Hematology

Publications

Alharbi, S; Merkle, S; Hammill, AM; Waters, AM; Le Cras, TD. RAS Pathway Mutations and Therapeutics in Vascular Anomalies. Pediatric Blood and Cancer. 2025; 72:e31605.

Al-Samkari, H; Kasthuri, RS; Mager, HJ; Zhou, JY; Serra, MM; Samuelson-Bannow, BT; Van Doren, LN; Piccirillo, JF; Clancy, MS; McCrae, KR; Mei-Zahav, M; von Drygalski, A; Olitsky, S; Faughnan, ME. Standardization of Terminology, Definitions, and Outcome Criteria for Bleeding in Hereditary Hemorrhagic Telangiectasia: International Consensus Report. American Journal of Hematology. 2025; 100:1813-1827.

Ambrose, EE; Kidenya, BR; Smart, LR; Manjurano, A. Alpha+ Thalassemia in Northwestern Tanzania: Molecular and Hematological Insights From Newborn Screening. Journal of Blood Medicine. 2025; 16:241-250.

Ambrose, EE; Sabuni, PA; Jason, DP; Ware, RE; Makubi, AN; Smart, LR. Hydroxyurea to decrease stroke risk in children with sickle cell anemia: a systematic review and meta-analysis. 2025; 1:100001.

Belton, TD; Steinway, CM; Teng, O; Shults, J; Barakat, LP; Aygun, B; Appiah-Kubi, A; Crosby, LE; Niss, O; Andemariam, B; Johnson, TB; Rubin, DM; Smith-Whitley, KM; Jan, S. The Community Health Workers and Mobile Health for Emerging Adults Transitioning Sickle Cell Disease Care (COMETS) Trial: Protocol for a Randomized Controlled Trial. JMIR Research Protocols. 2025; 14:e69239.

Boucher, AA; Dayton, VJ; Pratt, AR; Nassar, NN; Elgammal, Y; Kalfa, TA. Three-generation female cohort with macrocytic anemia and iron overload. American Journal of Hematology. 2025; 100:133-138.

Canaud, G; López-Gutiérrez, JC; Hammill, A; Weibel, L; Niglis, S; Paul, M; Papadimitriou, A; Wroclawska, M; Caro, N; Adams, D. 118: Randomized, Double-Blind, Phase 2 Study of Alpelisib in Pediatric and Adult Patients With PIK3CA-Related Overgrowth Spectrum (PROS). Journal of Investigative Dermatology. 2025; 145:e29.

Cantrell, R; Alex Feldman, H; Rosenfeldt, L; Ali, A; Gourley, B; Sprague, C; Leino, D; Crosby, J; Revenko, A; Monia, B; Waggoner, SN; Palumbo, JS. Prothrombin prevents fatal T cell-dependent anemia during chronic virus infection of mice. JCI insight. 2025; 10:e181063.

Chan, AK; Susen, S; Khoo, L; von Drygalski, A; Oldenburg, J; Shen, M; Peyvandi, F; Tarango, C; Chowdary, P; Mamikonian, L; Palmborg, H; Dumont, J; Santagostino, E; Hermans, C. Perioperative Management with Efanesoctocog Alfa in Adults, Adolescents, and Children with Severe Hemophilia A in the Phase 3 XTEND Clinical Program. Hamostaseologie. 2025; 45:s57-s58.

Chang, M; Semakula, D; Little, JA; Kanter, J; Manwani, DG; Owusu-Ansah, A; Cohen, AJ; Cronin, RM; Desai, PC; Strouse, JJ; Alvarez, OA; Saif Ur Rehman, S; Shah, S; Lanzkron, SM. Grandchildren of GRNDaD: Shifts in disease-modifying therapy at the adolescent transition in sickle cell disease. British Journal of Haematology. 2025; 207:1070-1075.

COHEN, RM; CRAIG, J; OMOLOLU, SO; TOZZO, V; SMITH, EP; ARBABI, S; GENCO, M; ABPLANALP, W; THIBAULT, D; QUINN, CT; LINDSELL, CJ; BERGENSTAL, RM; FRANCO, RS; HIGGINS, J. 166-OR: Mean RBC Age (MRBC) Variation Accounts for the Predominance of Mismatch (MM) between Measured HbA1c (mA1c) and CGM-Derived Estimated HbA1c (eA1c). Diabetes. 2025; 74.

