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Journal Articles

Does Gene Therapy in Beta Thalassemia Normalize Novel Markers of Ineffective Erythropoiesis and Iron Homeostasis?

Alexis A. Thompson, MD MPH, Tomas Ganz, MD PhD, Mary Therese Forsyth, Elizabeta Nemeth, PhD, Sherif M. Badawy, MD

Blood (2019) 134 (Supplement_1): 816.

https://doi.org/10.1182/blood-2019-129658

Published: 2019

Journal Articles

Experience with Low Dose Thalidomide in Transfusion Dependent Beta Thalassaemia in a Resource Limited Setting

Pallavi Mehta, MD DM, Neha Yadav, BDS , MBA, Priyanka Soni, MD FNB, Shinto Francis Thekuddan, MBBS,MDDM, Reema Singh, MSc, Vishvdeep Khushoo, MBBS,MDDM, Sumeet Prakash Mirgh, MBBS,MDDM, Narendra Agrawal, MD DM, Rayaz Ahmed, MDDM, Jyotsna Kapoor, MSc, Dinesh Bhurani, MD DM, FRCPA

Blood (2019) 134 (Supplement_1): 963.

https://doi.org/10.1182/blood-2019-131682

Published: 2019

Journal Articles

Evaluation of Endocrine Complications in Beta-Thalassemia Intermedia Patients: A Cross Sectional Multi-Center Study

Blood (2018) 132 (Supplement 1): 2343.

https://doi.org/10.1182/blood-2018-99-110903

Published: 2018

Journal Articles

Hepatitis C Virus Infection, but Not Hepatic Iron Overload Is the Dominant Risk Factor for the Manifestation of Hepatocellular Carcinoma Among Greek Thalassemic Patients

Blood (2018) 132 (Supplement 1): 2347.

https://doi.org/10.1182/blood-2018-99-119731

Published: 2018

Journal Articles

Neurological Manifestations Due to Extramedullary Hematopoiesis in Greek Patients with Thalassemia Intermedia: Not Such a Rare Clinical Finding

Michael D. Diamantidis, MD PhD MSc, Alexandra Kourakli, MD PhD, Efthymia Vlachaki, MBBS, Despoina Pantelidou, MD PhD, Evgenia Verigou, Evangelos Alexiou, Fotios Girtovitis, PhD MD, Paraskevi Fotiou, Elisavet Grouzi, Argiris Symeonidis, MD PhD

Blood (2018) 132 (Supplement 1): 2349.

https://doi.org/10.1182/blood-2018-99-119376

Published: 2018

Journal Articles

Stratification for Age in Non-Transfusion-Dependent Thalassemia: Data from a National Italian Networking

Alessia Pepe, MD, Laura Pistoia, Nicola Romano, Giuseppe Colaci, Aurelio Maggio, MD, Antonella Carollo, Saveria Campisi, MD, Maria Caterina Putti, MD, Ilaria Fotzi, MD, Domenico D'Ascola, MD, Emanuele Grassedonio, Vincenzo Positano, Antonella Meloni, PhD

Blood (2018) 132 (Supplement 1): 3639.

https://doi.org/10.1182/blood-2018-99-112044

Published: 2018

Journal Articles

B0 Vs. Non-B0 Genotype: Differences in Non-Transfusion-Dependent Thalassemia Patients

Alessia Pepe, MD, Laura Pistoia, Domenico Maddaloni, Teodosio Grippo, Monica Benni, Leonardo Sardella, Maria Grazia Sanna, Giovanni Giugno, Giulia Guerrini, Aurelio Maggio, MD, Stefania Renne, Massimiliano Missere, Vincenzo Positano, Antonella Meloni, PhD

Blood (2018) 132 (Supplement 1): 1056.

https://doi.org/10.1182/blood-2018-99-112553

Published: 2018

Journal Articles

Real-World Experience of Switching from Deferasirox Dispersible to Film-Coated Tablets: Impact on Adherence to Chelation Therapy, Iron Overload and Renal Function

Simon Cheesman, Raakhee Shah, MPharm, Sara Trompeter, MD, Perla Eleftheriou, FRCPath, MRCP, Bernadette Hylton, RN, Maciej W Garbowski, MDPhD, John Porter, MA MDFRCP FRCPath

Blood (2018) 132 (Supplement 1): 1062.

https://doi.org/10.1182/blood-2018-99-119030

Published: 2018

Journal Articles

SLN124, a Galnac-siRNA Conjugate Targeting TMPRSS6, for the Treatment of Iron Overload and Ineffective Erythropoiesis Such As in Beta-Thalassemia

