Class 5. Pulmonary Hypertension Associated with Hematological, Systemic, Metabolic, Nutritional and Other Disorders

Vasculopathy among children and adolescents with sickle cell disease: the crosstalk with annexin A1, vitamin D, and myocardial iron overload

Mohsen Saleh ElAlfy, Eman Abdel Rahman Ismail, Sara Mostafa Makkeyah, Ahmed Samir, Dina Husseiny Salama, Nanis Mohammed Salah Eldin, Dina Mohamed Fathy ElMaghraby, Nada Ayman Gad, Marwa Fath Ahmed Ali, Fatma Soliman El-Sayed EbeidAin Shams University. National Centre for Radiation Research and Technology, Egyptian Atomic Energy Authority. Minia UniversityEgypt Expert Review of HematologyExpert Rev Hematol […]

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Eliglustat and cardiac comorbidities in Gaucher disease: a pharmacogenomic approach to safety and efficacy

Noor Ul Ain, Armaan Saith, Audrey Ruan, Ruhua Yang, Aaron Burton, Pramod K. MistryYale School of Medicine and Yale New Haven Hospital.United States Frontiers in MedicineFront Med 2025; 12: DOI: 10.3389/fmed.2025.1535099 AbstractIntroduction: Gaucher disease (GD), a lysosomal storage disorder, results from the accumulation of glycosphingolipids due to deficient lysosomal glucocerebrosidase activity. This pathological accumulation triggers immune activation,

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Genetic Variants and Clinical Features of Patients With Glycogen Storage Disease Type Ib

Yu Xia, Yu Sun, Taozi Du, Chengkai Sun, Ying Xu, Wensong Ge, Lili Liang, Ruifang Wang, Manqing Sun, Bing Xiao, Wenjuan QiuXinhua Hospital, Ruijin Hospital and Shanghai Jiao Tong University. China Journal of the American Medical Association Network OpenJAMA Netw Open 2025; 8: DOI: 10.1001/jamanetworkopen.2024.61888 AbstractImportance: Glycogen storage disease type Ib (GSDIb) is a rare metabolic disorder

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Severe Precapillary Pulmonary Hypertension and Right Ventricular Failure After Hematopoietic Stem Cell Transplantation: Successful Outcomes With Aggressive Upfront Triple Therapy

Stine Andersen, Jesper Vandborg Bjerre, Karin Baekgaard, Lars Idorn, Mads Jønsson AndersenAarhus University Hospital and Aarhus University. Copenhagen University Hospital.Denmark Pediatric Blood and CancerPediatr Blood Cancer 2025; DOI: 10.1002/pbc.31623 AbstractAbstract Not Available CategoryClass V. Pulmonary Hypertension Associated with Hematological, Systemic, Metabolic and Other DisordersMedical Therapy. Efficacy or Lack of Efficacy Age Focus: Pediatric Pulmonary Vascular Diseasec

Severe Precapillary Pulmonary Hypertension and Right Ventricular Failure After Hematopoietic Stem Cell Transplantation: Successful Outcomes With Aggressive Upfront Triple Therapy Read More »

Case Report: severe pulmonary hypertension in a child with micronutrient deficiency

Laure Pache-Wannaz, Cristiana Voicu, Laurence Boillat, Nicole SekarskiLausanne University Hospital and University of Lausanne.Switzerland Frontiers in PediatricsFront Pediatr 2025; 13: DOI: 10.3389/fped.2025.1478889 AbstractWe describe the rare case of a previously healthy seven year-old boy, with an acute clinical onset of severe pulmonary hypertension. He recovered rapidly after vitamin supplementation. Patient history showed a highly selective food

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Targeting Lung Heme Iron by Aerosol Hemopexin Administration in Sickle Cell Disease Pulmonary Hypertension

Melissa Lucero, Christina Lisk, Francesca Cendali, Delaney Swindle, Saini Setua, Kiruphagaran Thangaraju, David I. Pak, Quinton O’Boyle, Shuwei Lu, Robert Tolson, Seth Zaeske, Nishant Rana, Saqib Khan, Natalie Westover, Pavel Davizon Castillo, Gemlyn George, Kathryn Hassel, Rachelle Nuss, Nathan Brinkman, Thomas Gentinetta, Andre F. Palmer, Angelo D’Alessandro, Paul W. Buelher, David C. IrwinUniversity of Colorado,

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Siblings With Berardinelli-Seip Congenital Lipodystrophy: Clinical Insights and Challenges

Sri Meghana Kankipati, Surbhi Dumra, Swati Thareja, Lyluma Ishfaq, Mah N. Zargar, Arghadip Das, Sreya Kongala, Salma YounasAndhra Medical College. Employees’ State Insurance Corporation (ESIC) Medical College and Hospital. The Hans Foundation. Central Michigan University College of Medicine. Fatima Jinnah Medical University. Nilratan Sircar Medical College and Hospital. Osmania Medical College. Punjab University College of

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The Role of Early Child Nutrition in Pulmonary Hypertension – A Narrative Review

Alina-Costina Luca, Cristina Stoica, Cosmin Diaconescu, Elena T. Tarcă, Solange Tamara Ros, Lăcrămioara Ionela Butnariu, Bogdan Aurelian Stana, Bogdan Gafton, Antoanela Curici, Eduard Vasile Ros, Dana Elena Mîndru“Grigore T. Popa” University of Medicine and Pharmacy. Emergency Hospital for Children “Sfanta Maria”. ”Carol Davila” University of Medicine and Pharmacy.Romania ChildrenChildren 2024; 11: DOI: 10.3390/children11111307 AbstractPulmonary hypertension is

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Late-Onset Noninfectious Pulmonary Complications after Hematopoietic Stem Cell Transplantation

Andrew C. Harris, Kimia Ganjaei, Camila Vilela, Alexander GeyerMemorial Sloan Kettering Cancer Center. Lenox Hill Hospital and Northwell Health. United States Transplantation and Cellular TherapyTransplant Cell Ther 2024; 30: DOI: 10.1016/j.jtct.2024.05.022 AbstractAbstract Not Available CategoryClass V. Pulmonary Hypertension Associated with Hematological, Systemic, Metabolic and Other DisordersReview Articles Concerning Pulmonary Vascular Disease Age Focus: Pediatric Pulmonary Vascular

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Clinical profile and thiamine transporter gene (SLC19A2 and SLC19A3) variations in infants with thiamine-responsive pulmonary hypertension and acute respiratory infection

Swathi Shenoy, Vijaya Kumar Deekshit, Swathi Sunil Rao, Prathibha Shankar Ashwini, Rathika Damodara ShenoyNITTE University Center for Science Education and Research. Doctor’s Hospital. American University of Antigua College of Medicine.India, Antigua and Barbuda Journal of Tropical PediatricsJ Trop Pediatr 2024; DOI: 10.1093/tropej/fmae030 AbstractMaternal thiamine deficiency is prevalent in low- and middle-income countries. Thiamine-responsive pulmonary hypertension (TRPHTN)

Clinical profile and thiamine transporter gene (SLC19A2 and SLC19A3) variations in infants with thiamine-responsive pulmonary hypertension and acute respiratory infection Read More »

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