Review
Pharmacology & Pharmacy
Rahyssa Rodrigues Sales, Barbara Lisboa Nogueira, Jessica Abdo Goncalves Tosatti, Karina Braga Gomes, Marcelo Rizzatti Luizon
Summary: Hydroxyurea is commonly used for the treatment of sickle cell anemia (SCA), but there is variability in patient response. This systematic review explores the impact of genetic polymorphisms on HbF levels in SCA patients treated with hydroxyurea, and identifies SNPs and genes associated with HbF changes. The findings suggest that changes in HbF levels upon hydroxyurea therapy are likely regulated by multiple loci.
FRONTIERS IN PHARMACOLOGY
(2022)
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
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)
Article
Hematology
Kristine Karkoska, Charles T. Quinn, Omar Niss, Amanda Pfeiffer, Min Dong, Alexander A. Vinks, Patrick T. McGann
Summary: Neurological complications are common in SCA patients, and cerebral oximetry can provide early prognostic information about tissue at risk of ischemic injury. Hydroxyurea may offer protection against neurophysiological changes seen in untreated SCA patients.
AMERICAN JOURNAL OF HEMATOLOGY
(2021)
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
Pharmacology & Pharmacy
Akancha Pandey, Jeremie H. Estepp, Rubesh Raja, Guolian Kang, Doraiswami Ramkrishna
Summary: This study developed pharmacokinetics and pharmacodynamics models to investigate the dosing and efficacy of hydroxyurea treatment in children with sickle cell disease (SCD). The models provided excellent fits for individuals with known or correctly inferred adherence, and can be used to determine optimal dosing regimens and study the impact of non-adherence on treatment outcomes.
Article
Medicine, General & Internal
Catherine Segbefia, Seth Kwabena Amponsah, Adwoa K. A. Afrane, Mame Yaa Nyarko, Yvonne Brew, Nihad Salifu, Samuel Yao Ahorhorlu, Abdul Malik Sulley, Lars Hviid, Michael Fokuo Ofori, George Obeng Adjei
Summary: In this study, interactions between hydroxyurea (HU) and antimalarials in sickle cell disease (SCD) were explored. The study found that parasite clearance and adverse event occurrence were similar in SCD children treated with artemether-lumefantrine (AL) regardless of HU status. However, distinct patterns of changes in laboratory indices suggest the potential need for larger, more focused studies in the future.
FRONTIERS IN MEDICINE
(2023)
Article
Hematology
Melanie E. Fields, Amy E. Mirro, Michael M. Binkley, Kristin P. Guilliams, Josiah B. Lewis, Slim Fellah, Yasheng Chen, Monica L. Hulbert, Hongyu An, Andria L. Ford, Jin-Moo Lee
Summary: This study found that children with sickle cell anemia (SCA) experience elevated oxygen extraction fraction (OEF) in the whole brain compared to anemic controls (AC) and healthy controls (HC). After controlling for the impact of anemia, SCA patients still show higher OEF levels, indicating that there may be other pathophysiological factors contributing to cerebral metabolic stress in these children.
AMERICAN JOURNAL OF HEMATOLOGY
(2022)
Review
Pediatrics
Seethal A. Jacob, Ravi Talati, Julie Kanter
Summary: This review discusses the current treatment landscape and emerging innovations in care for pediatric sickle cell disease, including established treatment methods and new medications. It also emphasizes the importance of long-term management for children receiving new therapies and calls for ongoing investment in people with sickle cell disease in low-income and middle-income countries.
LANCET CHILD & ADOLESCENT HEALTH
(2023)
Article
Medicine, General & Internal
Erica B. Esrick, Leslie E. Lehmann, Alessandra Biffi, Maureen Achebe, Christian Brendel, Marioara F. Ciuculescu, Heather Daley, Brenda MacKinnon, Emily Morris, Amy Federico, Daniela Abriss, Kari Boardman, Radia Khelladi, Kit Shaw, Helene Negre, Olivier Negre, Sarah Nikiforow, Jerome Ritz, Sung-Yun Pai, Wendy B. London, Colleen Dansereau, Matthew M. Heeney, Myriam Armant, John P. Manis, David A. Williams
Summary: This study confirms BCL11A inhibition as an effective approach for inducing HbF in sickle cell disease. Preliminary evidence suggests that shmik-based gene knockdown offers a favorable risk-benefit profile in treating sickle cell disease.
NEW ENGLAND JOURNAL OF MEDICINE
(2021)
Review
Biochemistry & Molecular Biology
Pagona Flevari, Ersi Voskaridou, Frederic Galacteros, Giovanna Cannas, Gylna Loko, Laure Joseph, Pablo Bartolucci, Justine Gellen-Dautremer, Emmanuelle Bernit, Corine Charneau, Anoosha Habibi
Summary: This article reports a case of myelodysplastic syndrome and highlights the lack of significant conclusions due to the scarcity of similar cases.
