Article
Hematology
Shehu U. Abdullahi, Surayya Sunusi, Mohammed Sani Abba, Saifuddeen Sani, Hauwau Aminu Inuwa, Safiya Gambo, Awwal Gambo, Bilya Musa, Brittany V. Covert Greene, Adetola A. Kassim, Mark Rodeghier, Nafiu Hussaini, Mariana Ciobanu, Muktar H. Aliyu, Lori C. Jordan, Michael R. DeBaun
Summary: This study investigated the effectiveness of fixed oral moderate-dose hydroxyurea compared with fixed oral low-dose hydroxyurea for secondary stroke prevention in children with sickle cell anemia. The results showed that there was no evidence to support the superiority of moderate-dose hydroxyurea in reducing the risk of stroke or death compared to low-dose hydroxyurea.
Article
Hematology
Sindy N. Escobar Alvarez, Elizabeth R. Myers
Summary: The Doris Duke Charitable Foundation provided funding for sickle cell disease (SCD) research through the Innovations in Clinical Research Awards program, resulting in substantial advancements in diagnosis and treatment of the disease. The research investment showed substantial financial return and led to significant innovations in the field.
Article
Hematology
Monica L. Hulbert, Melanie E. Fields, Kristin P. Guilliams, Priyesha Bijlani, Shalini Shenoy, Slim Fellah, Alison S. Towerman, Michael M. Binkley, Robert C. McKinstry, Joshua S. Shimony, Yasheng Chen, Cihat Eldeniz, Dustin K. Ragan, Katie Vo, Hongyu An, Jin-Moo Lee, Andria L. Ford
Summary: This study investigated the effect of hematopoietic stem cell transplant (HSCT) on cerebral hemodynamics in children with sickle cell disease (SCD). It found that HSCT normalized cerebral blood flow and oxygen extraction fraction in SCD children, reducing the risk of strokes.
Review
Pediatrics
Lakshmanan Krishnamurti
Summary: Sickle cell disease is a severe inherited disorder involving abnormal red blood cells, which can be treated with hematopoietic stem cell transplantation. Different types of HCT have varying outcomes, with HLA-matched transplants showing the most promising results.
FRONTIERS IN PEDIATRICS
(2021)
Article
Hematology
Shehu U. Abdullahi, Binta W. Jibir, Halima Bello-Manga, Safiya Gambo, Hauwa Inuwa, Aliyu G. Tijjani, Nura Idris, Aisha Galadanci, Mustapha S. Hikima, Najibah Galadanci, Awwal Borodo, Abdulkadir M. Tabari, Lawal Haliru, Aisha Suleiman, Jamila Ibrahim, Brittany C. Greene, Djamila L. Ghafuri, Mark Rodeghier, James C. Slaughter, Fenelia J. Kirkham, Kathleen Neville, Adetola Kassim, Edwin Trevathan, Lori C. Jordan, Mulctar H. Aliyu, Michael R. DeBaun
Summary: In Africa, there is no significant difference in stroke incidence rate between children with sickle cell anemia and abnormal transcranial Doppler velocities who receive initial moderate-dose or low-dose hydroxyurea therapy. However, moderate-dose hydroxyurea therapy may lower the incidence rate of all-cause hospitalizations.
LANCET HAEMATOLOGY
(2022)
Article
Hematology
Yan Zheng, Jeffrey M. Gossett, Pei-Lin Chen, Martha Barton, Missy Ryan, Jing Yu, Guolian Kang, Jane S. Hankins, Stella T. Chou
Summary: This study found that the inflammatory state of transfusion recipients affects the risk of red blood cell alloimmunization, which is not modified by hydroxyurea (HU) therapy. Judicious use of transfusion during proinflammatory events is critical for preventing alloimmunization.
