Population pharmacokinetics of sirolimus in pediatric patients with neurofibromatosis type 1

Therapeutic Drug Monitoring

Volumn 35, Issue 3, 2013, Pages 332-337

  Scott, Jeffrey R ^a   Courter, Joshua D ^a   Saldaña, Shannon N ^a,b,c   Widemann, Brigitte C ^d   Fisher, Michael ^e   Weiss, Brian ^b,c   Perentesis, John ^b,c   Vinks, Alexander A ^b,c  

^a Division of Pharmacy   (United States)

^b CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

^c UNIVERSITY OF CINCINNATI COLLEGE OF MEDICINE   (United States)

^d NATIONAL CANCER INSTITUTE   (United States)

^e CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

Author keywords

Neurofibromatosis; Pediatric; Pharmacokinetics; Sirolimus

Indexed keywords

RAPAMYCIN;

AFRICAN AMERICAN; AGE; ARTICLE; ASIAN; BODY HEIGHT; BODY SIZE; BODY SURFACE; BODY WEIGHT; CHILD; CLINICAL ARTICLE; DOSE TIME EFFECT RELATION; DRUG ABSORPTION; DRUG BLOOD LEVEL; DRUG CLEARANCE; DRUG METABOLISM; EUROPEAN AMERICAN; FEMALE; HUMAN; MALE; NEUROFIBROMATOSIS; PRESCHOOL CHILD; PRIORITY JOURNAL; RACE; SCHOOL CHILD; SEX; STEADY STATE; TREATMENT DURATION;

ADOLESCENT; AGE FACTORS; BAYES THEOREM; BODY SURFACE AREA; BODY WEIGHT; CHILD; CHILD, PRESCHOOL; DRUG MONITORING; FEMALE; HUMANS; IMMUNOSUPPRESSIVE AGENTS; MALE; MODELS, BIOLOGICAL; NEUROFIBROMATOSIS 1; NONLINEAR DYNAMICS; RETROSPECTIVE STUDIES; SIROLIMUS; TIME FACTORS;

EID: 84880063843     PISSN: 01634356     EISSN: 15363694     Source Type: Journal    
DOI: 10.1097/FTD.0b013e318286dd3f     Document Type: Article

References (35)

