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
Biochemistry & Molecular Biology
Catarina Ginete, Mariana Delgadinho, Brigida Santos, Vera Pinto, Carina Silva, Armandina Miranda, Miguel Brito
Summary: Sickle cell anemia is a common inherited disease in sub-Saharan Africa with heterogeneous phenotypes. Hydroxyurea is the main treatment for these patients, but response to treatment varies. Identifying genetic variants associated with hydroxyurea response is important for predicting treatment outcomes. A pharmacogenetic study in Angolan children identified 30 variants in 18 genes that may be associated with drug response and found associations with hematological, biochemical, and clinical parameters. Further research is needed to confirm these findings.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Review
Chemistry, Medicinal
Joelma Santana dos Santos Neres, Setondji Cocou Modeste Alexandre Yahouedehou, Marilda Souza Goncalves
Summary: Inconsistent therapeutic responses have been observed in SCA patients undergoing HU treatment. Personalized dosages based on individual pharmacokinetic profiles have shown improved effectiveness. However, further research is needed in this area.
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
Hematology
Julie Kanter, R. Clark Brown, Cynthia Norris, Santosh M. Nair, Abdullah Kutlar, Deepa Manwani, Nirmish Shah, Chiaki Tanaka, Shankaranand Bodla, Gessami Sanchez-Olle, Urs Albers, Darla Liles
Summary: This study analyzed the pharmacokinetics, pharmacodynamics, safety, and efficacy of crizanlizumab in patients with sickle cell disease. The results demonstrated the sustained efficacy and long-term safety of the drug in treating sickle cell disease.
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.
Article
Oncology
Kristine Karkoska, Kevin Todd, Omar Niss, Kelly Clapp, Lynette Fenchel, Theodosia A. Kalfa, Punam Malik, Charles T. Quinn, Russell E. Ware, Patrick T. McGann
Summary: Universal and early initiation of hydroxyurea therapy for children with sickle cell anemia has led to significant increase in utilization, younger age of initiation, and reduced SCA-related admissions, demonstrating measurable improvements in clinical outcomes. This approach can serve as a model for other programs to expand hydroxyurea use for more children with SCA.
PEDIATRIC BLOOD & CANCER
(2021)
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
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
Pharmacology & Pharmacy
Min Dong, Russell E. Ware, Andre Dallmann, Alexander A. Vinks
Summary: Sickle cell anemia (SCA) is a genetic condition that can contribute to high-risk pregnancies. With improved management, SCA has become a chronic disease of adults and many patients are now considering parenthood. It is important to provide continuous management and avoid SCA-related complications for a healthy pregnancy.
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)
Review
Pharmacology & Pharmacy
Prabhsimran Kaur, Bidwan Sekhar Behera, Sandeep Singh, Anjana Munshi
Summary: Diabetes, a major cause of death worldwide, is currently treated with various strategies that often come with side effects. The new approach of SGLT2 inhibition shows promising results in controlling glucose levels with minimal adverse effects.
EUROPEAN JOURNAL OF PHARMACOLOGY
(2021)
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.
Review
Pediatrics
Carolyn E. Beck, Evelyne D. Trottier, Melanie Kirby-Allen, Yves Pastore
Summary: This article provides an overview of sickle cell disease (SCD) and its comprehensive care. It highlights the principles of prevention, advocacy, and rapid treatment of common acute complications. Case vignettes demonstrate the care principles for various acute complications, and the use of hydroxyurea is introduced as a medication that reduces morbidity and mortality in children with SCD.
PAEDIATRICS & CHILD HEALTH
(2022)
Article
Multidisciplinary Sciences
Rita Masese, Dominique Bulgin, Mitchell R. Knisely, Liliana Preiss, Eleanor Stevenson, Jane S. Hankins, Marsha J. Treadwell, Allison A. King, Victor R. Gordeuk, Julie Kanter, Robert Gibson, Jeffrey A. Glassberg, Paula Tanabe, Nirmish Shah
Summary: The study found that female patients had worse reports of pain severity, more vaso-occlusive episodes, and a higher occurrence of 3 or more hospital admissions in the past year, while male patients were more likely to experience acute chest syndrome, cardiovascular and musculoskeletal complications, but less likely to have depression.
