A phase I/II study of CY and topotecan in patients with high-risk malignancies undergoing autologous hematopoietic cell transplantation: The St Jude long-term follow-up

Bone Marrow Transplantation

Volumn 47, Issue 11, 2012, Pages 1448-1454

  Kasow, K A ^a   Stewart, C F ^b   Barfield, R C ^c   Wright, N L ^b   Li, C ^b   Srivastava, D K ^b   Leung, W ^b   Horwitz, E M ^d   Bowman, L C ^b   Handgretinger, R ^e   Hale, G A ^f  

^a UNIVERSITY OF NORTH CAROLINA   (United States)

^b ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

^c DUKE UNIVERSITY   (United States)

^d CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^e UNIVERSITY HOSPITAL TÜBINGEN   (Germany)

^f JOHNS HOPKINS ALL CHILDREN S HOSPITAL   (United States)

Author keywords

autologous BMT; CY; lymphoma; neuroblastoma; sarcoma; topotecan

Indexed keywords

CYCLOPHOSPHAMIDE; TOPOTECAN;

ABDOMINAL PAIN; ADENOVIRUS INFECTION; ADOLESCENT; ADULT; ALLERGY; ANEMIA; ANOREXIA; AREA UNDER THE CURVE; ARTICLE; AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION; BACKACHE; BLADDER SPASM; BLEEDING DISORDER; BRAIN TUMOR; CANCER RISK; CANCER SURVIVAL; CANDIDIASIS; CARDIOVASCULAR SYMPTOM; CELLULITIS; CHILD; CLOSTRIDIUM DIFFICILE INFECTION; COLITIS; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DEHYDRATION; DESQUAMATION; DIARRHEA; DISEASE CLASSIFICATION; DRUG CLEARANCE; DRUG DOSE ESCALATION; DRUG ERUPTION; DRUG FATALITY; DRUG FEVER; DRUG HYPERSENSITIVITY; DRUG INDUCED HEADACHE; DRUG MEGADOSE; DRUG TOLERABILITY; DYSPNEA; ENDOCRINE DISEASE; ENGRAFTMENT; ENTEROBACTERIACEAE INFECTION; EPISTAXIS; ESOPHAGITIS; EVENT FREE SURVIVAL; FEBRILE NEUTROPENIA; FEMALE; FOLLOW UP; GASTROINTESTINAL SYMPTOM; HALLUCINATION; HEARING IMPAIRMENT; HEART VENTRICLE EXTRASYSTOLE; HEART VENTRICLE TACHYCARDIA; HEMATOLOGIC DISEASE; HEMATURIA; HEMORRHAGIC CYSTITIS; HEPATOMEGALY; HERPES SIMPLEX; HERPES ZOSTER; HIGH RISK POPULATION; HUMAN; HYPERBILIRUBINEMIA; HYPERGLYCEMIA; HYPERTENSION; HYPERURICEMIA; HYPOCALCEMIA; HYPOKALEMIA; HYPOPHOSPHATEMIA; HYPOTENSION; HYPOXIA; IMMUNOPATHOLOGY; INAPPROPRIATE VASOPRESSIN SECRETION; INFECTION; KIDNEY DISEASE; KIDNEY FAILURE; LEUKOPENIA; LIVER DISEASE; LIVER TOXICITY; LIVER VEIN OBSTRUCTION; LUNG DISEASE; LUNG EDEMA; LUNG INFILTRATE; LUNG NODULE; LYMPHOMA; MAJOR CLINICAL STUDY; MALE; METABOLIC DISORDER; MUCOSA INFLAMMATION; MULTIPLE CYCLE TREATMENT; MYCOSIS; NAUSEA; NEPHROBLASTOMA; NEUROBLASTOMA; NEUROLOGIC DISEASE; NEUTROPENIA; OTITIS MEDIA; OVERALL SURVIVAL; PAIN; PANCREATITIS; PHARYNGITIS; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; PLEURA EFFUSION; PNEUMONIA; PRESCHOOL CHILD; PRIORITY JOURNAL; PROSPECTIVE STUDY; PRURITUS; PSEUDOMONAS INFECTION; RETINOBLASTOMA; SARCOMA; SCHOOL CHILD; SIDE EFFECT; SKIN MANIFESTATION; SKIN PAIN; STAPHYLOCOCCUS INFECTION; STOMATITIS; THROMBOCYTOPENIA; TREATMENT OUTCOME; TYPHLITIS; UREA NITROGEN BLOOD LEVEL; UROGENITAL TRACT DISEASE; URTICARIA; VEIN OCCLUSION; VOMITING;