1. Williams VC, Lucas J, Babcock MA, et al. Neurofibromatosis type 1 revisited. Pediatrics. 2009;123:124-133.
2. Theos A, Korf BR. Pathophysiology of neurofibromatosis type 1. Ann Intern Med. 2006;144:842-849. (Pubitemid 46780620)
3. Korf BR. Plexiform neurofibromas. Am J Med Genet. 1999;89:31-37. (Pubitemid 30033174)
4. Korf BR. Malignancy in neurofibromatosis type 1. Oncologist. 2000;5: 477-485.
5. Needle MN, Cnaan A, Dattilo J, et al. Prognostic signs in the surgical management of plexiform neurofibroma: the Children's Hospital of Philadelphia experience, 1974-1994. J Pediatr. 1997;131:678-682. (Pubitemid 27524844)
6. Berven LA, Willard FS, Crouch MF. Role of the p70(S6K) pathway in regulating the actin cytoskeleton and cell migration. Exp Cell Res. 2004; 296:183-195. (Pubitemid 38624640)
7. Del Bufalo D, Ciuffreda L, Trisciuoglio D, et al. Antiangiogenic potential of the mammalian target of rapamycin inhibitor temsirolimus. Cancer Res. 2006;66:5549-5554. (Pubitemid 43927103)
8. Liu L, Li F, Cardelli JA, et al. Rapamycin inhibits cell motility by suppression of mTOR-mediated S6K1 and 4E-BP1 pathways. Oncogene. 2006;25:7029-7040. (Pubitemid 44722071)
9. Namba R, Young LJ, Abbey CK, et al. Rapamycin inhibits growth of premalignant and malignant mammary lesions in a mouse model of ductal carcinoma in situ. Clin Cancer Res. 2006;12:2613-2621.
10. Shaw RJ, Cantley LC. Ras, PI(3)K and mTOR signalling controls tumour cell growth. Nature. 2006;441:424-430. (Pubitemid 44050136)
11. Vignot S, Faivre S, Aguirre D, et al. mTOR-targeted therapy of cancer with rapamycin derivatives. Ann Oncol. 2005;16:525-537. (Pubitemid 40613318)
12. Wan X, Shen N, Mendoza A, et al. CCI-779 inhibits rhabdomyosarcoma xenograft growth by an antiangiogenic mechanism linked to the targeting of mTOR/Hif-1alpha/VEGF signaling. Neoplasia. 2006;8: 394-401. (Pubitemid 43830746)
13. Dasgupta B, Yi Y, Chen DY, et al. Proteomic analysis reveals hyperactivation of the mammalian target of rapamycin pathway in neurofibromatosis 1-Associated human and mouse brain tumors. Cancer Res. 2005; 65:2755-2760. (Pubitemid 40490077)
14. Johannessen CM, Reczek EE, James MF, et al. The NF1 tumor suppressor critically regulates TSC2 and mTOR. Proc Natl Acad Sci U S A. 2005;102:8573-8578. (Pubitemid 40862777)
15. Harris TE, Lawrence JC Jr. TOR signaling. Sci STKE. 2003;2003:re15.
16. Inoki K, Corradetti MN, Guan KL. Dysregulation of the TSC-mTOR pathway in human disease. Nat Genet. 2005;37:19-24. (Pubitemid 40070934)
17. Jacinto E, Hall MN. Tor signalling in bugs, brain and brawn. Nat Rev Mol Cell Biol. 2003;4:117-126. (Pubitemid 36172695)
18. Dansirikul C, Morris RG, Tett SE, et al. A Bayesian approach for population pharmacokinetic modelling of sirolimus. Br J Clin Pharmacol. 2006;62:420-434. (Pubitemid 44401525)
19. Schachter AD, Meyers KE, Spaneas LD, et al. Short sirolimus half-life in pediatric renal transplant recipients on a calcineurin inhibitor-free protocol. Pediatr Transplant. 2004;8:171-177. (Pubitemid 38508429)
20. Schachter AD, Benfield MR, Wyatt RJ, et al. Sirolimus pharmacokinetics in pediatric renal transplant recipients receiving calcineurin inhibitor cotherapy. Pediatr Transplant. 2006;10:914-919. (Pubitemid 44683635)
21. Anglicheau D, Le Corre D, Lechaton S, et al. Consequences of genetic polymorphisms for sirolimus requirements after renal transplant in patients on primary sirolimus therapy. Am J Transplant. 2005;5:595-603. (Pubitemid 40313352)
22. Aspeslet LJ, Yatscoff RW. Requirements for therapeutic drug monitoring of sirolimus, an immunosuppressive agent used in renal transplantation. Clin Ther. 2000;22(suppl B):B86-B92. (Pubitemid 30306048)
23. Djebli N, Rousseau A, Hoizey G, et al. Sirolimus population pharmacokinetic/pharmacogenetic analysis and Bayesian modelling in kidney transplant recipients. Clin Pharmacokinet. 2006;45:1135-1148. (Pubitemid 44631392)
24. MacDonald A, Scarola J, Burke JT, et al. Clinical pharmacokinetics and therapeutic drug monitoring of sirolimus. Clin Ther. 2000;22(suppl B): B101-B121. (Pubitemid 30306050)
25. Zimmerman JJ, Kahan BD. Pharmacokinetics of sirolimus in stable renal transplant patients after multiple oral dose administration. J Clin Pharmacol. 1997;37:405-415. (Pubitemid 27215068)
26. Korf B. A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas. Bethesda, MD: National Library of Medicine; 2012. Available at: http://clinicaltrials.gov/ct2/show/ NCT00634270. Accessed December 21, 2012.
27. Franz DN, Leonard J, Tudor C, et al. Rapamycin causes regression of astrocytomas in tuberous sclerosis complex. Ann Neurol. 2006;59: 490-498. (Pubitemid 43358069)
28. Proost JH, Meijer DK. MW/Pharm, an integrated software package for drug dosage regimen calculation and therapeutic drug monitoring. Comput Biol Med. 1992;22:155-163.
29. Anderson BJ, Holford NH. Mechanistic basis of using body size and maturation to predict clearance in humans. Drug Metab Pharmacokinet. 2009;24:25-36.
30. Kleiber M. Body size and metabolic rate. Physiol Rev. 1947;27: 511-541.
31. Holford NH. A size standard for pharmacokinetics. Clin Pharmacokinet. 1996;30:329-332. (Pubitemid 26177179)
32. Tejani A, Alexander S, Ettenger R, et al. Safety and pharmacokinetics of ascending single doses of sirolimus (Rapamune, rapamycin) in pediatric patients with stable chronic renal failure undergoing dialysis. Pediatr Transplant. 2004;8:151-160. (Pubitemid 38508426)
33. Schubert M, Venkataramanan R, Holt DW, et al. Pharmacokinetics of sirolimus and tacrolimus in pediatric transplant patients. Am J Transplant. 2004;4:767-773. (Pubitemid 38637576)
34. Le Meur Y, Djebli N, Szelag JC, et al. CYP3A53 influences sirolimus oral clearance in de novo and stable renal transplant recipients. Clin Pharmacol Ther. 2006;80:51-60. (Pubitemid 43946845)
35. Adams D. Safety and Efficacy Study of Sirolimus in Complicated Vascular Anomalies. Bethesda, MD; 2012. Available at: http://clinicaltrials.gov/show/ NCT00975819. Accessed December 21, 2012.