Technology Assessment: Report on the Evidence Regarding Off-Label Indications for Targeted Therapies used in Cancer Treatment

Technology Assessment: Report on the Evidence Regarding Off-Label Indications for Targeted Therapies used in Cancer Treatment
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Technology Assessment: Report on the Evidence Regarding Off-Label Indications for Targeted Therapies used in Cancer Treatment
Technology Assessment Report
Project ID: CANT1106

August 26, 2009

Duke Evidence-based Practice Center
Center for Clinical Health Policy Research

Authors
Amy P. Abernethy, MD
Remy R. Coeytaux, MD, PhD
Kenneth Carson, MD
Douglas McCrory, MD
Sally Y. Barbour, PharmD
Margaret Gradison, MD
R. Julian Irvine, MCM
Jane L. Wheeler, MSPH

This draft technology assessment is distributed solely for the purpose of public and peer review. It has not been otherwise disseminated by AHRQ. It does not represent and should not be construed to represent an AHRQ determination or policy.

This report is based on research conducted by the Duke Evidence-based Practice Center under contract to the Agency for Healthcare Research and Quality (AHRQ), Rockville, MD (Contract No. HHSA 290-02-0025). The findings and conclusions in this document are those of the authors who are responsible for its contents. The findings and conclusions do not necessarily represent the views of AHRQ. Therefore, no statement in this report should be construed as an official position of the Agency for Healthcare Research and Quality or of the U.S. Department of Health and Human Services.

None of the investigators has any affiliations or financial involvement related to the material presented in this report.

Contents
Table of Contents

Chapter 1. Background
Clinical Context
Technology Assessed
Policy Context
Scope of Study
Chapter 2. Methods
Selection Criteria
Search Strategy
Data Abstraction
Quality Assessment
Data Synthesis
Chapter 3. Results
Velocity of Evolution of the Literature
Systematic Review for Drug/Disease Combinations
Chapter 4. Discussion
References
Acronyms and Abbreviations

Appendix
Results of Systematic Literature Review for Specific Drug/Disease Combinations
Appendix Table of Contents
Alemtuzumab for Cutaneous T-Cell Lymphoma
Alemtuzumab for Non-Hodgkin Lymphoma
Alemtuzumab for T-Cell Prolymphocytic Leukemia
Bevacizumab for Breast Cancer
Bevacizumab for Epithelial Ovarian Cancer
Bevacizumab for Pancreatic Adenocarcinoma
Bevacizumab for Renal Cell Carcinoma
Bortezomib for Non-Hodgkin Lymphoma
Cetuximab for Pancreatic Adenocarcinoma
Erlotinib for Head and Neck Squamous Cell Carcinoma
Gefitinib for Head and Neck Squamous Cell Carcinoma
Imatinib Mesylate for Acute Lymphoblastic Leukemia
Imatinib Mesylate for Chronic Eosinophilic Leukemia
Imatinib Mesylate for Dermatofibrosarcoma Protuberans
Imatinib Mesylate for Myelodysplastic Syndrome
Imatinib for Systemic Mastocytosis
Rituximab for Chronic Lymphocytic Leukemia
Rituximab for Nodular Lymphocyte Predominant Hodgkin Disease
Rituximab for Waldenström’s Macroglobulinemia

