Title: Pediatric Oncology Research: The Impact of Collaborative Clinical Trials
1Pediatric Oncology ResearchThe Impact of
Collaborative Clinical Trials
- Mary Lou Schmidt, MD
- Head, Division of Pediatric Hematology/OncologyDe
partment of PediatricsUniversity of Illinois at
Chicago - College of Medicine
2Important Concepts
- Pediatric Oncology Partnerships have led to
dramatic gains against childhood cancer - 2/3 of survivors of childhood cancer have a major
disability by 25 years of age - 15-45 year olds have had NO improvement in their
survival from cancer in the last 30 years.
3Childhood Cancers Impact
- The leading cause of death by disease for
children lt 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
4Pediatric Oncology World Goal
- Improve the survival rates for childhood cancer
- Reduce the immediate and long-term side effects
including - second cancers, cognitive deficits,
musculoskeletal abnormalities, major organ
dysfunction (lungs, heart, kidneys, liver, etc.),
infertility
5Specific types of childhood cancer
- Hematologic acute leukemias-33
- lymphomas-12
- Solid tumors brain tumors-20
- muscle or bone tumors-12
- Solid Tumors that affect children lt 8 16
retinoblastoma, Wilms tumor, neuroblastoma
6The patients and families
75-Year Cancer Survival RatesU.S., 1960-1993
lt15 Year-Olds
A Bleyer
70
5-Year Survival
50
30
Modified from Landis SH et al, CA - Cancer J
Clin 486-29. 1998
1960-3
1970-3
1974-6
1980-2
1983-5
1986-93
1977-9
8Potential 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
9Improvement in Annual Cancer Mortality Rate among
U.S. Children lt15 Years of Age 1950-2000
A Bleyer
8
Mortality per 100,000, Age- Adjusted
6
( )
4
2
1950
1960
1970
1980
1990
10A Bleyer
IRSG
NWTSG
SWOG Pediatric Division
POG
CALGB Pediatric Division
CCG
1960
1970
1980
1990
2000
11The 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 240 other leading
academic medical centers - 7 Chicagoland COG programs
- UIC Rush ( Stroger)
- CMH, U of C, Loyola, Lutheran General, Christ
12Childrens Oncology Group Clinical Trials
Research Program
- Goal improve survival and decrease toxic side
effects by - comparing new experimental therapy to current
standard of care - 150 clinical trials currently available for
different diseases and patients - Each trial includes 30-2000 patients
- Trials frequently randomize patients between 2-4
different plans - 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
13Childrens Oncology GroupClinical 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
14When is a kid a kid?
- Why have children begun to survive at much higher
rates than adults? - Why have patients ages 15-45 had NO
- improvement in their survival in 30 years?
- How can we improve the survival rate for
adolescents and young adults?
15(No Transcript)
16AYA Patients
17Acute Lymphoblastic Leukemia Disease Free Survival
Stock W Sather H, Dodge RK, Bloomfield CD, Larson
A, Nachman J. Blood 96 467a, 2000.
DFS
16-20 Years (N 103)
CALGB
20-29 Years (N 123)
18(No Transcript)
19Relative Participation of U.S. Children and
Adults with Cancer on Clinical Trials
Participating in Clinical Trials
A Bleyer
100
80
60
40
20
0
Adults
Children
20Accrual to Cooperative Group Clinical Trials
10/97 to 9/98
A Bleyer
1855
2000
Number of Patients on Clinical Trials
1500
1263
997
1000
761
819
413
500
162
126
0
0-4
5-9
10-14
15-19
20-24
25-29
30-34
35-39
Age (Years)
21Estimated Proportion of Adolescent Young Adults
with Cancer on Clinical Trials
A Bleyer
Number of Patients
0-4
5-9
10-14
15-19
20-30
Age (Years)
22AYA Patients
23Impact of support
- Push all facets of research forward by improving
enrollment on all possible Children's Oncology
Group clinical trials at each local institution - Specifically focus on adolescents and young
adults - Educate the public and community-based
practitioners to refer adolescents and young
adults to the COG centers
24Funding for COG Clinical Trials
- National Institutes of Health? ? COG
- CureSearch website, tissue banks and research
labs, the 150 COG clinical trials - publish clinical trials 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 or philanthropists
25Requirements 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
26Immediate 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
27 Thank You!
28Questions?
- MARY LOU SCHMIDT, MD
- Head, Division of Pediatric Hematology/OncologyDe
partment of PediatricsUniversity of Illinois at
Chicago - College of Medicine
- telephone (312) 996-1791
- e-mail mls3_at_uic.edu