Cheng Q, Cheng C, Crews KR, Ribeiro RC, Pui CH, Relling MV, Evans WE Epigenetic regulation of human gamma-glutamyl hydrolase activity in acute lymphoblastic leukemia cells. Am J Hum Genet. 2006 Aug;79(2):264-74. Epub 2006 Jun 6. Erratum in: Am J Hum Genet. 2010 Jul 9;87(1):161.
Cheng Q, Wu B, Kager L, Panetta JC, Zheng J, Pui CH, Relling MV, Evans WE A substrate specific functional polymorphism of human gamma-glutamyl hydrolase alters catalytic activity and methotrexate polyglutamate accumulation in acute lymphoblastic leukaemia cells. Pharmacogenetics. 2004 Aug;14(8):557-67.
Cheung YT, Eskind A, Inaba H, Hudson MM, Pui CH, Krull KR, Wolf J Association of Bacteremic Sepsis With Long-term Neurocognitive Dysfunction in Pediatric Patients With Acute Lymphoblastic Leukemia. JAMA Pediatr. 2018 Nov 1;172(11):1092-1095. doi: 10.1001/jamapediatrics.2018.2500.
Crews KR, Zhou Y, Pauley JL, Howard SC, Jeha S, Relling MV, Pui CH Effect of allopurinol versus urate oxidase on methotrexate pharmacokinetics in children with newly diagnosed acute lymphoblastic leukemia. Cancer. 2010 Jan 1;116(1):227-32. doi: 10.1002/cncr.24681.
Panetta JC, Sparreboom A, Pui CH, Relling MV, Evans WE Modeling mechanisms of in vivo variability in methotrexate accumulation and folate pathway inhibition in acute lymphoblastic leukemia cells. PLoS Comput Biol. 2010 Dec 2;6(12):e1001019. doi: 10.1371/journal.pcbi.1001019.
Pounds S, Cao X, Cheng C, Yang JJ, Campana D, Pui CH, Evans WE, Relling MV Integrated analysis of pharmacologic, clinical and SNP microarray data using Projection Onto the Most Interesting Statistical Evidence with Adaptive Permutation Testing. Int J Data Min Bioinform. 2011;5(2):143-57. doi: 10.1504/IJDMB.2011.039174.
Pui CH, Evans WE A 50-year journey to cure childhood acute lymphoblastic leukemia. Semin Hematol. 2013 Jul;50(3):185-96. doi: 10.1053/j.seminhematol.2013.06.007. Review.
Pui CH, Evans WE Treatment of acute lymphoblastic leukemia. N Engl J Med. 2006 Jan 12;354(2):166-78. Review.
Pui CH, Mullighan CG, Evans WE, Relling MV Pediatric acute lymphoblastic leukemia: where are we going and how do we get there? Blood. 2012 Aug 9;120(6):1165-74. doi: 10.1182/blood-2012-05-378943. Epub 2012 Jun 22. Review.
Pui CH, Nichols KE, Yang JJ Somatic and germline genomics in paediatric acute lymphoblastic leukaemia. Nat Rev Clin Oncol. 2019 Apr;16(4):227-240. doi: 10.1038/s41571-018-0136-6. Review.
Pui CH Recent research advances in childhood acute lymphoblastic leukemia. J Formos Med Assoc. 2010 Nov;109(11):777-87. doi: 10.1016/S0929-6646(10)60123-4. Review.
Reddick WE, Glass JO, Johnson DP, Laningham FH, Pui CH Voxel-based analysis of T2 hyperintensities in white matter during treatment of childhood leukemia. AJNR Am J Neuroradiol. 2009 Nov;30(10):1947-54. doi: 10.3174/ajnr.A1733. Epub 2009 Jul 30.
Roberson JR, Raju S, Shelso J, Pui CH, Howard SC Diabetic ketoacidosis during therapy for pediatric acute lymphoblastic leukemia. Pediatr Blood Cancer. 2008 Jun;50(6):1207-12. doi: 10.1002/pbc.21505.
Yang W, Treviño LR, Yang JJ, Scheet P, Pui CH, Evans WE, Relling MV ARID5B SNP rs10821936 is associated with risk of childhood acute lymphoblastic leukemia in blacks and contributes to racial differences in leukemia incidence. Leukemia. 2010 Apr;24(4):894-6. doi: 10.1038/leu.2009.277. Epub 2010 Jan 7.
Interventional studies are often prospective and are specifically tailored to evaluate direct impacts of treatment or preventive measures on disease.
Observational studies are often retrospective and are used to assess potential causation in exposure-outcome relationships and therefore influence preventive methods.
Expanded access is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot participate in a controlled clinical trial.
Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.
Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.