John B. Porter
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View article: Safety and pharmacodynamics of the ferroportin inhibitor vamifeport in patients with non-transfusion-dependent β-thalassemia: results from a randomized phase 2a study
Safety and pharmacodynamics of the ferroportin inhibitor vamifeport in patients with non-transfusion-dependent β-thalassemia: results from a randomized phase 2a study Open
Background Non-transfusion-dependent beta-thalassemia (β-NTDT) is characterized by ineffective erythropoiesis, increased intestinal iron absorption, and iron overload. The ferroportin inhibitor, vamifeport, has been shown to improve erythr…
View article: Delayed Inflammatory Hypermetabolism in Meningioma After Immunotherapy
Delayed Inflammatory Hypermetabolism in Meningioma After Immunotherapy Open
We report a case of malignant melanoma treated with immune checkpoint inhibitors, resulting in hypermetabolic activity in 2 meningiomas on FDG-PET, initially suggesting brain metastases. The lesions demonstrated no hypermetabolism pretreat…
View article: Efficacy and safety of luspatercept in non–transfusion-dependent β-thalassemia: long-term results from the BEYOND study
Efficacy and safety of luspatercept in non–transfusion-dependent β-thalassemia: long-term results from the BEYOND study Open
Chronic anemia due to non–transfusion-dependent β-thalassemia (NTDT) can result in clinical morbidities, particularly with inadequate management. Luspatercept was previously shown to improve hemoglobin levels in patients with NTDT in the p…
View article: Switching Patients With Congenital Adrenal Hyperplasia to Modified‐Release Hydrocortisone Capsules: Relative Bioavailability and Disease Control
Switching Patients With Congenital Adrenal Hyperplasia to Modified‐Release Hydrocortisone Capsules: Relative Bioavailability and Disease Control Open
Background Replacement therapy with modified‐release hydrocortisone capsules (MRHC) restores the physiological circadian cortisol rhythm in congenital adrenal hyperplasia (CAH). Aims To determine the relative bioavailability of MRHC and ev…
View article: ENERGIZE-T: A Global, Phase 3, Double-Blind, Randomized, Placebo-Controlled Study of Mitapivat in Adults with Transfusion-Dependent Alpha- or Beta-Thalassemia
ENERGIZE-T: A Global, Phase 3, Double-Blind, Randomized, Placebo-Controlled Study of Mitapivat in Adults with Transfusion-Dependent Alpha- or Beta-Thalassemia Open
Introduction: Transfusions and iron chelators have improved survival and outcomes in transfusion-dependent (TD)-thalassemia. However, they are associated with adverse effects, such as end organ complications and high healthcare resource ut…
View article: Improvement of Iron Overload Parameters in Patients with Transfusion-Dependent β-Thalassemia Treated with Luspatercept: Data from the Phase 3b Long-Term Rollover Study Following the BELIEVE Trial
Improvement of Iron Overload Parameters in Patients with Transfusion-Dependent β-Thalassemia Treated with Luspatercept: Data from the Phase 3b Long-Term Rollover Study Following the BELIEVE Trial Open
Introduction: Patients (pts) with transfusion-dependent (TD) β-thalassemia require lifelong red blood cell (RBC) transfusions, which can contribute to iron overload and increased morbidity and mortality. In the phase 3 BELIEVE trial (NCT02…
View article: 9341 CHAMPAIN Study: Initial Results From A Phase II Study Of Efficacy, Safety And Tolerability Of Modified-release Hydrocortisones: Chronocort® (Efmody®) Versus Plenadren®, In Primary Adrenal Insufficiency
9341 CHAMPAIN Study: Initial Results From A Phase II Study Of Efficacy, Safety And Tolerability Of Modified-release Hydrocortisones: Chronocort® (Efmody®) Versus Plenadren®, In Primary Adrenal Insufficiency Open
