Pediatrik Hastalıklarda Hedefe Yönelik ve Biyolojik Tedavilerin Yeni Çağı

Forrest SJ, et al. Precision medicine in pediatric oncology. Paediatr Drugs. 2018;20:13-23. PMID: 29189430. PMCID: PMC5770114.

Takita J. Precision medicine and molecular target drugs in pediatric acute lymphoblastic leukemia. Int J Hematol. 2020;112(3):353-360. PMID: 32624540.

Church AJ, et al. Molecular profiling identifies targeted therapy opportunities in pediatric solid tumors. Nat Med. 2022;28:125-134.

Jones LM, et al. Targeted therapy in pediatric acute myeloid leukemia: an evolving landscape. Paediatr Drugs. 2021;23(6):523-536. PMID: 34420195.

Obszański P, et al. Molecular targeted therapy of pediatric acute myeloid leukemia. Molecules. 2022;27(12):3720. PMID: 35745032.

Yi RC, Moran SK, Gantz HY, Strowd LC, Feldman SR. Biologics and small molecule targeted therapies for pediatric alopecia areata, psoriasis, atopic dermatitis, and hidradenitis suppurativa in the US: a narrative review. Children (Basel). 2024;11(8):892.Monti E, Cappellini MD. Targeted therapies for pediatric endocrine and metabolic diseases: principles and current evidence. Endocr Rev. 2024. PMID: 40234568.

Anderson GD. Developmental pharmacokinetics. Semin Pediatr Neurol. 2010;17(4):208-213. PMID: 21183126.

Funk RS, Brown JT, Abdel-Rahman SM. Pediatric pharmacokinetics: human development and drug disposition. Pediatr Clin North Am. 2012;59(5):1001-1016. PMID: 23036241.

Ferrara G, et al. Clinical use and molecular action of corticosteroids in the pediatric age. Int J Mol Sci. 2019;20(2):444. PMID: 30669566.

De Vleeschhauwer F, et al. Systemic adverse events associated with corticosteroids in children. Children (Basel). 2024;11(8):951.

Lejman M, Kuśmierczuk K, Bednarz K, Ostapińska K, Zawitkowska J. Targeted therapy in the treatment of pediatric acute lymphoblastic leukemia: therapy and toxicity mechanisms. Int J Mol Sci. 2021;22(18):9827. PMID: 34575992.

Bellantoni AJ, Wagner LM. The efficacy and evidence-based use of biologics in children and adolescents: monoclonal antibodies and fusion proteins as treatments. Pediatr Drugs. 2019;21:377-391. PMID: 31711560.

Wang D, Tang X, Shi Q, Wang R, Ji T, Tang X, Guo W. Denosumab in pediatric bone disorders and the role of RANKL blockade: a narrative review. Transl Pediatr. 2023;12(3):470-486. PMID: 37035391. PMCID: PMC10080477.

Perera A, Chugh N, Garcia-Bournissen F. Systematic review of adverse events of IL-1 and IL-6 inhibitor use in pediatrics. J Pediatr Pharmacol Ther. 2025;30(2):152-169. PMID: 40717752.

Zhou L, Wang Y, Xu Y, Zhang Y, Zhu C. Advances in AAV-mediated gene replacement therapy for pediatric monogenic neurological disorders. Mol Ther Methods Clin Dev. 2024;32:101357. PMID: 39559557.

El Sayed R, Shankar KM, Mankame AR, Cox CS Jr. Innovations in cell therapy in pediatric diseases: a narrative review. Transl Pediatr. 2023;12(6):1239-1257. PMID: 37427072.

Somekh I, Hendel A, Somech R. Evolution of gene therapy for inborn errors of immunity. JAMA Pediatr. 2024;178(7):645-646.

Laurent M, Geoffroy M, Pavani G, Guiraud S. CRISPR-based gene therapies: from preclinical to clinical treatments. Cells. 2024;13(10):800. PMID: 38786024.

Elzagallaai A, et al. Advancing precision medicine in paediatrics: past, present and future. Br J Clin Pharmacol. 2023;89:2152-2164. PMID: 37567890.

Giancane G, Alongi A, Ravelli A, et al. The treatment of juvenile idiopathic arthritis with biologic agents. Expert Rev Clin Immunol. 2018;14(1):57-73. PMID: 29166152.

Chinen J, Buckley RH. Network for primary immunodeficiency and targeted biologic therapy: optimizing immune modulation in children. Curr Opin Allergy Clin Immunol. 2020;20(6):567-576. PMID: 32901666.

Park JH, Rivière I, Gonen M, et al. Long-term follow-up of CD19 CAR therapy in pediatric acute lymphoblastic leukemia. N Engl J Med. 2018;378(5):449-459. PMID: 29385370.