Sandro Altamura, PhD, Sandro Altamura, PhD, Martina U. Muckenthaler, PhD, Sibylle Dames, MSc, Christian Frauendorf, PhD, Steffen Schubert, PhD, Manuela Aleku, MSc, Jim Vadolas, PhD, George Grigoriadis, MBBS PhD FRACP FRCPA, Ulrich Zügel, PhD

Blood (2018) 132 (Supplement 1): 2340.

https://doi.org/10.1182/blood-2018-99-110163

Published: 2018

Journal Articles

Transmembrane Protease, Serine 6 (TMPRSS6) Antisense Oligonucleotide (IONIS-TMPRSS6-L RX ) Reduces Plasma Iron Levels of Healthy Volunteers in a Phase 1 Clinical Study

Michael McCaleb, PhD, Jason Lickliter, MBBS, PhD, Andrew Dibble, PhD, Eugene Schneider, MD, Mariam Aghajan, Shuling Guo, Steve Hughes, MD MBA, Richard S Geary, PhD, Brett P. Monia, PhD

Blood (2018) 132 (Supplement 1): 3634.

https://doi.org/10.1182/blood-2018-99-115339

Published: 2018

Journal Articles

Is the Time of Revisiting Classification of Thalassemia Syndromes According with the Phenotype Severity?

Angela Vitrano, PhD, Giuseppina Calvaruso, MD, Lorella Pitrolo, MD, Alessia Pepe, MD, Antonella Meloni, PhD, Laura Pistoia, MD, Maria Rita Gamberini, MD, Vincenzo Caruso, MD, Aldo Filosa, MD, Paolo Ricchi, MD, Liana Cuccia, MD, Domenico D'Ascola, MD, Francesco Sorrentino, MD, Angelo Peluso, MD, Aurelio Maggio, MD

Blood (2017) 130 (Supplement 1): 935.

https://doi.org/10.1182/blood.V130.Suppl_1.935.935

Published: 2017

Journal Articles

Inadequacy of Ferritin Trends for Predicting Changes in LIC Risk Category in Transfusion Depedent and Well Chelated Patients with Haemoglobinopathies

Angela Vitrano, PhD, Massimiliano Sacco, DR, Rosamaria Rosso, MD, Alessandra Quota, MD, Daniela Fiorino, MD, Esther Natalie Oliva, MD, Calogera Gerardi, MD, Gaetano Roccamo, MD, Vincenzo Spadola, MD, Aldo Filosa, MD, Lorenzo Tesé, MD, Giuseppina Calvaruso, MD, Lorella Pitrolo, MD, Laura Mistretta, DR, Filippo Cassarà, DR, Rosario Di Maggio, MD, Aurelio Maggio, MD

Blood (2017) 130 (Supplement 1): 936.

https://doi.org/10.1182/blood.V130.Suppl_1.936.936

Published: 2017

Journal Articles

Low Impact of Genetic Modifiers on the Phenotype of Homozygous Beta Thalassemia in the Last Decennial Cohort of Thalassemia Newborns in Sardinia

Paolo Moi, MD, Matteo Marcello, MD, Stefania Satta, PhD, Laura Manunza, PhD, Franca Rosa De Martis, Raffaella Origa, MD, Giovanni Caocci, MD, Susanna Barella, MD

Blood (2017) 130 (Supplement 1): 951.

https://doi.org/10.1182/blood.V130.Suppl_1.951.951

Published: 2017

Journal Articles

Developing a Galnac-Conjugated TMPRSS6 Antisense Therapy for the Treatment of β-Thalassemia

Mariam Aghajan, PhD, Carla Casu, PhD, Vania lo Presti, Sheri Booten, Brett P. Monia, PhD, Stefano Rivella, PhD, Shuling Guo, PhD

Blood (2016) 128 (22): 1013.

https://doi.org/10.1182/blood.V128.22.1013.1013

Published: 2016

Journal Articles

Iron Overload Status in Patients with Non-Transfusion-Dependent Thalassemia in China

Rong rong Liu, MD, Yu mei Huang, Peng Peng, MD, Xiao yun Wei, Yu Lei, Jiao di Liu, Gao hui Yang, Yong rong Lai, MD

Blood (2016) 128 (22): 1287.

https://doi.org/10.1182/blood.V128.22.1287.1287

Published: 2016

Journal Articles

Regional Susceptibility to Chronic Anemia in WM Microstructure Using Diffusion Tensor Imaging

Soyoung Choi, BA, Adam M Bush, MS, Matt Borzage, PhD, Anand Joshi, PhD, Thomas D. Coates, MD, Richard Leahy, PhD, John C Wood, MD PhD

Blood (2016) 128 (22): 3640.

https://doi.org/10.1182/blood.V128.22.3640.3640

Published: 2016

Journal Articles

Extramedullary Hematopoiesis in Patients with Primary Myelofibrosis Rare and Serious Complications