Coleman, KD; McKinley, K; Ellison, AM; Alpern, ER; Hariharan, S; Topoz, I; Wurtz, M; Nielsen, B; Cook, LJ; Morris, CR; King, AA; Baumann, A; Frankenberger, W; Brousseau, DC. Associations Between Pain Scores and Opioid Doses With Emergency Department Disposition and Return Visit Rates in Children With Sickle Cell Disease. Pediatric Blood and Cancer. 2025; 72:e31750.

Costa, E; Ware, R; Tshilolo, L; Luzzatto, L. Thirty Years of Hydroxyurea for Sickle Cell Anemia - Scientific Progress, Global Health Gaps. The New England Journal of Medicine. 2025; 393:1556-1559.

Costa, E; Ware, RE; Tshilolo, L; Makani, J; Leufkens, HG M; Luzzatto, L. Globalization in clinical drug development for sickle cell disease. American Journal of Hematology. 2025; 100:4-9.

Davies, SM; Grimley, MS; Shrestha, A; Shova, A; Asnani, M; Kent, M; Sayani, F; Quinn, CT; Niss, O; Lutzko, CM; Witting, S; Latham, T; Bushman, FD; Malik, P. Gene Therapy with Reduced-Intensity Conditioning for Sickle Cell Disease. Transplantation and Cellular Therapy. 2025; 31:s2-s3.

Dei-Adomakoh, YA; Segbefia, CI; Latham, TS; Lane, AC; Dzefi-Tettey, K; Amissah-Arthur, K; Corquaye, O; Korang, L; Mensah, E; Ekpale, P; Seedah, D; Stuber, SE; Smart, LR; Ware, RE. Hydroxyurea for Children and Adults with Hemoglobin SC Disease. 2025; 4:EVIDoa2400402.

Dynan, L; Smith, RB; Quinn, CT. Inpatient and emergency healthcare utilization and payments for care of sickle cell disease: an analysis of all payers in Florida 2010-2019. 2025.

Eng, W; Iacobas, I; Perkins, J; Zampino, G; Leoni, C; Buonuomo, PS; Simonetti, A; Goel, H; Briones, M; Huang, M; Goldmacher, G; Liaw, D; Hammill, A. Safety findings from the phase 1/2 MOSAIC study of miransertib for patients with PIK3CA-related overgrowth spectrum or Proteus syndrome. Orphanet Journal of Rare Diseases. 2025; 20:375.

Engel, ER; Luchtman-Jones, L. Comment on: ""Comment on: Bivalirudin During Thrombolysis With Catheter-Directed tPA in a Heparin-Refractory Patient: A Case Report "": The Promise of Dilute Thrombin Time and Drug-Calibrated Assays for Improved Bivalirudin Monitoring. Pediatric Blood and Cancer. 2025; 72:e31418.

Goldenberg, NA; Schulman, S; Kittelson, JM; Abshire, TC; Casella, JF; Dale, R; Halperin, JL; Hanson, J; Kessler, CM; Manco-Johnson, MJ; McDevitt, L; Sidonio, RF; Spyropoulos, AC; Steg, PG; Bonaca, MP. Duration of anticoagulation for venous thromboembolism in pediatric patients: Evaluation of the Duration of Therapy for Thrombosis in Children (Kids-DOTT) trial outcomes at 2 years. Journal of Thrombosis and Haemostasis. 2025; 23:651-656.

Grimley, M; Davies, SM; Shrestha, A; Shova, A; Asnani, M; Kent, M; Sayani, F; Quinn, CT; Niss, O; Lutzko, C; Witting, S; Latham, T; Bushman, FD; Malik, P. Lentiviral gene therapy with reduced-intensity conditioning for sickle cell disease: a phase 1/2 trial. Nature Medicine. 2025; 31:2204-2212.