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
Charles T. Quinn, Omar Niss, Min Dong, Amanda Pfeiffer, Jennifer Korpik, Mary Reynaud, Holly Bonar, Theodosia A. Kalfa, Luke R. Smart, Punam Malik, Russell E. Ware, Alexander A. Vinks, Patrick T. McGann
Summary: Hydroxyurea is an effective treatment for sickle cell anaemia, with individualized, pharmacokinetics-guided dosing achieving sustained near-pancellular or pancellular HbF expression, which should be considered an achievable goal for children with SCA treated with hydroxyurea at optimal doses.
BRITISH JOURNAL OF HAEMATOLOGY
(2021)
Review
Hematology
Jean-Antoine Ribeil, Galia Pollock, Haydar Frangoul, Martin H. Steinberg
Summary: Hydroxyurea is the first approved drug for sickle cell disease, showing excellent effects in young children but variable responses in adults. The goal of pharmacotherapy is to reduce morbidity and mortality by preventing sickle hemoglobin polymerization and disrupting downstream pathophysiologic pathways. Maximal doses of hydroxyurea are recommended, and when insufficient, voxelotor and crizanlizumab can be added. Combination therapy may be necessary in poor responders to monotherapy or dual-drug therapy, considering the potential of cellular therapeutics and exchange transfusion programs.
AMERICAN JOURNAL OF HEMATOLOGY
(2023)
Article
Biochemistry & Molecular Biology
Mariana Delgadinho, Catarina Ginete, Brigida Santos, Carolina Fernandes, Carina Silva, Armandina Miranda, Jocelyne Neto de Vasconcelos, Miguel Brito
Summary: This study investigated the impact of hydroxyurea treatment on the gut microbiome of Angolan children with sickle cell anemia (SCA). The results showed that hydroxyurea increased the abundance of beneficial bacteria and reduced the abundance of pathogenic bacteria. These microorganisms could potentially serve as candidate biomarkers for disease severity and hydroxyurea efficacy.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Article
Pediatrics
Arlene Smaldone, Deepa Manwani, Banu Aygun, Kim Smith-Whitley, Haomiao Jia, Jean-Marie Bruzzese, Sally Findley, Joshua Massei, Nancy S. Green
Article
Oncology
Victoria Fashakin, Sanyukta Mathur, Peter Kwagala, Ezekial Mupere, Sarah Kiguli, Sabrina B. Kitaka, Michael B. Henry, Luke Van Ells, Sharifu Tusuubira, Lukia Mulumba, Jimmy Duong, Melissa S. Stockwell, Philip LaRussa, Nancy S. Green
JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY
(2020)
Review
Oncology
Scott D. Grosse, Nancy S. Green, Sarah L. Reeves
PEDIATRIC BLOOD & CANCER
(2020)
Article
Education, Scientific Disciplines
Deborah F. Stiles, Brenda L. Ruotolo, Helen Kim, Jane Cho, Paul S. Appelbaum, Nancy S. Green
Summary: The article discusses how Columbia University addressed challenges of protecting research participants and facilitating biomedical research during the COVID-19 pandemic. They implemented measures to temporarily halt and later speed up human subjects research, as well as prioritized effective communication and collaboration.
Article
Oncology
Miriam Kwarteng-Siaw, Amma Benneh-Akwasi Kuma, Nancy S. Green
Summary: Expanding services for sickle cell disease in Ghana is expected to increase the need for transition to adult care. This study focused on the experiences of adolescents and young adults at an adult sickle cell clinic in Accra, Ghana, and found that pretransition preparation may ease the peritransition experience.
JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY
(2022)
Article
Oncology
Cristina R. Fernandez, Maureen Licursi, Randi Wolf, Margaret T. Lee, Nancy S. Green
Summary: This study investigated the association between food insecurity, housing instability, and dietary quality among individuals with Sickle Cell Disease (SCD) in the US. Findings suggest that having one or more social determinants of health (SDoH) is linked to poorer diet quality, highlighting the importance of screening for food insecurity and housing instability in families of children with SCD to identify potential nutrition-related social needs.
PEDIATRIC BLOOD & CANCER
(2022)
Article
Neurosciences
Richard Idro, Amelia K. Boehme, Michael Kawooya, Samson K. Lubowa, Deogratias Munube, Paul Bangirana, Robert Opoka, Ezekiel Mupere, Angela Lignelli, Philip Kasirye, Nancy S. Green, Frank J. Minja
Summary: The study found that over half of Ugandan children with sickle cell anaemia had cerebrovascular infarcts and/or arterial stenoses. These vascular diseases were often undetectable by clinical assessments, highlighting the importance of MRI-MRA brain imaging in defining SCA cerebrovascular disease.
