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Pediatric Oncology Research: The Impact of Collaborative Clinical Trials Mary Lou Schmidt, MD Head, Division of Pediatric Hematology/Oncology Department.

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Presentation on theme: "Pediatric Oncology Research: The Impact of Collaborative Clinical Trials Mary Lou Schmidt, MD Head, Division of Pediatric Hematology/Oncology Department."— Presentation transcript:

1 Pediatric Oncology Research: The Impact of Collaborative Clinical Trials Mary Lou Schmidt, MD Head, Division of Pediatric Hematology/Oncology Department of Pediatrics University of Illinois at Chicago College of Medicine

2 Important Concepts Pediatric Oncology Partnerships have led to dramatic gains against childhood cancer: (age <15 yrs: survival 80%) 2/3 of survivors of childhood cancer have a major disability by 25 years of age year olds have had NO improvement in their survival from cancer in the last 30 years.

3 Childhood Cancers Impact The leading cause of death by disease for children < 15 years of age (greater than HIV, heart disease, cystic fibrosis, infection combined) 12,000 US cases/year, 160,000 cases worldwide 20% death rate in Western World (1 in 5 die) 65% of survivors have a major disability 25% of survivors have 3-4 disabilities/one of which is life-threatening

4 Pediatric Oncology World Goal Improve the survival rates for childhood cancer Reduce the immediate and long-term side effects causing deficits/disabilities: cognitive musculoskeletal pulmonary fibrosiscardiomyopathy renal insufficiencyinfertility Endocrinopathiessecond cancers vision losshearing loss

5 Specific types of childhood cancer Hematologic: acute leukemias-33% lymphomas-12% Solid tumors: brain tumors-20% muscle or bone tumors-12% Solid Tumors in children < 8yrs: 16% retinoblastoma, Wilms tumor, neuroblastoma, hepatoblastoma

6 The patients and families……

7 5-Year Cancer Survival Rates U.S., %5-YearSurvival Modified from Landis SH et al, CA - Cancer J Clin 48: <15 Year-Olds Adults A Bleyer

8 Potential Areas for Cancer Research Epidemiology: causes of cancer Pre-clinical studies Biology studies from clinical specimens Clinical Trials: Phase I, II, III Quality of Life Studies Late Effects Studies Disparities Studies End of Life Studies

9 Improvement in Annual Cancer Mortality Rate among U.S. Children <15 Years of Age: ( ) Mortality per 100,000, Age- Adjusted CCG NWTSG IRSG POG CALGB Pediatric Division SWOG Pediatric Division A Bleyer

10 O.O.O.O. C.C. G Oncology Group CCG NWTSG IRSG POG CALGB Pediatric Division SWOG Pediatric Division 2000 A Bleyer

11 The Childrens Oncology Group 250 institutions: US, Australia, Canada, Switzerland, the Netherlands and New Zealand including: St. Jude, Sloan-Kettering, Mayo Clinic, Harvard, Stanford, MD Anderson 7 Chicagoland COG programs: UIC + Rush (+ Stroger); CMH, U of C, Loyola, Lutheran General, Christ

12 Childrens Oncology Group Clinical Trials Research Program Goal: improve survival & decrease toxic side effects by comparing new experimental therapy to current standard of care 150 clinical trials currently available Each trial enrolls: patients Trials frequently randomize patients between 2-4 different arms Each trial must be locally approved and managed, With high quality data entered on time and on-line, And institutional audits passed every 3 years Results are published collaboratively

13 Childrens Oncology Group Clinical Trials Research Program Succeeds because of Full participation by all US Pediatric Oncologists in a Collaborative Spirit, using scarce shared resources which has led to amazing forward progress Fueling further research & education

14 COG Members Pediatric Oncologists Nurses Clinical Research Associates Radiologists Surgeons (ophtho, ortho, neuro, peds) Pathologists (cytogenetics, surgical) Radiation Therapists Pharm Ds (clinicians, researchers) Social Workers, psychologists Researchers: MD, PhD, MD/PhDs

15 COG Studies Childhood Cancer Research Network Registry a)track incidence, demographics for all new pediatric cancers b)Obtain contact info and consent from patient and family to contact for future studies: EpidemiologyQuality of Life SurvivorshipLate Effects FertilityInsurance/Employment Educational levelEthics/End of Life Care

16 Acute Lymphoblastic Leukemia (n=3000) 20 open studies/14 for newly dxd pts Studies: biology, ethnic differences, pharmacokinetics, therapeutic Clinical Trials: precursor B-cell ALL: infant (survival=30%) standard risk-(85%) high risk (65%) very high risk (40%) relapsed (late 50%), early (20%) T-cell (70%) B-cell (80%)

17 ALL COG Studies Use clinical/biologic markers to define risk and eligibility for therapeutic trials WBC, CSF +/-, testicular involve Leukemia cell cytogenetics Minimal residual disease markers when BMA shows remission Clinical Trial: alter Rx for best outcome and least late effects (randomize when possible)

18 Brain Tumors (n=2000) Medulloblastoma, astrocytoma, ependymoma, germ cell tumors Prognosis depends on surgical accessibility, histology, grading (+/-): (surv=0-80%) 13 open studies: bio, epi, banking, qol, Clinical trial: Radiation Therapy as variable: conformal, reduced dosing, combined with chemo Chemo: various regimens, temazolomide, intrathecal topotecan, high dose chemo/auto stem cell tx