Gwarzo, I; Coleman, KD; Mckinley, K; Ellison, AM; Alpern, ER; Corboy, J; Hariharan, S; Topoz, I; Wurtz, M; Nielsen, B; King, AA; Baumann, A; Frankenberger, W; Brousseau, DC. Opioid Timeliness in the Emergency Department and Hospitalizations for Acute Sickle Cell Pain. JAMA pediatrics. 2025; 179:1194-1202.

Haacker, L; Littner, L; Martin, M; Brokamp, C; Beck, AF; Luchtman-Jones, L. Reply to: Comment on Anemia in Young Children and the Association With Socioeconomic Deprivation Indices. Pediatric Blood and Cancer. 2025; 72:e31743.

Haacker, L; Littner, L; Martin, M; Brokamp, C; Beck, AF; Luchtman-Jones, L. Anemia in Young Children and the Association With Socioeconomic Deprivation Indices. Pediatric Blood and Cancer. 2025; 72:e31663.

Hassanzadeh, S; Zeches, BA; Husami, A; Kalfa, TA; Knupp, CL; Richardson, MD. 467 Hereditary predisposition syndrome for myelodysplastic syndrome with SF3B1 mutation; a case report. American Journal of Clinical Pathology. 2025; 164:aqaf121.272.

Hau, DK; Fadhil, S; Lee, MH; Desderius, B; Willkens, M; Kalluvya, SE; Tebuka, E; Smart, LR; Peck, RN. Causes and Consequences of Persistent Anemia after 6 Months of Antiretroviral Therapy in Tanzania: An Observational Comparative Cohort Study. American Journal of Tropical Medicine and Hygiene. 2025; 112:234-241.

Hildenbrand, AK; Mara, CA; Murphy, B; Hood, AM; Johnson, Y; Shook, LM; Real, FJ; Nwankwo, C; Hackworth, R; Badawy, SM; Saving, KL; Treadwell, M; Quinn, CT; Crosby, LE. Engaging Parents of Children With Sickle Cell Disease in Shared Decision-Making for Hydroxyurea: The ENGAGE-HU Study. Pediatric Blood and Cancer. 2025; 72:e31639.

Iyer, VN; Provan, SA; Sexton, J; Vethanayagam, D; Christensen, IE; Al-Samkari, H; Kristianslund, EK; Beslow, LA; Kvien, TK; Chakinala, M; Decker, JE; Faughnan, ME; Galiatsatos, P; Gane, S; Geisthoff, U; White, AJ; Zhou, J; Friday, C; Clancy, M. Letter to the editor. European Archives of Oto-Rhino-Laryngology. 2025; 39:1-3.

Jan, S; Steinway, C; Belton, T; Shults, J; Bennett, L; Teng, O; Griffis, H; Aygun, B; Appiah-Kubi, A; Apollonsky, N; Andemariam, B; Mulchan, SS; Rubin, D; Smith-Whitley, K. Community Health Worker and Mobile Health Interventions for Quality of Life Among Young Adults With Sickle Cell Disease: A Randomized Clinical Trial. JAMA Network Open. 2025; 8:e2543571.

Kawtharany, H; Azzam, M; Alkhader, A; Betensky, M; Hamarsha, Q; Abou Zeid, HK; Mansour, R; Tabak, C; Patel, P; Baghdadi, SL; Woods, G; Zia, A; Monagle, P; Mustafa, RA. Efficacy and safety of thrombolysis for pediatric venous thromboembolism: a systematic review and meta-analysis. Blood Advances. 2025; 9:5512-5527.

Lang, AC; Mosley, C; Rosen, BL; Oke, E; Thompson, AA; Barriteau, C; Schwartz, L; Smith-Whitley, K; Crosby, LE; Shook, LM. Co-designing educational materials about SARS-CoV-2 (COVID-19) vaccines with individuals with sickle cell disease (SCD) and their families. 2025; 2.

Latham, TS; Czabanowska, K; Babich, S; Yego-Kosgei, F; Shook, LM; Ware, RE. Primary Stroke Screening and Hydroxyurea Treatment for Sickle Cell Anemia in Pediatric Healthcare Settings in East and Central Africa: A Narrative Review of Capacity Gaps and Opportunities. Public Health Reviews. 2025; 46:1608359.