Ortolano S, Garelli E, Bembi B. Enzyme replacement therapy in pediatric inherited metabolic diseases: benefits and limitations. Mol Genet Metab. 2019;128(1-2):75-92. PMID: 30686234.

Ali SS, Li Q, Agrawal PB. Implementation of multi-omics in diagnosis of pediatric rare diseases. Pediatr Res. 2025;97(4):1337-1344. PMID: 39562738.

Roman YM. Pharmacogenomics and rare diseases: optimizing drug development and personalized therapeutics. Pharmacogenomics. 2025;26(3-4):121-128. PMID: 40194983.

Makhlouf Y, Ayed HB, Miladi S, et al. Effect of biologic treatments on growth in children with juvenile idiopathic arthritis: a systematic review. PLoS One. 2025;20(5):e0324440.

Danziger-Isakov L, Sands A. Infections in children on biologics. Infect Dis Clin North Am. 2018;32(1):161-176. PMID: 29233577.

Toplak N, Uziel Y. Vaccination for children on biologics. Curr Rheumatol Rep. 2020;22(7):26. PMID: 32436130.

Randell RL, Becker ML. Safety updates in novel therapeutics for pediatric rheumatic disease. Curr Opin Rheumatol. 2021;33(5):403-408. PMID: 34397604. PMCID: PMC8826486.

Vaz BL, Marrinhas D, Pereira A. Biologic therapies and quality of life in pediatric patients with asthma: a systematic review. Healthcare (Basel). 2025;13(22):2824. PMID: 41302212.

Bleicher J, et al. Pediatric drug development: issues and challenges. J Clin Pharmacol. 2020;60(Suppl 2):S133-S144. PMID: 32718554.

Bernardini R, Toschi-Vespasiani G, Giannetti A. An overview of off-label use of humanized monoclonal antibodies in paediatrics. Medicina (Kaunas). 2022;58(5):625. PMID: 35630042. PMCID: PMC9144580.

Dipasquale V, Cucinotta U, Romano C. Biosimilars in pediatric IBD: updated considerations for disease management. Biologics. 2022;16:57-66. PMID: 35721798. PMCID: PMC9205321.

Biologic therapies in pediatric asthma: a clinical review of current advances. ClinicMed. 2025;6(5):1-4.

Mondillo G, Perrotta A, Masino M, Colosimo S, Frattolillo V, Abbate FG. Artificial intelligence and precision pharmacotherapy in pediatrics: a new paradigm in therapeutic decision-making. Therapeutics;3(1):6.

Forrest SJ, et al. Precision medicine in pediatric oncology. Paediatr Drugs. 2018;20:13-23. PMID: 29189430. PMCID: PMC5770114.

Takita J. Precision medicine and molecular target drugs in pediatric acute lymphoblastic leukemia. Int J Hematol. 2020;112(3):353-360. PMID: 32624540.

Church AJ, et al. Molecular profiling identifies targeted therapy opportunities in pediatric solid tumors. Nat Med. 2022;28:125-134.

Jones LM, et al. Targeted therapy in pediatric acute myeloid leukemia: an evolving landscape. Paediatr Drugs. 2021;23(6):523-536. PMID: 34420195.

Obszański P, et al. Molecular targeted therapy of pediatric acute myeloid leukemia. Molecules. 2022;27(12):3720. PMID: 35745032.

Yi RC, Moran SK, Gantz HY, Strowd LC, Feldman SR. Biologics and small molecule targeted therapies for pediatric alopecia areata, psoriasis, atopic dermatitis, and hidradenitis suppurativa in the US: a narrative review. Children (Basel). 2024;11(8):892.Monti E, Cappellini MD. Targeted therapies for pediatric endocrine and metabolic diseases: principles and current evidence. Endocr Rev. 2024. PMID: 40234568.

Anderson GD. Developmental pharmacokinetics. Semin Pediatr Neurol. 2010;17(4):208-213. PMID: 21183126.

Funk RS, Brown JT, Abdel-Rahman SM. Pediatric pharmacokinetics: human development and drug disposition. Pediatr Clin North Am. 2012;59(5):1001-1016. PMID: 23036241.

Ferrara G, et al. Clinical use and molecular action of corticosteroids in the pediatric age. Int J Mol Sci. 2019;20(2):444. PMID: 30669566.

De Vleeschhauwer F, et al. Systemic adverse events associated with corticosteroids in children. Children (Basel). 2024;11(8):951.

Lejman M, Kuśmierczuk K, Bednarz K, Ostapińska K, Zawitkowska J. Targeted therapy in the treatment of pediatric acute lymphoblastic leukemia: therapy and toxicity mechanisms. Int J Mol Sci. 2021;22(18):9827. PMID: 34575992.

Bellantoni AJ, Wagner LM. The efficacy and evidence-based use of biologics in children and adolescents: monoclonal antibodies and fusion proteins as treatments. Pediatr Drugs. 2019;21:377-391. PMID: 31711560.

