A 134:144-50. 2005..Pending the collection of longitudinal data, these results suggest that urinary GAG levels predict clinical morbidity, and longer-term survival is associated with urinary GAG levels below a threshold of 100 mug/mg creatinine... - Entering a new treatment age for mucopolysaccharidosis VI disease: a search for better markers of disease progression and response to treatmentPaul Harmatz
J Pediatr (Rio J) 84:103-6. 2008 - Transfusional iron burden and liver toxicity after bone marrow transplantation for acute myelogenous leukemia and hemoglobinopathiesWasil Jastaniah
University of British Columbia and British Columbia s Children s Hospital, Vancouver, British Columbia, Canada
Pediatr Blood Cancer 50:319-24. 2008.... - Mutational analysis of mucopolysaccharidosis type VI patients undergoing a phase II trial of enzyme replacement therapyLitsa Karageorgos
Lysosomal Diseases Research Unit, Department of Genetic Medicine, Children, Youth and Women s Health Service, North Adelaide, SA 5006, Australia
Mol Genet Metab 90:164-70. 2007..972A>G and c.1191A>G. Cultured fibroblast ARSB mutant protein and residual activity were determined for each patient and, together with genotype information, used to predict the expected clinical severity of each patient... - The effect of vitamins C and E on biomarkers of oxidative stress depends on baseline levelGladys Block
School of Public Health, University of California, Berkeley, CA 94720 7360, USA
Free Radic Biol Med 45:377-84. 2008..Further studies are needed to establish whether treatment with vitamins C or E in persons with concentrations above that cut point could slow the development of cardiovascular disease... - Does liver biopsy overestimate liver iron concentration?Roland Fischer
Blood 108:778. 2006 - Management guidelines for mucopolysaccharidosis VIRoberto Giugliani
Medical Genetics Service, Hospital de Clinicas de Porto Alegre, Porto Alegre, Brazil
Pediatrics 120:405-18. 2007..The guidelines provide a detailed outline of disease manifestations by body system, recommendations for regular assessments, and an overview of current treatment options...