Lenahan, SF; Blackmore, A; Fenchel, M; Thomas, E; Palumbo, JS; Tarango, C. Recurrent thrombosis and major bleeding in children treated for VTE. Blood Advances. 2025; 9:3824-3831.

Littner, L; Hu, J; Manuel, M; Gerber, C; Tarango, C. Inhibitors and Obesity: The Prevalence and Strength of Association in People With Haemophilia. Haemophilia. 2025; 31:1226-1235.

Littner, L; Thomas, E; Mooney, L; Doyle, J; Hendrickson, J; Tarango, C. Improving Time to Bleeding Disorder Treatment in the Emergency Department. Pediatric Blood and Cancer. 2025; 72:e31570.

Machogu, E; Aanyu-Tukamuhebwa, H; Mellencamp, K; Birungi, I; Namutangula, B; Namazzi, R; Latham, T; Opoka, R; Ware, R; Davis, SD; John, C. Benefits of Hydroxyurea in Mitigating Early Onset Lung Disease in Children With Sickle Cell Anemia. American Journal of Respiratory and Critical Care Medicine. 2025; 211:a1272.

Malec, L; Mathias, M; Dunn, AL; Nolan, B; Wong, N; Bystrická, L; Khan, U; Neill, G; Santagostino, E; Gunawardena, S; Tarango, C; Peyvandi, F. Post hoc analysis of bleeding episodes and clinically relevant pharmacokinetic parameters among children <12 years old with severe hemophilia A receiving once-weekly efanesoctocog alfa prophylaxis in the XTEND-Kids phase 3 multinational trial. Journal of Thrombosis and Haemostasis. 2025; 23:3481-3491.

Monagle, P; Azzam, M; Bercovitz, R; Betensky, M; Bhat, R; Biss, T; Branchford, B; Brandão, LR; Chan, AK C; Faustino, VE S; Wilson, HP; Woods, G; Zia, A; Mustafa, RA. American Society of Hematology/International Society on Thrombosis and Haemostasis 2024 updated guidelines for treatment of venous thromboembolism in pediatric patients. Blood Advances. 2025; 9:2587-2636.

Muteti, M; Akoko, E; Nyakundi, C; Mochamah, G; Makale, J; Ombati, P; Tawa, B; Nyanjom, S; Boateng, LA; Ware, RE; Williams, TN; Uyoga, S. Erythrocyte alloimmunization in children with sickle cell anemia living in Kilifi, Kenya. 2025; 1:100016.

Namazzi, R; Mellencamp, KA; Opoka, RO; Datta, D; Lima-Cooper, G; Liepmann, C; Sherman, J; Rodriguez, A; Kazinga, C; Ware, RE; Abreu, M; Schwantes-An, TH; John, CC; Conroy, AL. Circulating Immune Complexes and Glucose-6-Phosphate Dehydrogenase Deficiency Predict Recurrent Blackwater Fever in Ugandan Children With Severe Malaria. The Journal of Infectious Diseases. 2025; 232:285-297.

Nayak, RC; Emberesh, S; Trump, LR; Wellendorf, AM; Singh, AK; Korkmaz, B; Horwitz, MS; Myers, KC; Kalfa, TA; Lutzko, CM; Cancelas, JA. G-CSF resistance of ELANE-mutant neutropenia depends on SERF1-containing truncated-neutrophil elastase aggregates. The Journal of Clinical Investigation. 2025; 135:e177342.

Nieves, RM; Latham, T; Marte, N; Berges, M; Sánchez, LM; Urcuyo, G; Florencio, C; Gonzalez, C; del Villar, P; Chen, S; Schultz, WH; Lane, AC; Mena, R; Ware, RE. Stroke prevention in Hispanic children with sickle cell anemia: the SACRED trial. Blood Advances. 2025; 9:1791-1800.

Niss, O; Morin, CE; Hashemi, S; Alsaied, T; Lang, SM; Taylor, MD; Tasset, M; Malik, P; Quinn, CT. Longitudinal changes and predictors of cardiac extracellular volume fraction in sickle cell anemia. 2025; 1:100031.