ADOLESCENT; ANTINEOPLASTIC COMBINED CHEMOTHERAPY PROTOCOLS; CHILD; CHILD, PRESCHOOL; COMBINED MODALITY THERAPY; CYCLOPHOSPHAMIDE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; NEOPLASMS; RISK FACTORS; SURVIVAL RATE; TOPOISOMERASE I INHIBITORS; TOPOTECAN; TRANSPLANTATION, AUTOLOGOUS;

EID: 84869089853     PISSN: 02683369     EISSN: 14765365     Source Type: Journal    
DOI: 10.1038/bmt.2012.51     Document Type: Article

References (46)

1. Shih CS, Hale GA, Gronewold L, Tong X, Laningham FH, Gilger EA et al. High-dose chemotherapy with autologous stem cell rescue for children with recurrent malignant brain tumors. Cancer 2008; 112: 1345-1353. (Pubitemid 351429750)
2. Hale GA. Autologous hematopoietic stem cell transplantation for pediatric solid tumors. Expert Rev Anticancer Ther 2005; 5: 835-846. (Pubitemid 41599959)
3. London WB, Frantz CN, Campbell LA, Seeger RC, Brumback BA, Cohn SL et al. Phase II randomized comparison of topotecan plus cyclophosphamide versus topotecan alone in children with recurrent or refractory neuroblastoma: a Children's Oncology Group study. J Clin Oncol 2010; 28: 3808-3815.
4. Zage PE, Kletzel M, Marcus R, Castleberry R, Zhang Y, London WB et al. Outcomes of the POG 9340/9341/9342 trials for children with high-risk neuroblastoma: a report from the Children's Oncology Group. Pediatr Blood Cancer 2008; 51: 747-753.
5. Carol H, Houghton PJ, Morton CL, Kolb EA, Gorlick R, Reynolds CP et al. Initial testing of topotecan by the pediatric preclinical testing program. Pediatr Blood Cancer 2010; 54: 707-715.
6. Metzger ML, Stewart CF, Freeman III BB, Billups CA, Hoffer FA, Wu J et al. Topotecan is active against Wilms' tumor cells: results of a multi-institutional II phase study. J Clin Oncol 2007; 25: 3130-3136. (Pubitemid 47218062)
7. Daw NC, Santana VM, Iacono LC, Furman WL, Hawkins DR, Houghton PJ et al. Phase I and pharmacokinetic study of topotecan administered orally once daily for 5 days for 2 consecutive weeks to pediatric patients with refractory solid tumors. J Clin Oncol 2004; 22: 829-837. (Pubitemid 41103594)
8. Saylors RL, Stewart CF, Zamboni WC, Wall DA, Bell B, Stine KC et al. Phase I study of topotecan in combination with cyclophosphamide in pediatric patients with malignant solid tumors: a Pediatric Oncology Group study. J Clin Oncol 1998; 16: 945-952. (Pubitemid 28108727)
9. Santana VM, Furman WL, Billups CA, Hoffer F, Davidoff AM, Houghton PJ et al. Improved response in high-risk neuroblastoma with protracted topotecan administration. J Clin Oncol 2005; 23: 4039-4037.
10. National Cancer Institute Cancer Therapy Evaluation Program. Common Toxicity Criteria, Version 2.0, DCTD, NCI, NIH, DHHS. March 1998.
11. Simon R. Optimal two-stage designs for phase II clinical trials. Control Clin Trials 1989; 10: 1-10. (Pubitemid 19099520)
12. Kaplan EL, Meier P. Nonparametric estimation from incomplete observations. J Am Stat Assn 1958; 53: 457-481.
13. Mantel N. Evaluation of survival data and two new rank order statistics arising in its consideration. Cancer Chemother Rep 1966; 50: 163-170.
14. Kalbfleisch JD, Prentice RL. Relative risk (Cox) regression models. In: Kalbfleisch JD, Prentice RL (eds). The Statistical Analysis of Failure Time Data. John Wiley & Sons: New York, 1980, pp 163-188.
15. Gray RJ. A class of K-sample tests for comparing the cumulative incidence of a competing risk. Ann Stat 1988; 16: 1141-1154.
16. Hubbard KE, Schaiquevich P, Bai F, Fraga CH, Miller L, Panetta JC et al. Application of a highly specific and sensitive fluorescent HPLC method for topotecan lactone in whole blood. Biomed Chromatogr 2009; 23: 707-713.