Article
Mathematical & Computational Biology
Wenjian Bi, Yun Li, Matthew P. Smeltzer, Guimin Gao, Shengli Zhao, Guolian Kang
Article
Oncology
Amber M. Yates, Vijaya M. Joshi, Banu Aygun, Joseph Moen, Matthew P. Smeltzer, Devi Govindaswamy, Jola Dowdy, Alyssa Cotton, Guolian Kang, Russell E. Ware, Jane S. Hankins
PEDIATRIC BLOOD & CANCER
(2019)
Article
Oncology
Hannah E. Williams, Carrie R. Howell, Wassim Chemaitilly, Carmen L. Wilson, Seth E. Karol, Vikki G. Nolan, Matthew P. Smeltzer, Daniel M. Green, Matthew J. Ehrhardt, Daniel A. Mulrooney, Ching-Hon Pui, Melissa M. Hudson, Leslie L. Robison, Kirsten K. Ness
Article
Oncology
Raymond U. Osarogiagbon, Nicholas R. Faris, Walter Stevens, Carrie Fehnel, Cheryl Houston-Harris, Philip Ojeabulu, Olawale A. Akinbobola, Yu-Shen Lee, Meredith A. Ray, Matthew P. Smeltzer
JOURNAL OF THORACIC ONCOLOGY
(2020)
Article
Oncology
Katharine Thomas, Brianne A. Voros, Meghan Meadows-Taylor, Matthew P. Smeltzer, Ryan Griffin, J. Philip Boudreaux, Ramcharan Thiagarajan, Eugene A. Woltering, Robert A. Ramirez
Article
Oncology
Matthew P. Smeltzer, Murry W. Wynes, Sylvie Lantuejoul, Ross Soo, Suresh S. Ramalingam, Marileila Varella-Garcia, Meghan Meadows Taylor, Kristin Richeimer, Kelsey Wood, Kristen E. Howell, Mercedes Lilana Dalurzo, Enriqueta Felip, Gina Hollenbeck, Keith Kerr, Edward S. Kim, Clarissa Mathias, Jose Pacheco, Pieter Postmus, Charles Powell, Masahiro Tsuboi, Ignacio I. Wistuba, Heather A. Wakelee, Chandra P. Belani, Giorgio V. Scagliotti, Fred R. Hirsch
JOURNAL OF THORACIC ONCOLOGY
(2020)
Article
Health Care Sciences & Services
Nicole M. Alberts, Sherif M. Badawy, Jason Hodges, Jeremie H. Estepp, Chinonyelum Nwosu, Hamda Khan, Matthew P. Smeltzer, Ramin Homayouni, Sarah Norell, Lisa Klesges, Jerlym S. Porter, Jane S. Hankins
JMIR MHEALTH AND UHEALTH
(2020)
Letter
Hematology
Laila Elsherif, Lara C. Scott, David Wichlan, Susan K. Jones, Joacy G. Mathias, Jessica H. Shen, Matthew P. Smeltzer, Kenneth I. Ataga
BRITISH JOURNAL OF HAEMATOLOGY
(2021)
Article
Hematology
Winfred Wang, Molly Freeman, Latacha Hamilton, Yvonne Carroll, Guolian Kang, Joseph Moen, Matthew P. Smeltzer, Jane Schreiber, Andrew M. Heitzer, Jeremie Estepp, Banu Aygun
Summary: This study found that three-year-old children with sickle cell disease have a higher rate of underachievement in neurodevelopment, and this is associated with low household income and larger household size.
