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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. Important Concepts.

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pediatric oncology research the impact of collaborative clinical trials

Pediatric Oncology Research:The Impact of Collaborative Clinical Trials

Mary Lou Schmidt, MD

Head, Division of Pediatric Hematology/OncologyDepartment of PediatricsUniversity of Illinois at Chicago

College of Medicine

important concepts
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
  • 15-45 year olds have had NO improvement in their survival from cancer in the last 30 years.
childhood cancer s impact
Childhood Cancer’s 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

pediatric oncology world goal
Pediatric Oncology WorldGoal
  • Improve the survival rates for childhood cancer
  • Reduce the immediate and long-term side effects causing deficits/disabilities:

cognitive musculoskeletal pulmonary fibrosis cardiomyopathy renal insufficiency infertility

Endocrinopathies second cancers

vision loss hearing loss

specific types of childhood cancer
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, Wilm’s tumor, neuroblastoma, hepatoblastoma

5 year cancer survival rates u s 1960 1993

Adults

5-Year Cancer Survival RatesU.S., 1960-1993

<15 Year-Olds

A Bleyer

70

%

5-Year

Survival

50

30

Modified from Landis SH et al, CA - Cancer J Clin 48:6-29. 1998

1960-3

1970-3

1974-6

1977-9

1980-2

1983-5

1986-93

potential areas for cancer research
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

improvement in annual cancer mortality rate among u s children 15 years of age 1950 2000

IRSG

NWTSG

SWOG Pediatric Division

POG

CALGB Pediatric Division

CCG

Improvement in Annual Cancer Mortality Rate among U.S. Children <15 Years of Age: 1950-2000

A Bleyer

8

Mortality per 100,000, Age-

Adjusted

6

( )

4

2

1950

1960

1970

1980

1990

slide10

'

Children

s

Oncology Group

C.

O.

G

A Bleyer

IRSG

NWTSG

SWOG Pediatric Division

POG

CALGB Pediatric Division

CCG

1960

1970

1980

1990

2000

the children s oncology group
The Children’s 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

children s oncology group clinical trials research program
Children’s 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: 30-2000 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

children s oncology group clinical trials research program succeeds because of
Children’s 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

cog members
COG Members
  • Pediatric Oncologists
  • Nurses
  • Clinical Research Associates
  • Radiologists
  • Surgeons (ophtho, ortho, neuro, peds)
  • Pathologists (cytogenetics, surgical)
  • Radiation Therapists
  • Pharm D’s (clinicians, researchers)
  • Social Workers, psychologists
  • Researchers: MD, PhD, MD/PhD’s
cog studies
COG Studies
  • Childhood Cancer Research Network Registry
  • track incidence, demographics for all new pediatric cancers
  • Obtain contact info and consent from patient and family to contact for future studies:

Epidemiology Quality of Life

Survivorship Late Effects

Fertility Insurance/Employment

Educational level Ethics/End of Life Care

acute lymphoblastic leukemia n 3000
Acute Lymphoblastic Leukemia (n=3000)
  • 20 open studies/14 for newly dx’d 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%)

all cog studies
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)
brain tumors n 2000
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
neuroblastoma n 500
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)

retinoblastoma n 250
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

  • 1st National RB studies in COG
  • Limited institutions includes UIC
  • Goal: Limit radiation, central review by Ophthalmologists & ocular pathologists
hodgkins lymphoma n 400
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

sarcomas n 750
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
ewings sarcoma
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

when is a kid a kid
When 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?
slide27

CCG-1800 Series 16-21 Year-Olds (N = 175)

68+2%

Acute 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)

relative participation of u s children and adults with cancer on clinical trials

Therapy at Centers Participating in Clinical Trials

Adults

Children

Relative Participation of U.S. Children and Adults with Cancer on Clinical Trials

Participating in Clinical Trials

A Bleyer

100%

80%

60%

40%

20%

0%

Children

Adults

slide30

Number of Patients with Cancer

Only 22 (of 761) entered onto

adult Cooperative Group trials

Accrual 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)

slide31

At Cooperative Group Institution;not on Cooperative Group Trial

On Cooperative Group Trial

The

Adolescent-Young Adult Gap

in Cancer Clinical Trials

40%

60%

33%

29%

11%

60%

50%

6%

10%

2%

Estimated 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)

to improve survival for aya pts
To improve survival for AYA pts
  • Improve access to COG clinical trials
  • Interface with oncologists caring for AYA pts
  • Provide support for AYA’s and MD’s

Nurse/CRA’s, Social Workers, Ped Onc MD back-up

benefit of inclusion in cog
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
funding for cog clinical trials
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

requirements for maximum participation in cog clinical trials
Requirements for maximum participation in COG Clinical Trials
  • Salary support for:

Principal Investigator/MD’s

Clinical Research Nurses

Regulatory Researchers

Community educator to improve referrals

  • Travel Funds to COG meetings
  • Supplies-computers, specimen submissions
cog @ uic rush stroger
COG @ 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 2nd performance site
  • 2009: UIC: 40 COG studies/

Rush: 8 COG studies/Stroger agreements being finalized

st baldrick s foundation
St. Baldrick’s Foundation
  • A thank you note can go a long way!
  • $ 550,000 (2006-2009) @ UIC/Rush/Stroger
  • Supports salaries for clinical research associates, RN’s, travel to COG meetings, supplies
immediate impact on childhood and adolescent young adult cancer
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
questions

Questions?

MARY LOU SCHMIDT, MD

Head, Division of Pediatric Hematology/OncologyDepartment of PediatricsUniversity of Illinois at Chicago

College of Medicine

telephone:  (312) 996-1791

e-mail: mls3@uic.edu