19 Neuroblastoma (n=500) Low/intermediate risk: stages 1-4, 4s if age < 18mos, MYCN-non-amplified (surv=> 90%) with surgery +/- mod dose chemo Clinical trial: reduce therapy: minimize surgery (neonate, cord); only need 50% reduction in tumor, avoid RT High risk: stage 3,4 > 18 mos, any tumor with MYCN amplification (surv=30-50%) Clinical Trial: chemo/RT/auto transplant 1 vs. 2), retinoic acid, antibody therapy (yes vs no)

20 Retinoblastoma (n=250) 7 open protocols (epi, biology, therapeutic) Prognosis> 95% survival, 95% vision Clinical Trials: Unilateral dz: surg/path study Bilateral dz: chemo/thermoRx study Metastatic dz: chemo/RT/stem cell tx 1 st National RB studies in COG Limited institutions includes UIC Goal: Limit radiation, central review by Ophthalmologists & ocular pathologists

21 Hodgkins Lymphoma (n=400) 7 open COG studies Biology, tumor banking, quality of life, therapeutic studies: low (surv=98%), intermediate (90%), high risk dz (80%) and relapsed/refractory dz (11-50%) Clinical Trial: 1) eliminate radiation for rapid and complete responders 2) Intensify therapy for slow responders 3) Improve survivorship for relapsed dz

22 Sarcomas (n=750) Rhabdomyosarcoma: low risk: young, embryonal histology (surv= >90%) intermediate: older, alveolar (surv=68%) 9 studies-banking, bio, epi, therapeutic Clinical Trial: reduce RT for favorable dz, intensify therapy for less favorable dz

23 Ewings Sarcoma Localized-(surv=70%) with chemo Pulm mets only (surv=40%) with chemo/RT Bone mets (surv< 10%) Studies-bio, epi, therapeutic: Clinical Trial: high dose chemo/SCT for pts w/ EWS + pulm mets

24 When is a kid a kid? Why have children begun to survive at much higher rates than adults? Why have patients ages had NO improvement in their survival in 30 years? How can we improve the survival rate for adolescents and young adults?


26 AYA Patients

27 Acute Lymphoblastic Leukemia Disease Free Survival Years (N = 103) Years (N = 123) DFS CCG-1800 Series Year-Olds (N = 175) 68+2% Stock W Sather H, Dodge RK, Bloomfield CD, Larson A, Nachman J. Blood 96: 467a, CALGB


29 Relative Participation of U.S. Children and Adults with Cancer on Clinical Trials Participating in Clinical Trials 0% 20% 40% 60% 80% 100% AdultsChildren Therapy at Centers Participating in Clinical Trials Adults Children A Bleyer

30 Number of Patients with Cancer Accrual to Cooperative Group Clinical Trials 10/97 to 9/ Age (Years) Number of Patients on Clinical Trials Only 22 (of 761) entered onto adult Cooperative Group trials A Bleyer

31 At Cooperative Group Institution; not on Cooperative Group Trial On Cooperative Group Trial Estimated Proportion of Adolescent & Young Adults with Cancer on Clinical Trials 60% 2% 33% 29% 11% 6% 60% 40% 50% 10% Age (Years) Number of Patients The Adolescent- Young Adult Gap in Cancer Clinical Trials A Bleyer

32 AYA Patients

33 To improve survival for AYA pts Improve access to COG clinical trials Interface with oncologists caring for AYA pts Provide support for AYAs and MDs Nurse/CRAs, Social Workers, Ped Onc MD back-up

34 Benefit of inclusion in COG Push all facets of research forward by improving enrollment on all possible Children's Oncology Group clinical trials at each local institution (biol, therapeutic, epi, QOL, ethics, disparities) Improve survival rates: esp for adolescents and young adults Educate the public and community-based practitioners that all patients, esp AYA pts, can have improved survival if referred promptly for enrollment on COG trials

35 Funding for COG Clinical Trials National Institutes of Health $$$ COG: CureSearch website, tissue banks and research labs, conduct of 150 COG clinical trials, publishing costs for results auditing 250 COG institutions Estimated actual cost to the local COG institution: $ 7000/patient enrolled Each COG hospital/institution receives: ____________________________ $ 2400/patient enrolled Local Institutional Funding Gap Coverage: who pays? The treating institution and philanthropists

36 Requirements for maximum participation in COG Clinical Trials Salary support for: Principal Investigator/MDs Clinical Research Nurses Regulatory Researchers Community educator to improve referrals Travel Funds to COG meetings Supplies-computers, specimen submissions

37 UIC/Rush/Stroger 2007: UIC and Rush COG members in good standing (UIC 25 open protocols, Rush 5 open protocols), Stroger not in 8/07: UIC/Rush/Stroger with 55 faculty applied for joint COG membership 8/08: Merger accepted, UIC IRB began to re-review all protocols adding Rush as 2 nd performance site 2009: UIC: 40 COG studies/ Rush: 8 COG studies/Stroger agreements being finalized

38 St. Baldricks Foundation A thank you note can go a long way! $ 550,000 UIC/Rush/Stroger Supports salaries for clinical research associates, RNs, travel to COG meetings, supplies

39 Immediate Impact on Childhood and Adolescent/Young Adult Cancer Open the broadest pallet of COG clinical trials possible and fully participate in all aspects of COG Enroll, enroll, enroll all possible patients Collaborate, collaborate, collaborate

40 Thank You!

41 Questions? MARY LOU SCHMIDT, MD Head, Division of Pediatric Hematology/Oncology Department of Pediatrics University of Illinois at Chicago College of Medicine telephone: (312)

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