BRITISH JOURNAL OF HAEMATOLOGY
(2021)
Article
Medicine, General & Internal
Matthew P. Smeltzer, Kristen E. Howell, Marsha Treadwell, Liliana Preiss, Allison A. King, Jeffrey A. Glassberg, Paula Tanabe, Sherif M. Badawy, Lisa DiMartino, Robert Gibson, Julie Kanter, Lisa M. Klesges, Jane S. Hankins
Summary: Many providers were not familiar with NHLBI's SCD care guidelines, and barriers to providing care for patients with SCD were influenced by providers' specialty, training, and practice setting. Increasing provider knowledge could improve hydroxyurea utilization, pain management, and mental health support.
Review
Medicine, Research & Experimental
Matthew P. Smeltzer, Meredith A. Ray
Summary: This article aims to introduce common data types and analysis methods in clinical research, and provides references for further study and real-world examples. The authors summarize continuous and discrete data, summary statistics, hypothesis tests, and appropriate statistical tests, as well as underlying assumptions in a tabular format for enhanced accessibility.
EXPERIMENTAL BIOLOGY AND MEDICINE
(2022)
Article
Oncology
Matthew P. P. Smeltzer, Meredith A. A. Ray, Nicholas R. R. Faris, Meghan B. B. Meadows-Taylor, Fedoria Rugless, Courtney Berryman, Bianca Jackson, Carrie Fehnel, Alicia Pacheco, Laura McHugh, Edward T. T. Robbins, Kenneth D. D. Ward, Lisa M. M. Klesges, Raymond U. U. Osarogiagbon
Summary: A colocated multidisciplinary lung cancer clinic in a community-based health care system significantly improved the thoroughness of lung cancer staging, treatment appropriateness, and patient experience. However, there was no significant difference in overall survival between patients receiving multidisciplinary care and usual serial care.
JCO ONCOLOGY PRACTICE
(2023)
Article
Hematology
Laila Elsherif, Praghalathan Kanthakumar, Jeremiah Afolabi, Ashley F. F. Stratton, Ugochi Ogu, Marquita Nelson, Ayesha Mukhopadhyay, Matthew P. P. Smeltzer, Adebowale Adebiyi, Kenneth I. I. Ataga
Summary: This study explored the association between novel urinary biomarkers and albumin-creatinine ratio (ACR) in adults with sickle cell anaemia. Among the participants, 13 (35.2%) had persistent albuminuria (PA). Urinary levels of clusterin, retinol-binding protein 4, alpha-1 microglobulin, and angiotensinogen were significantly higher in participants with PA compared to those without PA. Multivariable analysis revealed that urinary angiotensinogen was associated with ACR, suggesting its potential in identifying sickle cell anaemia patients at risk for kidney disease.
BRITISH JOURNAL OF HAEMATOLOGY
(2023)
Article
Health Care Sciences & Services
Matthew P. Smeltzer, Sarah L. Reeves, William O. Cooper, Brandon K. Attell, John J. Strouse, Clifford M. Takemoto, Julie Kanter, Krista Latta, Allison P. Plaxco, Robert L. Davis, Daniel Hatch, Camila Reyes, Kevin Dombkowski, Angela Snyder, Susan Paulukonis, Ashima Singh, Mariam Kayle
Summary: Population-level data on sickle cell disease (SCD) in the United States is limited. The Centers for Disease Control and Prevention (CDC) has implemented the Sickle Cell Data Collection Programs (SCDC) to address this issue by developing a common informatics infrastructure and data model for standardized reporting. The model allows for the comparison of data across states and the compilation of core surveillance reports. This pilot project serves as a blueprint for similar initiatives in other rare diseases.
Article
Hematology
Ugochi O. Ogu, Ayesha Mukhopadhyay, Kruti Patel, Marquita N. Nelson, KayLee S. Strahan, Lin Wu, Matthew P. Smeltzer, Kenneth I. Ataga
Summary: This systematic review and meta-analysis found no difference in efficacy between escalated doses and fixed low-dose of hydroxyurea in adults with SCD. However, fixed low-dose resulted in a greater increase in hemoglobin levels.
EUROPEAN JOURNAL OF HAEMATOLOGY
(2023)