17. Matthay KK, Villablanca JG, Seeger RC, Stram DO, Harris RE, Ramsay NK et al. Treatment of high-risk neuroblastoma with intensive chemotherapy, radiotherapy, autologous bone marrow transplantation, and 13-cis-retinoic acid. Children's Cancer Group. N Engl J Med 1999; 341: 1165-1173. (Pubitemid 29480654)
18. Ladenstein R, Potschger U, Le Deley MC, Whelan J, Paulussen M, Oberlin O et al. Primary disseminated multifocal Ewing sarcoma: results of the Euro-EWING-99 trial. J Clin Oncol 2010; 28: 3284-3291.
19. Presson A, Moore TB, Kempert P. Efficacy of high-dose chemotherapy and autologous stem cell transplant for recurrent Wilms' tumor: a metanalysis. J Pediatr Hematol Oncol 2010; 32: 454-461.
20. Stiff PJ, Agovi MA, Antman KH, Blaise D, Camitta BM, Cairo MS et al. High-dose chemotherapy with blood or bone marrow transplants for rhabdomyosarcoma. Biol Blood Marrow Transplant 2010; 16: 525-532.
21. Bradley MB, Cairo MS. Stem cell transplantation for pediatric lymphoma: past, present, and future. Bone Marrow Transplant 2008; 41: 149-158. (Pubitemid 351188912)
22. Dunkel IJ, Khakoo Y, Kernan NA, Gershon T, Gilheeney S, Lyden DC et al. Intensive multimodality therapy for patients with stage 4a metastatic retinoblastoma. Pediatr Blood Cancer 2010; 55: 55-59.
23. Hunold A, Weddeling N, Paulussen M, Ranft A, Liebscher C, Jü rgens H. Topotecan and cyclophosphamide in patients with refractory or relapsed Ewing tumors. Pediatr Blood Cancer 2006; 47: 795-800. (Pubitemid 44476756)
24. Panetta JC, Schaiquevich P, Santana VM, Stewart CF. Using pharmacokinetic and pharmacodynamic modeling and simulation to evaluate importance of schedule in topotecan therapy for pediatric neuroblastoma. Clin Cancer Res 2008; 14: 318-325. (Pubitemid 351378008)
25. Penson RT, Seiden MV, Goodman A, Fuller Jr AF, Berkowitz RS, Matulonis UA et al. Phase 1 trial of escalating doses of topotecan in combination with a fixed dose of pegylated liposomal doxorubicin in women with mü llerian malignancies. Gynecol Oncol 2004; 93: 702-707. (Pubitemid 38766059)
26. Chen AY, Choy H, Rothenberg ML. DNA topoisomerase 1-targeting drugs as radiation sensitizers. Oncology (Williston Park) 1999; 13: 39-46.
27. Cho LC, Choy H. Topoisomerase 1 inhibitors in the combined-modality therapy of lung cancer. Oncology (Williston Park) 2004; 18: 29-39.
28. Horn B, Reiss U, Matthay K, McMillan A, Cowan M. Veno-occlusive disease of the liver in children with solid tumors undergoing autologous hematopoietic progenitor cell transplantation: a high incidence in patients with neuroblastoma. Bone Marrow Transplant 2002; 29: 409-415. (Pubitemid 34257715)
29. Fraser CJ, Weigel BJ, Perentesis JP, Dusenbery KE, DeFor TE, Baker KS et al. Autologous stem cell transplantation for high-risk Ewing's sarcoma and other pediatric solid tumors. Bone Marrow Transplant 2006; 37: 175-181. (Pubitemid 43108810)
30. Cheuk DK, Wang P, Lee TL, Chiang AK, Ha SY, Lau YL et al. Risk factors and mortality predictors of hepatic veno-occlusive disease after pediatric hematopoietic stem cell transplantation. Bone Marrow Transplant 2007; 40: 935-944. (Pubitemid 350048191)
31. Kushner BH, Cheung NK, Kramer K, Dunkel IJ, Calleja E, Boulad F. Topotecan combined with myeloablative doses of thiotepa and carboplatin for neuroblastoma, brain tumors and other poor-risk solid tumors in children and young adults. Bone Marrow Transplant 2001; 28: 551-556. (Pubitemid 32976354)
32. de Jonge ME, Huitema AD, Beijnen JH, Rodenhuis S. High exposures to bioactivated cyclophosphamide are related to the occurrence of veno-occlusive disease of the liver following high-dose chemotherapy. Br J Cancer 2006; 94: 1226-1230.
