Rare Tumor Clinic: The University of California San Diego Moores Cancer Center Experience with a Precision Therapy Approach

Autor: Kellie Kurasaki, Shumei Kato, Sadakatsu Ikeda, Razelle Kurzrock
Rok vydání: 2017
Předmět:
Male
0301 basic medicine
Oncology
Gerontology
Cancer Research
medicine.medical_treatment
Medical Oncology
Ambulatory Care Facilities
Targeted therapy
0302 clinical medicine
Neoplasms
Antineoplastic Combined Chemotherapy Protocols
Molecular Targeted Therapy
Precision Medicine
Protein analyses
Ameloblastoma
Cancer
Tumor
Hazard ratio
Genomics
Middle Aged
Prognosis
Combined Modality Therapy
3. Good health
Survival Rate
Local
Lymphatic Metastasis
030220 oncology & carcinogenesis
Female
Sarcoma
Biotechnology
Adult
medicine.medical_specialty
Cancer Diagnostics and Molecular Pathology
Liver tumor
Oncology and Carcinogenesis
Vaccine Related
03 medical and health sciences
Rare Diseases
Clinical Research
Internal medicine
Genetics
medicine
Humans
Neoplasm Invasiveness
Oncology & Carcinogenesis
Retrospective Studies
Aged
Rare tumors
Neoplastic
Precision therapy
business.industry
Human Genome
Precision medicine
medicine.disease
Clinical trial
Neoplasm Recurrence
Orphan Drug
030104 developmental biology
Gene Expression Regulation
Immunization
business
Biomarkers
Follow-Up Studies
Zdroj: The Oncologist
The oncologist, vol 23, iss 2
Kato, S; Kurasaki, K; Ikeda, S; & Kurzrock, R. (2018). Rare Tumor Clinic: The University of California San Diego Moores Cancer Center Experience with a Precision Therapy Approach. ONCOLOGIST, 23(2), 171-178. doi: 10.1634/theoncologist.2017-0199. UC San Diego: Retrieved from: http://www.escholarship.org/uc/item/6rv4q83g
ISSN: 1549-490X
1083-7159
Popis: A Rare Tumor Clinic, emphasizing precision medicine, was recently initiated. This article describes preliminary experiences at the clinic and reports a case highlighting the successful use of ErbB2‐targeting therapy in an ultrarare tumor.
Background. Patients with rare tumors may lack approved treatments and clinical trial access. Although each rare tumor is uncommon, cumulatively they account for approximately 25% of cancers. We recently initiated a Rare Tumor Clinic that emphasized a precision medicine strategy. Materials and Methods. We investigated the first 40 patients presenting at the Rare Tumor Clinic. Next‐generation sequencing (NGS) of tissue and plasma‐derived, circulating‐tumor DNA (ctDNA), and protein markers were assessed. Results. Median age was 58 years (range, 31–78 years); 70% (28/40) were women; median number of previous systemic therapies was 2 (range 0–7). The most common diagnoses were sarcoma (n = 7) for solid tumors and Erdheim‐Chester disease (n = 5) for hematologic malignancies. Twenty distinct diagnoses were seen. Examples of ultrarare tumors included ameloblastoma, yolk sac liver tumor, ampullary cancer, and Castleman's disease. Altogether, 32 of 33 patients (97%) with tissue NGS and 15 of 33 (45%) with ctDNA sequencing harbored ≥1 alteration. Overall, 92.5% of patients (37/40) had ≥1 actionable target based on either genomic (n = 32) or protein (n = 27) markers. In total, 52.5% (21/40) received matched therapy; 52.4% (11/21) achieved stable disease (SD) ≥6 months (n = 3), partial remission (PR; n = 6), or complete remission (CR; n = 2). Matched therapy resulted in significantly longer progression‐free survival compared with last prior unmatched therapy (hazard ratio 0.26, 95% confidence interval 0.10–0.71, p = .008). Conclusion. Identifying genomic and protein markers in patients with rare/ultrarare tumors was feasible. When therapies were matched, >50% of patients attained SD ≥6 months, PR, or CR. Further precision medicine clinical investigations focusing on rare and ultrarare tumors are urgently needed. Implications for Practice. Although rare tumors are infrequent by definition, when all subtypes of rare cancers are combined, they account for approximately 25% of adult malignancies. However, patients with rare tumors may lack approved treatments and clinical trial access. This paper describes an institutional a Rare Tumor Clinic focused on a precision medicine strategy. Performing genomics and protein analyses was feasible amongst patients with rare cancers. Over 50% of patients attained SD ≥6 months, PR, or CR when they received matched therapy (genomically targeted and/or immunotherapy). Further studies investigating the efficacy of the precision therapy approach among rare tumors are warranted.
Databáze: OpenAIRE
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