Disclosure: A. Prete: Research Investigator; Self; Diurnal. V. Theiler-Schwetz: Research Investigator; Self; Diurnal. W. Arlt: Research Investigator; Self; Diurnal. I.O. Chifu: Research Investigator; Self; Diurnal. B. Harbeck: Research Inv…
View article: Mitapivat improves ineffective erythropoiesis and iron overload in adult patients with pyruvate kinase deficiency
Mitapivat improves ineffective erythropoiesis and iron overload in adult patients with pyruvate kinase deficiency Open
Pyruvate kinase (PK) deficiency is a rare, hereditary disease characterized by chronic hemolytic anemia. Iron overload is a common complication regardless of age, genotype, or transfusion history. Mitapivat, an oral, allosteric PK activato…
View article: Luspatercept stimulates erythropoiesis, increases iron utilization, and redistributes body iron in transfusion‐dependent thalassemia
Luspatercept stimulates erythropoiesis, increases iron utilization, and redistributes body iron in transfusion‐dependent thalassemia Open
Luspatercept, a ligand‐trapping fusion protein, binds select TGF‐β superfamily ligands implicated in thalassemic erythropoiesis, promoting late‐stage erythroid maturation. Luspatercept reduced transfusion burden in the BELIEVE trial (NCT02…
View article: SAT304 Improved Biochemical Control With Dose Reduction In Chronic Glucocorticoid Therapy: A Phase III Extension Study Of Chronocort (Efmody) In The Treatment Of Congenital Adrenal Hyperplasia (CAH)
SAT304 Improved Biochemical Control With Dose Reduction In Chronic Glucocorticoid Therapy: A Phase III Extension Study Of Chronocort (Efmody) In The Treatment Of Congenital Adrenal Hyperplasia (CAH) Open
Disclosure: R.J. Ross: Employee; Self; Diurnal, A Neurocrine Biosciences Company. D.P. Merke: None. A. Mallappa: None. W. Arlt: None. A. Brac de la Perriere: None. A.L. Hirschberg: None. A. Juul: None. J.D. Newell-Price: None. C.G. Perry: …
View article: S273: LONG-TERM ERYTHROID RESPONSE DATA FROM PATIENTS (PTS) WITH NON-TRANSFUSION-DEPENDENT ΒETA-THALASSEMIA (NTDT) RECEIVING LUSPATERCEPT IN THE BEYOND TRIAL
S273: LONG-TERM ERYTHROID RESPONSE DATA FROM PATIENTS (PTS) WITH NON-TRANSFUSION-DEPENDENT ΒETA-THALASSEMIA (NTDT) RECEIVING LUSPATERCEPT IN THE BEYOND TRIAL Open
Background: Pts with NTDT can develop serious clinical complications due to untreated anemia. While there are no approved treatments (txs) for NTDT-associated anemia, luspatercept tx was shown to decrease transfusion burden and durably inc…
View article: P1493: DIAGNOSTIC ACCURACY OF AN AUTOMATED MAGNETIC RESONANCE IMAGE ANALYSIS SYSTEM FOR THE RAPID EVALUATION OF LIVER IRON CONCENTRATION IN PATIENTS WITH HFE RELATED HEMOCHROMATOSIS.
P1493: DIAGNOSTIC ACCURACY OF AN AUTOMATED MAGNETIC RESONANCE IMAGE ANALYSIS SYSTEM FOR THE RAPID EVALUATION OF LIVER IRON CONCENTRATION IN PATIENTS WITH HFE RELATED HEMOCHROMATOSIS. Open
Topic: 29. Iron metabolism, deficiency and overload Background: MRI is increasingly being used to measure liver iron concentration (LIC) in suspected or diagnosed hemochromatosis. To obtain an LIC result, MR image post-processing is requir…
View article: P1497: MITAPIVAT IMPROVES IRON OVERLOAD IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY WHO ARE REGULARLY TRANSFUSED
P1497: MITAPIVAT IMPROVES IRON OVERLOAD IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY WHO ARE REGULARLY TRANSFUSED Open
Topic: 29. Iron metabolism, deficiency and overload Background: Iron overload is highly prevalent in patients (pts) with pyruvate kinase (PK) deficiency, regardless of transfusion status, and can lead to serious complications including org…
View article: SLN124, a GalNAc conjugated 19‐mer siRNA targeting <i>tmprss6</i>, reduces plasma iron and increases hepcidin levels of healthy volunteers
SLN124, a GalNAc conjugated 19‐mer siRNA targeting <i>tmprss6</i>, reduces plasma iron and increases hepcidin levels of healthy volunteers Open