Review
Cell Biology
Paula Germino-Watnick, Malikiya Hinds, Anh Le, Rebecca Chu, Xiong Liu, Naoya Uchida
Summary: Autologous hematopoietic stem cell (HSC)-targeted gene therapy provides a one-time cure for various genetic diseases including sickle cell disease (SCD) and beta-thalassemia. This review discusses the methods of gene addition and gene editing in HSC-targeted gene therapy for SCD.
Article
Pediatrics
Asmaa M. Zahran, Khaled Saad, Khalid Elsayh, Shaimaa M. Khalaf, Khaled Hashim Mahmoud, Amira Elhoufey, Helal F. Hetta
Summary: Among children with SCD, HU treatment exhibited significant qualitative and quantitative effects on Tregs. HU treatment in SCD decreases the frequency of Tregs, as well as the levels of the most suppressive Tregs: HLA-DR+, CD39+, and CD69+. At the same time, HU increases the proportion of naive CD45RA+ Tregs. Our study showed the impact of HU therapy on Tregs in children with SCD.
PEDIATRIC RESEARCH
(2022)
Review
Immunology
Claire C. Sharpe, Abid Suddle, Sara Stuart-Smith
Summary: Sickle cell disease is a common genetic disorder with a global prevalence of over 300,000 people. While standard care can help patients live into adulthood, complications can still arise, leading to end-stage organ disease. Solid organ transplantation is an established treatment option for sickle cell patients, but careful patient selection and management are crucial for success. Hematopoietic stem cell transplantation and gene therapy may offer future treatments for adult patients with chronic organ damage, but they are not yet widely available. Currently, early intervention and comprehensive care can minimize the need for solid organ transplantation later in life.
Review
Chemistry, Medicinal
Annamaria Aprile, Silvia Sighinolfi, Laura Raggi, Giuliana Ferrari
Summary: Research in the last decade has shown multiple alterations of the BM niche in beta-thalassemia and sickle cell disease, emphasizing the importance of understanding the interaction between HSC biology and the BM microenvironment in improving clinical outcomes of transplantation.
Article
Biochemistry & Molecular Biology
Anoosha Habibi, Giovanna Cannas, Pablo Bartolucci, Ersi Voskaridou, Laure Joseph, Emmanuelle Bernit, Justine Gellen-Dautremer, Corine Charneau, Stephanie Ngo, Frederic Galacteros
Summary: Sickle cell disease is a group of inherited hemoglobin disorders characterized by altered deformability of red blood cells and various complications. Hydroxyurea is a major therapeutic agent for sickle cell patients, but its safety during pregnancy is not well-established. This study aims to describe pregnancy outcomes in the European Sickle Cell Disease COhoRT-HydroxyUrea study and share the results with the scientific community.
Review
Medicine, General & Internal
Nishka Bhalla, Anjali Bhargav, Sandeep Kumar Yadav, Aloukick Kumar Singh
Summary: Sickle cell disease (SCD) has been mentioned in the literature for a century, and advancements in understanding its molecular basis have provided various treatment options. Allogeneic hematopoietic stem cell transplantation (HSCT) is currently the only available option, although limitations such as donor availability and risks of complications exist. This review discusses different approaches to allogeneic HSCT for curing SCD, and highlights the potential of using megadose T-cell-depleted bone marrow in combination with donor-derived CD8 veto T cells under reduced intensity conditioning (RIC).
FRONTIERS IN MEDICINE
(2023)
Review
Genetics & Heredity
Thamal Darshana, David Rees, Anuja Premawardhena
Summary: The use of hydroxyurea and blood transfusion therapies for sickle cell disease in South Asia shows substantial variation and inconsistencies. Published data on these therapies are limited and heterogeneous, particularly from countries outside India, necessitating further evidence-based assessments and interventions.