Acronyms and Abbreviations
2-CDA 2-chlorodeoxyadenosine (cladribine)
4-DHAP dexamethasone, high-dose cytarabine, and cisplatin
AC Doxorubicin and cyclophosphamide
ADE adverse drug event
ADR adverse drug reaction
AE(s) adverse event(s)
AF atrial fibrillation
ALL acute lymphoblastic leukemia
Allo allograft
AML acute myelocytic leukemia
ANC absolute neutrophil count
ASCO American Society of Clinical Oncology
ASCT autologous stem cell transplant
ASH American Society of Hematology
AST/ALT aspartate aminotransferase/alanine transaminase
ATP adenosine triphosphate
AV atrioventricular
BC blast crisis
BCVA best correct visual acuity
BEAM bischloroethylnitrosourea, etoposide, cytarabine, and melphalan
Bili bilirubin
BID twice daily
BIW biweekly
BM bone marrow
BMN bone marrow necrosis
BMT bone marrow transplant
BP blood pressure
CA125 cancer antigen 125
CCR clinical complete response
CEL chronic eosinophilic leukemia
Chemo chemotherapy
CHF congestive heart failure
CHOP cyclophosphamide, hydroxydaunomycin, Oncovin®, and prednisone
CI confidence interval
CLL chronic lymphocytic leukemia
CML chronic myelogenous leukemia
CMML chronic myelomonocytic leukemia
CMV cytomegalovirus
CNS central nervous system
COG Children's Oncology Group
CR complete response
CRT cathode ray tube
CrCl creatinine clearance
Cru complete response unconfirmed
CT computerized tomography
CTC Common Toxicity Criteria
CTCL cutaneous T-cell lymphoma
CVAD cyclophosphamide, vincristine, Adriamycin®, and dexamethasone
CVP cyclophosphamide, vincristine, and prednisone
d/c discontinued
DCF deoxycoformycin
Dexa-BEAM dexamethasone, carmustine, etoposide, arabinoside C, and melphalan
DFCI Dana Farber Consortium Induction
DFS disease-free survival
DFSP dermatofibrosarcoma protuberans
DHAC dexamethasone, cytosine arabinoside, and carboplatin
DHAP dexamethasone, high-dose cytarabine, and cisplatin
DIV dexamethasone, Imatinib, and vincristine
DLBCL diffuse large B-cell lymphoma
DLI donor lymphocyte infusion
DVT/PE deep vein thrombosis / pulmonary embolism
ECOG Eastern Collaborative Oncology Group
EGFR epidermal growth factor receptor
EOC epithelial ovarian cancer
ER emergency room
ER estrogen receptor
ESHAP etoposide, cisplatin, cytarabine, and methylprednisolone
FDA Food and Drug Administration
FDG fluorodeoxyglucose
FIGO International Federation of Gynecology and Obstetrics
FMC fludarabine phosphate, mitoxantrone, and cyclophosphamide
FP FIP1L1-PDGFR
GBM glioblastoma multiforme
G-CSF granulocyte-specific colony-stimulating factor
GGT Gamma glutamyl transpeptidase
GFLIP gemcitabine, irinotecan, fluorouracil followed by leucovorin and cisplatin
GI gastrointestinal
GMALL German Multicenter Study Group for Adult ALL
GVHD graft versus host disease
HBV hepatitis B virus
HCV hepatitis C virus
HER2 human epidermal growth factor receptor
HES hyper eosinophilic syndrome
HG high grade
HIV human immunodeficiency virus
HLA human leukocyte antigen
H&N head and neck
HNSCC head and neck squamous cell cancers
HR-MDS high-risk myelodysplastic syndrome
HSTCL hepatosplenic T-cell non-Hodgkin lymphoma
HSV herpes simplex virus
HTN hypertension
IBC inflammatory breast cancer
ICE ifosfamide, carboplatin and etoposide
ICU intensive care unit
IFN interferon
IgM immunoglobulin M
IL interleukin
INV investigator
IRF independent research facility
IV intravenous
IVIG intravenous immunoglobulin
IWRC International Workshop Response Criteria
JALSG Japan Adult Leukemia Study Group
JC virus John Cunningham virus
KM curve Kaplan Meier curve
KS Kaposi Sarcoma
LABC locally advanced breast cancer
LDAC low-dose Ara-C
LFTs liver function tests
LVEF left ventricular ejection fraction
LyBC lymphoid blast crisis
MALT mucosa-associated lymphoid tissue
MCL mantle cell lymphoma
MDS myelodysplastic syndrome
MF mycosis fungoides or myelofibrosis
MI myocardial infarction
MINE mesna, ifosfamide, mitoxantrone, and etoposide
MM multiple myeloma
MRD minimal residual disease
MRI magnetic resonance imaging
MUD marrow unrelated donor
NCI National Cancer Institute
NCI-WG National Cancer Institute-sponsored Working Group
NHL non-Hodgkin lymphoma
NK natural killer
NLPHD nodular lymphocyte predominant Hodgkin's disease
NMST nonmyeloablative allogenic stem cell transplantation
NPC nasopharyngeal carcinoma
NR not reported
NYHA New York Heart Association
OIC oxaliplatin, irinotecan, and cetuximab
OR overall response
ORR overall response rate
OS overall survival
PD progressive disease
PCR pathological complete response
PDGFR platelet-derived growth factor receptor
PEG percutaneous endoscopic gastrostomy
PET positron emission tomography
PFI progression-free interval
PFS progression-free survival
Ph+ Philadelphia chromosome-positive
PLL prolymphocytic leukemia
PML progressive multifocal leukoencephalopathy
PO orally
PR partial response
PR+ progesterone receptor positive
PS performance status
PTL (PTCL) peripheral T-cell lymphoma
PTLD post-transplant lymphoproliferative disorder
PUVA photochemotherapy
Q every
QoL quality of life
RAEB refractory anemia with excess blasts
RBC red blood cell
RCC renal cell carcinoma
R-CHOP rituximab, cyclophosphamide, hydroxydaunomycin, Oncovin (vincristine), and prednisone
RCT randomized controlled trial
RECIST Response Evaluation Criteria In Solid Tumors
RFS relapse-free survival
RIC reduced intensity conditioning
RI-UCBT reduced intensity unrelated cord blood transplantation
RPTD recommended phase two dose
RR response rate
RSC Reed-Sternberg cells
RT radiation therapy
RT-PCR reverse transcription polymerase chain reaction
SC or SQ subcutaneous
SCC squamous cell carcinoma
SCR screen
SCT stem cell transplant
SCTCL subcutaneous T-cell lymphoma
SD stable disease
SLE systemic lupus erythematosus
SLL small lymphocytic lymphoma
SM systemic mastocytosis
SUV standardized uptake value
TBIL total bilirubin
TID thrice daily
TIW thrice weekly
TK tyrosine kinases
TMP/SMS trimethoprim/sulfamethoxazole
T-NHL T-cell non-Hodgkin lymphoma
T-PLL T-cell prolymphocytic leukemia
TRM transplantation-related mortality
TTP time to tumor progression
ULN per limit of normal
VCR vincristine
VEGF vascular endothelial growth factor
VHL von Hippel–Lindau
VZV varicella zoster virus
WBC white blood cell
WHO World Health Organization
WM Waldenström's macroglobulinemia
XRT x-ray therapy
y/o year(s) old