SLN124, an N‐acetylgalactosamine conjugated 19‐mer short interfering RNA, is being developed to treat iron‐loading anemias (including beta‐thalassemia and myelodysplastic syndromes) and myeloproliferative neoplasms (polycythemia vera). Thr…
View article: Health‐related quality of life in patients with β‐thalassemia: Data from the phase 3 <scp>BELIEVE</scp> trial of luspatercept
Health‐related quality of life in patients with β‐thalassemia: Data from the phase 3 <span>BELIEVE</span> trial of luspatercept Open
Background Patients with transfusion‐dependent (TD) β‐thalassemia require long‐term red blood cell transfusions (RBCTs) that lead to iron overload, impacting health‐related quality of life (HRQoL). Methods The impact of luspatercept, a fir…
View article: Long-term Outcomes of 63 Patients with Transfusion-Dependent β-Thalassemia (TDT) Followed-up to 7 Years after Treatment with betibeglogene autotemcel (beti-cel) Gene Therapy (GT) and Factors Impacting Neutrophil and Platelet Engraftment
Long-term Outcomes of 63 Patients with Transfusion-Dependent β-Thalassemia (TDT) Followed-up to 7 Years after Treatment with betibeglogene autotemcel (beti-cel) Gene Therapy (GT) and Factors Impacting Neutrophil and Platelet Engraftment Open
The use of thiotepa-treosulfan-fludarabine conditioning regimen and peripheral blood stem cell grafts is associated with improved outcomes of hematopoietic stem cell transplantation (HCT) in patients with high-risk thalassemia major. Howev…
View article: Early‐onset reduced bone mineral density in patients with pyruvate kinase deficiency
Early‐onset reduced bone mineral density in patients with pyruvate kinase deficiency Open
To the Editor: Pyruvate kinase (PK) deficiency is a rare disease caused by mutations in the PKLR gene encoding the red blood cell (RBC) PK enzyme, leading to defective glycolysis and chronic hemolytic anemia.1 The estimated prevalence of c…
View article: Modified-release hydrocortisone is associated with lower plasma renin activity in patients with salt-wasting congenital adrenal hyperplasia
Modified-release hydrocortisone is associated with lower plasma renin activity in patients with salt-wasting congenital adrenal hyperplasia Open
Objective Poorly controlled salt-wasting (SW) congenital adrenal hyperplasia (CAH) patients often require high 9α-fluorocortisol doses as they show high levels of 17-hydroxyprogesterone (17OHP), which is a mineralocorticoid (MC)-receptor a…
View article: The clinical relevance of detectable plasma iron species in iron overload states and subsequent to intravenous iron‐carbohydrate administration
The clinical relevance of detectable plasma iron species in iron overload states and subsequent to intravenous iron‐carbohydrate administration Open
Many disorders of iron homeostasis (e.g., iron overload) are associated with the dynamic kinetic profiles of multiple non‐transferrin bound iron (NTBI) species, chronic exposure to which is associated with deleterious end‐organ effects. He…
View article: Effects of green tea extract treatment on erythropoiesis and iron parameters in iron-overloaded β-thalassemic mice
Effects of green tea extract treatment on erythropoiesis and iron parameters in iron-overloaded β-thalassemic mice Open
β-Thalassemia is characterized by ineffective erythropoiesis leading to chronic anemia. Thus, increased iron absorption from the duodenum and via blood transfusions is required to maintain normal blood hemoglobin (Hb) levels and iron chela…
View article: Untreated Anemia in Nontransfusion-dependent β-thalassemia: Time to Sound the Alarm
Untreated Anemia in Nontransfusion-dependent β-thalassemia: Time to Sound the Alarm Open
