P Harmatz

Summary

Affiliation: Children's Hospital and Research Center
Country: USA

Publications

  1. pmc Oxidative stress and inflammation in iron-overloaded patients with beta-thalassaemia or sickle cell disease
    Patrick B Walter
    Children s Hospital Oakland Research Institute, Oakland, CA 94609, USA
    Br J Haematol 135:254-63. 2006
  2. pmc Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme®) therapy
    E Braunlin
    Pediatric Cardiology, University of Minnesota, Minneapolis, MN, USA
    J Inherit Metab Dis 36:385-94. 2013
  3. pmc Mucopolysaccharidosis VI
    Vassili Valayannopoulos
    Reference Center for Inherited Metabolic Diseases, Necker Enfants Malades Hospital, Paris, France
    Orphanet J Rare Dis 5:5. 2010
  4. pmc Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase
    Paul Harmatz
    Children s Hospital and Research Center Oakland, CA 94609, USA
    J Inherit Metab Dis 33:51-60. 2010
  5. doi request reprint Safety and efficacy of pegylated interferon alpha-2a and ribavirin for the treatment of hepatitis C in patients with thalassemia
    Paul Harmatz
    Children s Hospital and Research Center Oakland, 747 52 Street, Oakland, CA 94609, USA
    Haematologica 93:1247-51. 2008
  6. doi request reprint 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-sulfatase
    Paul Harmatz
    Children s Hospital and Research Center Oakland, 747 52nd Street, Oakland, CA 94609, USA
    Mol Genet Metab 94:469-75. 2008
  7. ncbi request reprint Phase Ib clinical trial of starch-conjugated deferoxamine (40SD02): a novel long-acting iron chelator
    Paul Harmatz
    Department of Gastroenterology, The Children s Hospital and Research Center Oakland, CA, USA
    Br J Haematol 138:374-81. 2007
  8. ncbi request reprint Severity of iron overload in patients with sickle cell disease receiving chronic red blood cell transfusion therapy
    P Harmatz
    Departments of Gastroenterology, Hematology Oncology, and Pathology, Children s Hospital Oakland, Oakland, CA 94609, USA
    Blood 96:76-9. 2000
  9. ncbi request reprint 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-l
    Paul 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
  10. ncbi request reprint 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-acetylga
    Paul Harmatz
    Children s Hospital and Research Center at Oakland, 747 52nd St, Oakland, CA 94609, USA
    Pediatrics 115:e681-9. 2005