33. McDonald GB, Slattery JT, Bouvier ME, Ren S, Batchelder AL, Kalhorn TF et al. Cyclophosphamide metabolism, liver toxicity, and mortality following hematopoietic stem cell transplantation. Blood 2003; 101: 2043-2048. (Pubitemid 36237612)
34. Donato ML, Aleman A, Champlin RE, Weber D, Alexanian R, Ippoliti CM et al. Highdose topotecan, melphalan, and cyclophosphamide (TMC) with stem cell support: a new regimen for the treatment of multiple myeloma. Leuk Lymphoma 2004; 45: 755-759. (Pubitemid 38256038)
35. Kazmi SM, Saliba RM, Donato M, Wang M, Hosing C, Qureshi S et al. Phase II trial of high-dose topotecan, melphalan, and CY with autologous stem cell support for multiple myeloma. Bone Marrow Transplant 2011; 46: 510-515.
36. Litzow MR, Peethambaram PP, Safgren SL, Keeney GL, Ansell SM, Dispenzieri A et al. Phase I trial of autologous hematopoietic SCT with escalating doses of topotecan combined with CY and carboplatin in patients with relapsed or persistent ovarian or primary peritoneal carcinoma. Bone Marrow Transplant 2010; 45: 490-497.
37. Donato ML, Aleman A, Champlin RE, Saliba RM, Wharton JT, Burke TW et al. Analysis of 96 patients with advanced ovarian carcinoma treated with high-dose chemotherapy and autologous stem cell transplantation. Bone Marrow Transplant 2004; 33: 1219-1224. (Pubitemid 38961480)
38. Gilheeney SW, Khakoo Y, Souweidane M, Wolden S, Boulad F, Dunkel IJ. Thiotepa/topotecan/carboplatin with autologous stem cell rescue in recurrent/refractory/poor prognosis pediatric malignancies of the central nervous system. Pediatr Blood Cancer 2010; 54: 591-595.
39. Bernbeck B, Bahci S, Meisel R, Troeger A, Schö nberger S, Laws HJ et al. Serial intense chemotherapy combining topotecan, etoposide, carboplatin and cyclophosphamide (RTECC) followed by autologous hematopoietic stem cell support in patients with high risk soft tissue sarcoma (STS). Klin Padiatr 2007; 219: 318-322. (Pubitemid 350239732)
40. Pérez-Martinez A, Lassaletta A, González-Vicent M, Sevilla J, D?́az MA, Madero L. High-dose chemotherapy with autologous stem cell rescue for children with high risk and recurrent medulloblastoma and supratentorial primitive neuroectodermal tumors. J Neurooncol 2005; 71: 33-38. (Pubitemid 40277251)
41. Pérez-Martinez A, Quintero V, Vicent MG, Sevilla J, D?́az MA, Madero L. High-dose chemotherapy with autologous stem cell rescue as first line of treatment in young children with medulloblastoma and supratentorial primitive neuroectodermal tumors. J Neurooncol 2004; 67: 101-106. (Pubitemid 38418255)
42. Crump M, Couban S, Meyer R, Rudinskas L, Zanke B, Gluck S et al. Phase II study of sequential topotecan and etoposide in patients with intermediate grade non-Hodgkin's lymphoma: a National Cancer Institute of Canada Clinical Trials Group study. Leuk Lymphoma 2002; 43: 1581-1587. (Pubitemid 34823480)
43. Steinherz PG, Shukla N, Kobos R, Steinherz L. Remission re-induction chemotherapy with clofarabine, topotecan, thiotepa, and vinorelbine for patients with relapsed or refractory leukemia. Pediatr Blood Cancer 2010; 54: 687-693.
44. Tiersten A, Selleck M, Smith DH, Wertheim I, Kaufman E, Hershman D et al. Phase I/II study of tandem cycles of high-dose chemotherapy followed by autologous hematopoietic stem cell support in women with advanced ovarian cancer. Int J Gynecol Cancer 2006; 16: 57-64.
45. McLeod HL, Krynetski EY, Relling MV, Evans WE. Genetic polymorphisms of thiopurinemethyltransferase and its clinical relevance for childhood acute lymphoblastic leukemia. Leukemia 2000; 14: 567-572. (Pubitemid 30195445)
46. Evans WE, Relling MV. Pharmacogenomics: translating functional genomics into rational therapeutics. Science 1999; 286: 487-491.