Niss, O; Ware, RE; Quinn, CT. A New First-Line Option for Pediatric ITP-Incremental Progress or Paradigm Shift?. Journal of the American Medical Association (JAMA). 2025; 334:1802-1803.

Power-Hays, A; Namazzi, R; Dong, M; Kazinga, C; Kato, C; Aliwuya, S; McElhinney, K; Conroy, AL; Lane, A; John, C; Vinks, AA; Latham, T; Opoka, RO; Ware, RE. The feasibility of pharmacokinetic-based dosing of hydroxyurea for children with sickle cell anaemia in Uganda: Baseline results of the alternative dosing and prevention of transfusions trial. British Journal of Clinical Pharmacology. 2025; 91:1865-1872.

Power-Hays, A; Namazzi, R; Dong, M; Kazinga, C; Kato, C; Aliwuya, S; McElhinney, K; Conroy, AL; Lane, A; John, C; Vinks, AA; Latham, T; Opoka, RO; Ware, RE. The feasibility of pharmacokinetic-based dosing of hydroxyurea for children with sickle cell anaemia in Uganda: Baseline results of the alternative dosing and prevention of transfusions trial. British Journal of Clinical Pharmacology. 2025; 91:1865-1872.

Power-Hays, A; Namazzi, R; Kato, C; McElhinney, KE; Conroy, AL; Hume, H; John, C; O'Hara, SM; Stuber, SE; Lane, A; Latham, TS; Opoka, RO; Ware, RE. Pharmacokinetic-Guided Hydroxyurea to Reduce Transfusions in Ugandan Children with Sickle Cell Anemia: Study Design of the Alternative Dosing And Prevention of Transfusions Trial. Acta Haematologica. 2025; 148:208-219.

Quinn, CT; Ware, RE. The modern use of hydroxyurea for children with sickle cell anemia. Haematologica: the hematology journal. 2025; 110:1061-1073.

Quinn, CT; Ware, RE. The Voxelotor Effect: Decreased Affinity for New Drugs for Sickle Cell Disease?. Pediatric Blood and Cancer. 2025; 72:e31475.

Richmond, A; Hansen, S; Dykman, M; Adams, DM; Antaya, RJ; Bennett, JT; Frieden, IJ; Hammill, AM; Liang, MG; Maheshwari, M; Chamlin, S; Cotton, C; Lalor, L; Lee, LW. 44: Capillary Malformation-Arteriovenous Malformation Syndrome: Proposed Consensus Guidelines. Journal of Investigative Dermatology. 2025; 145:e12.

Rineer, S; Kwakye, D; Ambrose, EE; Jason, ID P; Smart, LR. Validation of Pediatric Self-Report Pain Scales in Sub-Saharan Africa: A Systematic Review. Journal of Pain and Symptom Management. 2025; 69:e103-e112.

Risinger, M; Zhang, W; Kalfa, TA. Advances on the genetic basis of red cell membrane disorders. Current Opinion in Hematology. 2025; 32:279-286.

Seither, K; Cortezzo, DE; McKinney, D; Ricci, K; Riddle, S. Congenital Lymphatic Malformations. NeoReviews.org. 2025; 26:e578-e586.

Shook, LM; Haygood, D; Quinn, CT. Clinical Utility of the Addition of Molecular Genetic Testing to Newborn Screening for Hemoglobinopathies for Confirmation of Alpha-Thalassemia Trait. International Journal of Neonatal Screening. 2025; 11:12.

Shook, LM; House, B; Farrell, CB; Stewart, R; Lanzkron, S; King, AA; Varughese, T; Strouse, JJ; Treadwell, M; Kanter, J. Challenges of Engaging Primary Care Providers in Specialized Telementoring Education About Sickle Cell Disease for Sickle Cell Specialists: Results from the Sickle Cell Disease Training and Mentoring Program for Primary Care Providers (STAMP) Project ECHO. AJPM Focus. 2025; 4:100304.

Shukla, RP; Aroosh, M; Cazelles, R; Ware, RE; Vinks, AA; Ben-Yoav, H. Microsensor Array for the Electrochemical Analysis of Hydroxyurea in Blood Samples of Children Affected by Sickle Cell Anemia. Analytical Chemistry. 2025; 97:24399-24414.