ORPHANET JOURNAL OF RARE DISEASES
(2021)
Article
Immunology
Julia Teixeira Cottas de Azevedo, Thalita Cristina de Mello Costa, Keli Cristina Lima, Thiago Trovati Maciel, Patricia Vianna Bonini Palma, Luiz Guilherme Darrigo-Junior, Carlos Eduardo Setanni Grecco, Ana Beatriz P. L. Stracieri, Juliana Bernardes Elias, Fabiano Pieroni, Renato Luiz Guerino-Cunha, Ana Cristina Silva Pinto, Gil Cunha De Santis, Dimas Tadeu Covas, Olivier Hermine, Belinda Pinto Simoes, Maria Carolina Oliveira, Kelen Cristina Ribeiro Malmegrim
Summary: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a curative treatment for sickle cell disease (SCD). However, its effects on SCD pathophysiology are not fully understood. HSCT can normalize hematological parameters and decrease hemolysis in SCD patients, but systemic inflammation may persist for at least five years after transplantation, indicating that allo-HSCT does not equally affect all aspects of SCD pathophysiology.
FRONTIERS IN IMMUNOLOGY
(2021)
Article
Hematology
Alice Tang, Ana Nicolle Strat, Mahmudur Rahman, Helen Zhang, Weili Bao, Yunfeng Liu, David Shi, Xiuli An, Deepa Manwani, Patricia Shi, Karina Yazdanbakhsh, Avital Mendelson
Summary: Studies have found that murine SCD MSCs exhibit altered gene signatures, reduced stem cell properties, and increased oxidative stress, leading to decreased HSC maintenance ability. The activation of Toll-like receptor-4 through p65 in MSCs further exacerbates MSC dysfunction in SCD.
Article
Hematology
Ashkan Emadi, Rawan Faramand, Brandon Carter-Cooper, Seda Tolu, Laurie A. Ford, Rena G. Lapidus, Meir Wetzler, Eunice S. Wang, Arash Etemadi, Elizabeth A. Griffiths
AMERICAN JOURNAL OF HEMATOLOGY
(2015)
Article
Hematology
Emmanuel N. Olivier, Shouping Zhang, Zi Yan, Sandra Suzuka, Karl Roberts, Kai Wang, Eric E. Bouhassira
EXPERIMENTAL HEMATOLOGY
(2019)
Letter
Hematology
Seda S. Tolu, Morayma Reyes-Gil, Ugochi O. Ogu, Merin Thomas, Eric E. Bouhassira, Caterina P. Minniti
AMERICAN JOURNAL OF HEMATOLOGY
(2020)
Meeting Abstract
Hematology
Kai Wang, Khulan Batbayar, Karl Roberts, Emmanuel Olivier, Eric E. Bouhassira
Article
Cell Biology
Caterina P. Minniti, Seda S. Tolu, Kai Wang, Zi Yan, Karl Robert, Shouping Zhang, Andrew S. Crouch, Joan Uehlinger, Deepa Manwani, Eric E. Bouhassira
Article
Hematology
Seda S. Tolu, Andrew Crouch, Jaeun Choi, Qi Gao, Moramaya Reyes-Gil, Ugochi Olivia Ogu, Giacomo Vinces, Caterina P. Minniti
Summary: The presence of leg ulcers in individuals with sickle cell disease often indicates early signs of vasculopathy and future organ damage. HbF levels are associated with lower leg ulcer incidence, while hydroxyurea use is correlated with higher risk. Therefore, caution should be taken when considering hydroxyurea therapy in SCD patients with leg ulcers.
ANNALS OF HEMATOLOGY
(2022)
Article
Medicine, General & Internal
Gurasees S. Chawla, Seda S. Tolu, Shitij Arora
Summary: This case presents an elderly male patient in his 80s who experienced epigastric abdominal pain and fatigue for 2 weeks. His medical history revealed a previous excision of left toe acral melanoma 6 years ago and a lung mass, which he declined further workup for. On examination, he was found to have iron deficiency anemia, and esophagogastroduodenoscopy revealed a gastric mass. Histopathological analysis confirmed the presence of metastatic melanoma in both the gastric and pulmonary lesions, highlighting the slow progression of untreated pulmonary metastasis in melanoma.