Despite advances in management and the consequent improvement in survival, a diagnosis of β-thalassemia continues to confer considerable burden on patients, their families, and the healthcare system.1 This is primarily driven by the comple…
View article: 2021 Thalassaemia International Federation Guidelines for the Management of Transfusion-dependent Thalassemia
2021 Thalassaemia International Federation Guidelines for the Management of Transfusion-dependent Thalassemia Open
Beta-thalassemia and particularly its transfusion-dependent form (TDT) is a demanding clinical condition, requiring life-long care and follow-up, ideally in specialized centers and by multidisciplinary teams of experts. Despite the signifi…
View article: S270: LONGER-TERM ANALYSIS OF EFFICACY OF LUSPATERCEPT VERSUS PLACEBO IN PATIENTS WITH TRANSFUSION-DEPENDENT BETA-THALASSEMIA ENROLLED IN THE BELIEVE STUDY
S270: LONGER-TERM ANALYSIS OF EFFICACY OF LUSPATERCEPT VERSUS PLACEBO IN PATIENTS WITH TRANSFUSION-DEPENDENT BETA-THALASSEMIA ENROLLED IN THE BELIEVE STUDY Open
Background: Ineffective erythropoiesis and anemia are hallmarks of β-thalassemia. Luspatercept, a first-in-class erythroid maturation agent, has shown efficacy in the phase 3 BELIEVE (NCT02604433) trial in patients (pts) with β-thalassemia…
View article: P1545: DURABILITY OF HEMOGLOBIN RESPONSE AND REDUCTION IN TRANSFUSION BURDEN IS MAINTAINED OVER TIME IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY TREATED WITH MITAPIVAT IN A LONG-TERM EXTENSION STUDY
P1545: DURABILITY OF HEMOGLOBIN RESPONSE AND REDUCTION IN TRANSFUSION BURDEN IS MAINTAINED OVER TIME IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY TREATED WITH MITAPIVAT IN A LONG-TERM EXTENSION STUDY Open
Background: Mitapivat (AG-348), a first-in-class, oral, allosteric activator of red blood cell (RBC) PK enzyme, demonstrated improvements in hemoglobin (Hb) and markers of hemolysis and hematopoiesis (ACTIVATE, NCT03548220), and reduction …
View article: P1565: MITAPIVAT IMPROVES INEFFECTIVE ERYTHROPOIESIS AND REDUCES IRON OVERLOAD IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY
P1565: MITAPIVAT IMPROVES INEFFECTIVE ERYTHROPOIESIS AND REDUCES IRON OVERLOAD IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY Open
Background: Pyruvate kinase (PK) deficiency is a rare hereditary disease resulting in chronic hemolytic anemia, which is associated with serious complications, including iron overload (FeO). Ineffective erythropoiesis is linked to FeO in p…
View article: P1518: LONG-TERM SAFETY RESULTS OF THE BELIEVE STUDY OF LUSPATERCEPT IN ADULTS WITH ΒETA-THALASSEMIA
P1518: LONG-TERM SAFETY RESULTS OF THE BELIEVE STUDY OF LUSPATERCEPT IN ADULTS WITH ΒETA-THALASSEMIA Open
Background: The phase 3, placebo-controlled BELIEVE trial was shown luspatercept to be well tolerated in patients (pts) with transfusion-dependent β-thalassemia. Among the treatment-emergent adverse events (TEAEs) more commonly associated …
View article: P1544: BONE MINERAL DENSITY REMAINS STABLE IN PYRUVATE KINASE DEFICIENCY PATIENTS RECEIVING LONG-TERM TREATMENT WITH MITAPIVAT
P1544: BONE MINERAL DENSITY REMAINS STABLE IN PYRUVATE KINASE DEFICIENCY PATIENTS RECEIVING LONG-TERM TREATMENT WITH MITAPIVAT Open
Background: Hereditary pyruvate kinase (PK) deficiency results in chronic hemolysis that may lead to reduced bone mineral density (BMD). Mitapivat is an oral activator of PK in red blood cells (RBC) that improves hemoglobin concentration a…
View article: Betibeglogene Autotemcel Gene Therapy for Non–β <sup>0</sup> /β <sup>0</sup> Genotype β-Thalassemia
Betibeglogene Autotemcel Gene Therapy for Non–β <sup>0</sup> /β <sup>0</sup> Genotype β-Thalassemia Open
Treatment with beti-cel resulted in a sustained HbAT87Q level and a total hemoglobin level that was high enough to enable transfusion independence in most patients with a non-β0/β0 genotype, including those younger than 12 years of age. (F…