Sirotich, E; Chowdhury, SR; Modi, D; Guyatt, G; St John, M; Eisa, K; Cuker, A; Beck, CE; Grace, RF; Yan, JW; Kim, D; Thabane, L; Zeraatkar, D; Arnold, DM. Critical bleeding in adults and children with immune thrombocytopenia: a multicenter cohort study. Blood Advances. 2025; 9:3238-3248.

Sisk, BA; Eng, W; Aye, J; Mack, J; Tran, A; Ogino, J; Pearson, E; Bies, J; Smink, G; Shah, SD; Echols, T; Kumar, S; Fox, M; King, KA. Sociodemographic Characteristics of Patients Seen at Vascular Anomaly Centers in the United States. Pediatric Blood and Cancer. 2025; 72:e31661.

Thornburg, CD; van den Berg, HM; Chandler, M; Malec, L; Manuel, M; O'Neill, C; Recht, M; Taggart, E; Carpenter, SL; American Thrombosis and Hemostasis Network (ATHN) , . Inhibitor development and clinical characteristics in children with severe hemophilia A in the ATHN 8 US cohort study. 2025; 2:100082.

Todd, K; Hogue, SJ; Tweddell, JS; Reagor, JA; Mullins, E; Block, MG; Rosenfeldt, L; Francisco, B; Jodele, S; Sharma, BK; Lane, A; Slusher, C; Kharnaf, M; Morales, DL S; Palumbo, JS. Hemostatic derangements associated with cardiopulmonary bypass predict outcomes in pediatric patients undergoing corrective heart surgery. Journal of Thrombosis and Haemostasis. 2025; 23:492-503.

Tomlinson, GA; Backeljauw, P; Mochamah, G; Smart, LR; Latham, TS; Ware, RE; Williams, TN. Development of disease-specific growth curves from Kenyan children with sickle cell anemia. 2025; 1:100031.

TOZZO, V; CRAIG, J; OMOLOLU, SO; GENCO, M; SMITH, EP; THIBAULT, D; ARBABI, S; ABPLANALP, W; QUINN, CT; LINDSELL, CJ; BERGENSTAL, RM; FRANCO, RS; HIGGINS, J; COHEN, RM. 1013-P: Estimates of Mean Red Blood Cell Age (MRBC) Obtained with Continuous Glucose Monitoring (CGM)-Based Computational Methods Are Comparable to Those Obtained with Stable Isotope (SI) In Vivo Measurements. Diabetes. 2025; 74.

Vore, E; Cooke-Barber, J; Brungardt, J; Tiao, G; Hammill, A; Ricci, K; Patel, M; Dasgupta, R. Management, Trends, and Recommendations for Intra-abdominal Lymphatic Malformations: A Single-Center Retrospective Review. Journal of Pediatric Surgery. 2025; 60:162223.

Williams, TN; Ware, RE. Malaria prophylaxis in sickle cell anaemia: some answers, more questions. The Lancet Infectious Diseases. 2025; 25:601-602.

Wilson, HP; Mosha, M; Miller, A; Betensky, M; Amankwah, E; Fargo, J; Thornburg, CD; Tarango, C; Acharya, S; Male, C; Narang, S; Schulman, S; Goldenberg, NA. Secondary Anticoagulation Use in Patients < 21 Years Old following Primary Anticoagulant Treatment for Provoked Venous Thromboembolism: Findings from the Kids-DOTT Trial. Seminars in Thrombosis and Hemostasis. 2025; 51:343-347.

Zia, A; Nelson, MD; Ren, J; Zhang, S; Mattrey, RF; Han, BL; Hussain, T; Greer, JS; Al-Qahtani, M; Malone, K; Levine, B; Goldenberg, NA; Babb, TG; Zhang, S. The Functional Characterization of Venous Thromboembolic Disease (FUVID) study: rationale, design, and methods of a prospective, observational, multicenter study to evaluate mechanisms of exercise intolerance and dyspnea following pediatric pulmonary embolism. Research and Practice in Thrombosis and Haemostasis. 2025; 9:102669.