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

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 »

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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Late-Onset Noninfectious Pulmonary Complications after Hematopoietic Stem Cell Transplantation

AndrewC. 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 Disease

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Comorbidities and complications in adult and paediatric patients with pyruvate kinase deficiency: Analysis from the Peak Registry

Andreas Glenthøj, Rachael F. Grace, Carl Lander, Eduard J. van Beers, Bertil Glader, Kevin H. M. Kuo, Yan Yan, Bryan McGee, Audra N. Boscoe, Junlong Li, Paola BianchiCopenhagen University Hospital – Rigshospitalet. Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and Harvard Medical School. Thrive with PK Deficiency. University Medical Center Utrecht and Utrecht University. Stanford

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Cord blood transfusions in extremely low gestational age neonates to reduce severe retinopathy of prematurity: results of a prespecified interim analysis of the randomized BORN trial

Luciana Teofili, Patrizia Papacci, Carlo Dani, Francesco Cresi, Giulia Remaschi, Claudio Pellegrino, Maria Bianchi, Giulia Ansaldi, Maria Francesca Campagnoli, Barbara Vania, Domenico Lepore, Fabrizio Gaetano Saverio Franco, Marco Fabbri, Roberta Penta de Vera d’ Aragona, Anna Molisso, Enrico Beccastrini, Antonella Dragonetti, Lorenzo Orazi, Tina Pasciuto, Iolanda Mozzetta, Antonio Baldascino, Emanuela Locatelli, Caterina Giovanna Valentini, Carmen

Cord blood transfusions in extremely low gestational age neonates to reduce severe retinopathy of prematurity: results of a prespecified interim analysis of the randomized BORN trial Read More »

Epidemiology of Diffuse Alveolar Hemorrhage in Pediatric Allogeneic Hematopoietic Cell Transplant Recipients

Geoffrey Cheng, Michael A. Smith, Rachel Phelan, Ruta Brazauskas, Joelle Strom, Kwang Woo Ahn, Betty Hamilton, Andrew Peterson, Bipin Savani, Hélene Schoemans, Michelle Schoettler, Mohamed Sorror, Christine Higman, SandhyaKharbanda, Chrstopher C. Dvorak, Matt S. ZinterUniversity of California, San Francisco. Medical College of Wisconsin. Cleveland Clinic. Vanderbilt University Medical Center. University Hospital Gasthuisberg. Children’s Healthcare of

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Updates in Pediatric Sickle Cell Lung Disease

Michelle Gillespie, Olufunke Afolabi-Brown, Evans Machogu, Shaina Willen, Benjamin T. KoppOhio State University Wexner Medical Center and Nationwide Children’s Hospital.Children’ Hospital of Philadelphia. Riley Hospital for Children and Indiana University School of Medicine. University of California Davis Medical Center. Children’s Healthcare of Atlanta.United States Clinics in Chest MedicineClin Chest Med 2024; 45: 749-760DOI: 10.1016/j.ccm.2024.02.022 AbstractSickle cell

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