Mohamed A. Yassin, MBBS, MSc, CABM, FACP, Abdulqadir Nashwan, Shehab Mohamed

Blood (2016) 128 (22): 5490.

https://doi.org/10.1182/blood.V128.22.5490.5490

Published: 2016

Journal Articles

Extra Medullary Hematopoiesis in Patients with Polycythemia Vera Rare and Unusual Presentations

Mohamed A. Yassin, MBBS, MSc, CABM, FACP, Shehab Mohamed, Abdulqadir Nashwan

Blood (2016) 128 (22): 5491.

https://doi.org/10.1182/blood.V128.22.5491.5491

Published: 2016

Journal Articles

Systemic Heme and Iron Overload Results in Depletion of Serum Hemopexin, Haptoglobin and Transferrin and Correlates with Markers of Endothelial Activation and Lipid Oxidation in Beta Thalassemia Major and Intermedia

Francesca Vinchi, PhD, Gregory M Vercellotti, MD, John D. Belcher, PhD, Eitan Fibach, PhD, Hala Zreid, MD, Rushdy Rasras, Hussam Ghoti, MD, Martina U. Muckenthaler, PhD, Eliezer A. Rachmilewitz, MD

Blood (2016) 128 (22): 2469.

https://doi.org/10.1182/blood.V128.22.2469.2469

Published: 2016

Journal Articles

Genetic Investigation of the Role of GDF11 in the Treatment of β-Thalassemia and MDS

Paraskevi Rea Oikonomidou, MD, Ping La, PhD, Ritama Gupta, MSc, Vania Lo Presti, Carla Casu, PhD, Laura Breda, PhD, Deborah Watson, PhD, Rajasekhar NVS Suragani, PhD, Ravindra Kumar, PhD, Stefano Rivella, PhD

Blood (2016) 128 (22): 2439.

https://doi.org/10.1182/blood.V128.22.2439.2439

Published: 2016

NARROW

Does Gene Therapy in Beta Thalassemia Normalize Novel Markers of Ineffective Erythropoiesis and Iron Homeostasis?

Experience with Low Dose Thalidomide in Transfusion Dependent Beta Thalassaemia in a Resource Limited Setting

Evaluation of Endocrine Complications in Beta-Thalassemia Intermedia Patients: A Cross Sectional Multi-Center Study

Hepatitis C Virus Infection, but Not Hepatic Iron Overload Is the Dominant Risk Factor for the Manifestation of Hepatocellular Carcinoma Among Greek Thalassemic Patients

Neurological Manifestations Due to Extramedullary Hematopoiesis in Greek Patients with Thalassemia Intermedia: Not Such a Rare Clinical Finding

Stratification for Age in Non-Transfusion-Dependent Thalassemia: Data from a National Italian Networking

B0 Vs. Non-B0 Genotype: Differences in Non-Transfusion-Dependent Thalassemia Patients

Real-World Experience of Switching from Deferasirox Dispersible to Film-Coated Tablets: Impact on Adherence to Chelation Therapy, Iron Overload and Renal Function

SLN124, a Galnac-siRNA Conjugate Targeting TMPRSS6, for the Treatment of Iron Overload and Ineffective Erythropoiesis Such As in Beta-Thalassemia

Transmembrane Protease, Serine 6 (TMPRSS6) Antisense Oligonucleotide (IONIS-TMPRSS6-L RX ) Reduces Plasma Iron Levels of Healthy Volunteers in a Phase 1 Clinical Study

Is the Time of Revisiting Classification of Thalassemia Syndromes According with the Phenotype Severity?

Inadequacy of Ferritin Trends for Predicting Changes in LIC Risk Category in Transfusion Depedent and Well Chelated Patients with Haemoglobinopathies

Low Impact of Genetic Modifiers on the Phenotype of Homozygous Beta Thalassemia in the Last Decennial Cohort of Thalassemia Newborns in Sardinia

Developing a Galnac-Conjugated TMPRSS6 Antisense Therapy for the Treatment of β-Thalassemia

Iron Overload Status in Patients with Non-Transfusion-Dependent Thalassemia in China

Regional Susceptibility to Chronic Anemia in WM Microstructure Using Diffusion Tensor Imaging

Extramedullary Hematopoiesis in Patients with Primary Myelofibrosis Rare and Serious Complications

Extra Medullary Hematopoiesis in Patients with Polycythemia Vera Rare and Unusual Presentations

Systemic Heme and Iron Overload Results in Depletion of Serum Hemopexin, Haptoglobin and Transferrin and Correlates with Markers of Endothelial Activation and Lipid Oxidation in Beta Thalassemia Major and Intermedia

Genetic Investigation of the Role of GDF11 in the Treatment of β-Thalassemia and MDS

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American Society of Hematology

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American Society of Hematology

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