Wang D, Tang X, Shi Q, Wang R, Ji T, Tang X, Guo W. Denosumab in pediatric bone disorders and the role of RANKL blockade: a narrative review. Transl Pediatr. 2023;12(3):470-486. PMID: 37035391. PMCID: PMC10080477.

Perera A, Chugh N, Garcia-Bournissen F. Systematic review of adverse events of IL-1 and IL-6 inhibitor use in pediatrics. J Pediatr Pharmacol Ther. 2025;30(2):152-169. PMID: 40717752.

Zhou L, Wang Y, Xu Y, Zhang Y, Zhu C. Advances in AAV-mediated gene replacement therapy for pediatric monogenic neurological disorders. Mol Ther Methods Clin Dev. 2024;32:101357. PMID: 39559557.

El Sayed R, Shankar KM, Mankame AR, Cox CS Jr. Innovations in cell therapy in pediatric diseases: a narrative review. Transl Pediatr. 2023;12(6):1239-1257. PMID: 37427072.

Somekh I, Hendel A, Somech R. Evolution of gene therapy for inborn errors of immunity. JAMA Pediatr. 2024;178(7):645-646.

Laurent M, Geoffroy M, Pavani G, Guiraud S. CRISPR-based gene therapies: from preclinical to clinical treatments. Cells. 2024;13(10):800. PMID: 38786024.

Elzagallaai A, et al. Advancing precision medicine in paediatrics: past, present and future. Br J Clin Pharmacol. 2023;89:2152-2164. PMID: 37567890.

Giancane G, Alongi A, Ravelli A, et al. The treatment of juvenile idiopathic arthritis with biologic agents. Expert Rev Clin Immunol. 2018;14(1):57-73. PMID: 29166152.

Chinen J, Buckley RH. Network for primary immunodeficiency and targeted biologic therapy: optimizing immune modulation in children. Curr Opin Allergy Clin Immunol. 2020;20(6):567-576. PMID: 32901666.

Park JH, Rivière I, Gonen M, et al. Long-term follow-up of CD19 CAR therapy in pediatric acute lymphoblastic leukemia. N Engl J Med. 2018;378(5):449-459. PMID: 29385370.

Ortolano S, Garelli E, Bembi B. Enzyme replacement therapy in pediatric inherited metabolic diseases: benefits and limitations. Mol Genet Metab. 2019;128(1-2):75-92. PMID: 30686234.

Ali SS, Li Q, Agrawal PB. Implementation of multi-omics in diagnosis of pediatric rare diseases. Pediatr Res. 2025;97(4):1337-1344. PMID: 39562738.

Roman YM. Pharmacogenomics and rare diseases: optimizing drug development and personalized therapeutics. Pharmacogenomics. 2025;26(3-4):121-128. PMID: 40194983.

Makhlouf Y, Ayed HB, Miladi S, et al. Effect of biologic treatments on growth in children with juvenile idiopathic arthritis: a systematic review. PLoS One. 2025;20(5):e0324440.

Danziger-Isakov L, Sands A. Infections in children on biologics. Infect Dis Clin North Am. 2018;32(1):161-176. PMID: 29233577.

Toplak N, Uziel Y. Vaccination for children on biologics. Curr Rheumatol Rep. 2020;22(7):26. PMID: 32436130.

Randell RL, Becker ML. Safety updates in novel therapeutics for pediatric rheumatic disease. Curr Opin Rheumatol. 2021;33(5):403-408. PMID: 34397604. PMCID: PMC8826486.

Vaz BL, Marrinhas D, Pereira A. Biologic therapies and quality of life in pediatric patients with asthma: a systematic review. Healthcare (Basel). 2025;13(22):2824. PMID: 41302212.

Bleicher J, et al. Pediatric drug development: issues and challenges. J Clin Pharmacol. 2020;60(Suppl 2):S133-S144. PMID: 32718554.

Bernardini R, Toschi-Vespasiani G, Giannetti A. An overview of off-label use of humanized monoclonal antibodies in paediatrics. Medicina (Kaunas). 2022;58(5):625. PMID: 35630042. PMCID: PMC9144580.

Dipasquale V, Cucinotta U, Romano C. Biosimilars in pediatric IBD: updated considerations for disease management. Biologics. 2022;16:57-66. PMID: 35721798. PMCID: PMC9205321.

Biologic therapies in pediatric asthma: a clinical review of current advances. ClinicMed. 2025;6(5):1-4.

Mondillo G, Perrotta A, Masino M, Colosimo S, Frattolillo V, Abbate FG. Artificial intelligence and precision pharmacotherapy in pediatrics: a new paradigm in therapeutic decision-making. Therapeutics;3(1):6.