Detail Information

Publications39

  1. pmc Oxidative stress and inflammation in iron-overloaded patients with beta-thalassaemia or sickle cell disease
    Patrick 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...
  2. pmc Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme®) therapy
    E Braunlin
    Pediatric Cardiology, University of Minnesota, Minneapolis, MN, USA
    J Inherit Metab Dis 36:385-94. 2013
    ..The results suggest that long-term ERT is effective in reducing intraventricular septal hypertrophy and preventing progression of cardiac valve abnormalities when administered to those <12 years of age...
  3. pmc Mucopolysaccharidosis VI
    Vassili 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...
  4. pmc Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase
    Paul 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...
  5. doi request reprint Safety and efficacy of pegylated interferon alpha-2a and ribavirin for the treatment of hepatitis C in patients with thalassemia
    Paul 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...
  6. doi request reprint 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-sulfatase
    Paul Harmatz
    Children s Hospital and Research Center Oakland, 747 52nd Street, Oakland, CA 94609, USA
    Mol Genet Metab 94:469-75. 2008
    ..Safety was evaluated by compliance, adverse event (AE) reporting and adherence to treatment...
  7. ncbi request reprint Phase Ib clinical trial of starch-conjugated deferoxamine (40SD02): a novel long-acting iron chelator
    Paul 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...
  8. ncbi request reprint Severity of iron overload in patients with sickle cell disease receiving chronic red blood cell transfusion therapy
    P 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...
  9. ncbi request reprint 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-l
    Paul 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
    ....
  10. ncbi request reprint 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-acetylga
    Paul Harmatz
    Children s Hospital and Research Center at Oakland, 747 52nd St, Oakland, CA 94609, USA
    Pediatrics 115:e681-9. 2005
    ....
  11. ncbi request reprint 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)...
  12. ncbi request reprint Pharmacokinetic profile of recombinant human N-acetylgalactosamine 4-sulphatase enzyme replacement therapy in patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): a phase I/II study
    P 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...
  13. ncbi request reprint Treatment of hepatitis C virus infection in thalassemia
    Ellen 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...
  14. doi request reprint Elevated exhaled carbon monoxide concentration in hemoglobinopathies and its relation to red blood cell transfusion therapy
    Ellen Butensky James
    Department of Gastroenterology, Children s Hospital and Research Center Oakland, Oakland, California 94609, USA
    Pediatr Hematol Oncol 27:112-21. 2010
    ....
  15. ncbi request reprint Serum ferritin underestimates liver iron concentration in transfusion independent thalassemia patients as compared to regularly transfused thalassemia and sickle cell patients
    Zahra 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...
  16. ncbi request reprint Iron homeostasis during transfusional iron overload in beta-thalassemia and sickle cell disease: changes in iron regulatory protein, hepcidin, and ferritin expression
    Zandra 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...
  17. doi request reprint Inflammation and oxidant-stress in beta-thalassemia patients treated with iron chelators deferasirox (ICL670) or deferoxamine: an ancillary study of the Novartis CICL670A0107 trial
    Patrick 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...
  18. doi request reprint Altered iron metabolism in children with human immunodeficiency virus disease
    Ellen 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...
  19. ncbi request reprint Potential mechanisms for altered iron metabolism in human immunodeficiency virus disease
    Ellen 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...
  20. ncbi request reprint Comparison of organ dysfunction in transfused patients with SCD or beta thalassemia
    Elliott 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...
  21. ncbi request reprint Morbidity and mortality in chronically transfused subjects with thalassemia and sickle cell disease: A report from the multi-center study of iron overload
    Ellen 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...
  22. ncbi request reprint A simple model to assess and improve adherence to iron chelation therapy with deferoxamine in patients with thalassemia
    Zahra 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...
  23. ncbi request reprint Monitoring long-term efficacy of iron chelation treatment with biomagnetic liver susceptometry
    Roland 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...
  24. ncbi request reprint The new SQUID biosusceptometer at Oakland: first year of experience
    E 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...
  25. pmc Reversed papilledema in an MPS VI patient with galsulfase (Naglazyme) therapy
    Selim 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)...
  26. doi request reprint Iron metabolism and iron chelation in sickle cell disease
    Patrick 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...
  27. ncbi request reprint Variability in hepatic iron concentration in percutaneous needle biopsy specimens from patients with transfusional hemosiderosis
    Ellen 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...
  28. doi request reprint Importance of surgical history in diagnosing mucopolysaccharidosis type II (Hunter syndrome): data from the Hunter Outcome Survey
    Nancy J Mendelsohn
    Department of Medical Genetics, Children s Hospitals and Clinics of Minnesota, Minneapolis, Minnesota 55404, USA
    Genet Med 12:816-22. 2010
    ..To characterize surgical histories typical of patients with mucopolysaccharidosis type II, thereby broadening understanding of the natural history of these patients and helping physicians recognize the disease...
  29. ncbi request reprint 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...
  30. ncbi request reprint Mutational analysis of 105 mucopolysaccharidosis type VI patients
    Litsa 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...
  31. doi request reprint Entering a new treatment age for mucopolysaccharidosis VI disease: a search for better markers of disease progression and response to treatment
    Paul Harmatz
    J Pediatr (Rio J) 84:103-6. 2008
  32. ncbi request reprint 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
    ..To evaluate the safety and efficacy of recombinant human iduronate-2-sulfatase (idursulfase) in the treatment of mucopolysaccharidosis II...
  33. ncbi request reprint Does liver biopsy overestimate liver iron concentration?
    Roland Fischer
    Blood 108:1775-6; author reply 1776. 2006
  34. ncbi request reprint Transfusional iron burden and liver toxicity after bone marrow transplantation for acute myelogenous leukemia and hemoglobinopathies
    Wasil Jastaniah
    University of British Columbia and British Columbia s Children s Hospital, Vancouver, British Columbia, Canada
    Pediatr Blood Cancer 50:319-24. 2008
    ....
  35. ncbi request reprint Caregiving time in sickle cell disease: psychological effects in maternal caregivers
    Judith 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)...
  36. ncbi request reprint Does liver biopsy overestimate liver iron concentration?
    Roland Fischer
    Blood 108:778. 2006
  37. pmc The effect of vitamins C and E on biomarkers of oxidative stress depends on baseline level
    Gladys 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...
  38. ncbi request reprint Management guidelines for mucopolysaccharidosis VI
    Roberto 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...
  39. ncbi request reprint Mutational analysis of mucopolysaccharidosis type VI patients undergoing a phase II trial of enzyme replacement therapy
    Litsa 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...