0000000000016890

AUTHOR

Jaco Botha

showing 4 related works from this author

Evaluation of the long-term treatment effects of idursulfase using statistical modelling: Data from the Hunter Outcome Survey (HOS)

2019

Treatment for mucopolysaccharidosis type II (MPS II Hunter syndrome) is available in the form of intravenous enzyme replacement therapy (ERT) with idursulfase (Shire, Lexington, MA, USA). This analysis used statistical modelling to evaluate the long-term treatment effects of idursulfase on selected clinical parameters based on data from HOS, a global, observational registry (Shire, Lexington, MA, USA). Mixed modelling was used to analyse data from male patients followed prospectively in HOS who had received idursulfase for 5-8 years and information available for two or more timepoints, of which one was pre-ERT. Data were excluded from patients with only pre-ERT information available, who ha…

Vital capacitymedicine.medical_specialtyIdursulfasebusiness.industryEndocrinology Diabetes and MetabolismHunter syndromeEnzyme replacement therapymedicine.diseaseBiochemistryClinical trialFEV1/FVC ratioEndocrinologyInternal medicineGeneticsmedicineObservational studyMucopolysaccharidosis type IIbusinessMolecular Biologymedicine.drugMolecular Genetics and Metabolism
researchProduct

Management of patients with hereditary angioedema in Germany: comparison with other countries in the Icatibant Outcome Survey

2018

Abstract Background The Icatibant Outcome Survey (IOS; NCT01034969) is a Shire‐sponsored, international, observational study monitoring the safety and effectiveness of icatibant, a bradykinin B2 receptor antagonist approved for the acute treatment of adults with hereditary angioedema with C1 inhibitor deficiency (HAE‐C1‐INH). Objective To report IOS data comparing demographic and icatibant treatment outcomes in patients with HAE‐C1‐INH from Germany to HAE‐C1‐INH patients from 11 other IOS countries. Methods A descriptive, retrospective, comparative analysis of data from 685 IOS patients with HAE‐C1‐INH from seven centres in Germany (n = 93) vs. centres from Austria, Brazil, Czech Republic, …

0301 basic medicineAdultMalePediatricsmedicine.medical_specialtyTime FactorsC1 inhibitor deficiencyTime to treatmentDermatologyBradykininAutoimmune DiseasesTime-to-Treatment03 medical and health scienceschemistry.chemical_compound0302 clinical medicineIcatibantGermanyBradykinin B2 Receptor AntagonistsmedicineHumansIn patientSymptom onsetRetrospective Studiesbusiness.industryAngioedemas HereditaryMiddle Agedmedicine.diseaseSymptom Flare UpHealth Surveys030104 developmental biologyInfectious Diseases030228 respiratory systemchemistryHereditary angioedemaObservational studyOriginal ArticleFemaleOutcome databusinessJournal of the European Academy of Dermatology and Venereology
researchProduct

<p>Cardio- Renal Outcomes With Long- Term Agalsidase Alfa Enzyme Replacement Therapy: A 10- Year Fabry Outcome Survey (FOS) Analysis</p>

2019

Purpose Following the publication of 5-year agalsidase alfa enzyme replacement therapy (ERT) outcomes data from the Fabry Outcome Survey (FOS), 10-year data were analyzed. Patients and methods FOS (ClinicalTrials.gov identifier: NCT03289065) data (April 2001 to August 2018) were retrospectively analyzed. Estimated glomerular filtration rate (eGFR) and left ventricular mass indexed to height (LVMI) were analyzed after treatment start (baseline) for patients with ≥3 measurements, including baseline and year 10. Results Median (range) age (years) of the evaluable treated renal cohort at treatment start was 48.8 (17.9-67.3) for females (n=62), 34.4 (18.0-66.8) for males (n=90). With eGFR ≥60 mL…

Pharmacologymedicine.medical_specialtyAlpha-galactosidasebiology030232 urology & nephrologyUrologyPharmaceutical ScienceRenal functionRetrospective cohort studyEnzyme replacement therapy030204 cardiovascular system & hematologymedicine.diseaseFabry disease03 medical and health sciences0302 clinical medicineDrug DiscoveryCohortmedicinebiology.proteinYoung adultCohort studyDrug Design, Development and Therapy
researchProduct

Characteristics of patients with mucopolysaccharidosis type II who have received a bone marrow transplant: Data from the Hunter Outcome Survey

2019

Mucopolysaccharidosis type II (MPS II Hunter syndrome) is a rare, life-limiting, X-linked lysosomal storage disease. The Hunter Outcome Survey (HOS) is a Shire-sponsored, global, observational registry initiated in 2005 that collects real-world data on the natural history of MPS II and long-term treatment with enzyme replacement therapy (ERT) with idursulfase. Patients receiving other forms of pharmacological ERT are excluded from HOS but individuals who have received a bone marrow transplant (BMT) may be enrolled. This analysis examined the characteristics of 36 male patients in HOS from Europe and North America who had received a BMT (March 2018 data). In total, 22 patients (61.1%) were E…

medicine.medical_specialtyBone marrow transplantIdursulfasebusiness.industryEndocrinology Diabetes and MetabolismHunter syndromeEnzyme replacement therapymedicine.diseaseBiochemistryNatural historyEndocrinologyGraft-versus-host diseaseRespiratory failureInternal medicineGeneticsmedicineMucopolysaccharidosis type IIbusinessMolecular Biologymedicine.drugMolecular Genetics and Metabolism
researchProduct