Dataset Information

Evaluating marker-guided treatment selection strategies.

ABSTRACT: A potential venue to improve healthcare efficiency is to effectively tailor individualized treatment strategies by incorporating patient level predictor information such as environmental exposure, biological, and genetic marker measurements. Many useful statistical methods for deriving individualized treatment rules (ITR) have become available in recent years. Prior to adopting any ITR in clinical practice, it is crucial to evaluate its value in improving patient outcomes. Existing methods for quantifying such values mainly consider either a single marker or semi-parametric methods that are subject to bias under model misspecification. In this article, we consider a general setting with multiple markers and propose a two-step robust method to derive ITRs and evaluate their values. We also propose procedures for comparing different ITRs, which can be used to quantify the incremental value of new markers in improving treatment selection. While working models are used in step I to approximate optimal ITRs, we add a layer of calibration to guard against model misspecification and further assess the value of the ITR non-parametrically, which ensures the validity of the inference. To account for the sampling variability of the estimated rules and their corresponding values, we propose a resampling procedure to provide valid confidence intervals for the value functions as well as for the incremental value of new markers for treatment selection. Our proposals are examined through extensive simulation studies and illustrated with the data from a clinical trial that studies the effects of two drug combinations on HIV-1 infected patients.

SUBMITTER: Matsouaka RA

PROVIDER: S-EPMC4213325 | biostudies-literature | 2014 Sep

REPOSITORIES: biostudies-literature

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Evaluating marker-guided treatment selection strategies.

Matsouaka Roland A RA Li Junlong J Cai Tianxi T

Biometrics 20140429 3

A potential venue to improve healthcare efficiency is to effectively tailor individualized treatment strategies by incorporating patient level predictor information such as environmental exposure, biological, and genetic marker measurements. Many useful statistical methods for deriving individualized treatment rules (ITR) have become available in recent years. Prior to adopting any ITR in clinical practice, it is crucial to evaluate its value in improving patient outcomes. Existing methods for q ...[more]

PMID: 24779731

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Project description:Background:Vertebral disease is a major cause of morbidity in 70% of patients diagnosed with multiple myeloma (MM). Associated osteolytic lesions and vertebral fractures are well documented in causing debilitating pain, functional restrictions, spinal deformity, and cord compression. Currently, treatment modalities for refractory MM spinal pain include systemic therapy, radiotherapy, cementoplasty (vertebroplasty/kyphoplasty), and radio frequency ablation. Our objectives were to report on the efficacy of existing treatments for MM patients with refractory spinal pain, to determine if a standardized treatment algorithm has been described, and to set the foundation upon which future prospective studies can be designed. Methods:A systematic search of the PubMed database was performed for studies relevant to the treatment of vertebral disease in MM patients. A multitude of search terms in various combinations were used, including but not limited to: "vertebroplasty," "kyphoplasty," "radiation," "multiple myeloma," "radiotherapy," and "radiosurgery." Results:Our preliminary search resulted in 219 articles, which subsequently resulted in 19 papers following abstract, title, full-text, and bibliography review. These papers were then grouped by treatment modality: radiotherapy, cementoplasty, or combination therapy. Significant pain and functional score improvement across all treatment modalities was found in the majority of the literature. While complications of treatment occurred, few were noted to be clinically significant. Conclusions:Treatment options-radiotherapy and/or cementoplasty-for vertebral lesions and pathologic fractures in MM patients demonstrate significant radiographic and clinical improvement. However, there is no consensus in the literature as to the optimal treatment modality as a result of a limited number of studies reporting head-to-head comparisons. One study did find significantly improved pain and functional scores with preserved vertebral height in favor of kyphoplasty over radiotherapy. When not contraindicated, we advocate for some form of cementoplasty. Further prospective studies are required before implementation of a standardized treatment protocol. Level of Evidence:5.

Dataset Information

Evaluating marker-guided treatment selection strategies.

Publications

Evaluating marker-guided treatment selection strategies.

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