JOURNAL OF STROKE & CEREBROVASCULAR DISEASES
(2022)
Article
Oncology
Nancy S. Green, Deepa Manwani, Kim Smith-Whitley, Banu Aygun, Abena Appiah-Kubi, Arlene M. Smaldone
Summary: Youth with sickle cell disease and their caregivers are more vulnerable to stress and depression during the pandemic, and this may be worsened by the health and economic concerns associated with the pandemic. A survey conducted during the COVID-19 pandemic showed that the prevalence of mental health symptoms shifted among the youth-caregiver dyads, with fewer youth but more caregivers being affected. Both groups lacked optimism. Therefore, screening for mental health symptoms and food insecurity among youth with sickle cell disease during and after the pandemic appears necessary.
PEDIATRIC BLOOD & CANCER
(2022)
Letter
Hematology
Nancy S. Green, Layla Van Doren, Maureen Licursi, Daniel D. Billings, Luke A. Sandoval, Yona M. Z. Feit, Eldad A. Hod
BRITISH JOURNAL OF HAEMATOLOGY
(2022)
Article
Oncology
Megan A. Askew, Arlene M. Smaldone, Melanie A. Gold, Kim Smith-Whitley, John J. Strouse, Zhezhen Jin, Nancy S. Green
Summary: This study aimed to assess the contraceptive practices of pediatric hematology providers for female adolescents and young adults with sickle cell disease (SCD). The results showed that there were variations in provider contraceptive practices, especially based on provider status. Key motivators included patient request and disclosure of sexual activity, while key barriers included inadequate provider training and limited visit time.
PEDIATRIC BLOOD & CANCER
(2022)
Article
Hematology
Nancy S. Green, Andrew Zapfel, Obiageli E. Nnodu, Patience Franklin, Venee N. Tubman, Lulu Chirande, Charles Kiyaga, Catherine Chunda-Liyoka, Bernard Awuonda, Kwaku Ohene-Frempong, Baba P. D. Inusa, Russell E. Ware, Isaac Odame, Emmanuela E. Ambrose, Livingstone G. Dogara, Assaf P. Oron, Chase Willett, Alexis A. Thompson, Nancy Berliner, Theresa L. Coetzer, Enrico M. Novelli
Summary: Sickle cell disease is a common condition in sub-Saharan Africa with high under-5 mortality. The American Society of Hematology established the Consortium on Newborn Screening in Africa (CONSA) to implement standardized newborn hemoglobinopathy screening and early intervention. The primary objectives of the trial are to determine the birth incidence of SCD and assess the effectiveness of early standardized care in reducing mortality. Secondary objectives include establishing universal screening and interventions and evaluating trial implementation.
Article
Medicine, General & Internal
Sarah L. Reeves, Hannah K. Peng, Jeffrey J. Wing, Lindsay W. Cogan, Alka Goel, David Anders, Nancy S. Green, Lynda D. Lisabeth, Kevin J. Dombkowski
Summary: Hydroxyurea is underused among youths with sickle cell anemia (SCA), despite being the primary therapy for the disease. The release of new guidelines did not significantly increase hydroxyurea use. To increase its utilization, a multifaceted approach addressing various barriers is needed.
Article
Hematology
Nancy S. Green, Caterina Rosano, Paul Bangirana, Robert Opoka, Deogratias Munube, Philip Kasirye, Michael Kawooya, Samson K. Lubowa, Ezekiel Mupere, Andrea Conroy, Frank J. Minja, Amelia K. Boehme, Min Suk Kang, Lawrence S. Honig, Richard Idro
Summary: Cerebrovascular injury is common in children with sickle cell anaemia (SCA) in sub-Saharan Africa, but limited access to MRI-MRA hinders detection. Blood-based biomarkers, including neurofilament light chain (NfL), may help identify cerebral infarcts. In a study with Ugandan children with SCA, NfL levels were significantly higher in those with MRI-detected infarcts and elevated TCD velocity. NfL levels could potentially be used as a biomarker for SCA cerebrovascular injury.
BRITISH JOURNAL OF HAEMATOLOGY
(2023)
Article
Public, Environmental & Occupational Health
Jennel C. Osborne, Nancy S. Green, Arlene M. Smaldone
HISPANIC HEALTH CARE INTERNATIONAL
(2020)
Review
Hematology
Ann T. Farrell, Julie Panepinto, Ankit A. Desai, Adetola A. Kassim, Jeffrey Lebensburger, Mark C. Walters, Daniel E. Bauer, Rae M. Blaylark, Donna M. DiMichele, Mark T. Gladwin, Nancy S. Green, Kathryn Hassell, Gregory J. Kato, Elizabeth S. Klings, Donald B. Kohn, Lakshmanan Krishnamurti, Jane Little, Julie Makani, Punam Malik, Patrick T. McGann, Caterina Minniti, Claudia R. Morris, Isaac Odame, Patricia Ann Oneal, Rosanna Setse, Poornima Sharma, Shalini Shenoy