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.

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.

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.

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, 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.

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.

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.

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.

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; 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.

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 IN KILIFI, KENYA. 2025; 1:100016.

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.

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, DM E; 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.

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; Knoll, C; Reiss, U; Lucas, T; Balasa, V; Wheeler, A. 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.

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.