Page: 1 of 12. Bone Turnover Markers for Diagnosis and Management of Osteoporosis and Diseases Associated With High Bone Turnover

Similar documents
Page: 1 of 12. Bone Turnover Markers for Diagnosis and Management of Osteoporosis and Diseases Associated With High Bone Turnover

Bone Turnover Markers for the Diagnosis and Management of Osteoporosis and Diseases Associated with High Bone Turnover. Original Policy Date

POLICY PRODUCT VARIATIONS DESCRIPTION/BACKGROUND RATIONALE DEFINITIONS BENEFIT VARIATIONS DISCLAIMER CODING INFORMATION REFERENCES POLICY HISTORY

Bone Turnover Markers for Diagnosis and Management of Osteoporosis and Diseases Associated with High Bone Turnover

Clinician s Guide to Prevention and Treatment of Osteoporosis

COLLAGEN CROSSLINKS AND BIOCHEMICAL MARKERS OF BONE TURNOVER

COLLAGEN CROSSLINKS AND BIOCHEMICAL MARKERS OF BONE TURNOVER

Annual Rheumatology & Therapeutics Review for Organizations & Societies

COLLAGEN CROSSLINKS AND BIOCHEMICAL MARKERS OF BONE TURNOVER

COLLAGEN CROSSLINKS AND BIOCHEMICAL MARKERS OF BONE TURNOVER

Elecsys bone marker panel. Optimal patient management starts in the laboratory

European Journal of Endocrinology (1997) ISSN

Ca, Mg metabolism, bone diseases. Tamás Kőszegi Pécs University, Department of Laboratory Medicine Pécs, Hungary

Number: Last Review 06/09/2016 Effective: 09/25/2001 Next Review: 06/08/2017. Review History. Definitions

The Role of the Laboratory in Metabolic Bone Disease

CASE 1 WHY IS IT IMPORTANT TO TREAT? FACTS CONCERNS

Name of Policy: Zoledronic Acid (Reclast ) Injection

Sponsor / Company: sanofi-aventis and Proctor & Gamble Drug substance(s): Risedronate (HMR4003)

OSTEOPOROSIS: PREVENTION AND MANAGEMENT

nogg Guideline for the diagnosis and management of osteoporosis in postmenopausal women and men from the age of 50 years in the UK

Osteoporosis. Overview

Markers of bone turnover for the prediction of fracture risk and monitoring of osteoporosis treatment: a need for international reference standards

NEW DEVELOPMENTS IN OSTEOPOROSIS: SCREENING, PREVENTION AND TREATMENT

8/6/2018. Glucocorticoid induced osteoporosis: overlooked and undertreated? Disclosure. Objectives. Overview

This Coverage Policy applies to Individual Health Insurance Marketplace benefit plans only.

New Developments in Osteoporosis: Screening, Prevention and Treatment

International Journal of Health Sciences and Research ISSN:

DEVELOPMENT OF A RISK SCORING SYSTEM TO PREDICT A RISK OF OSTEOPOROTIC VERTEBRAL FRACTURES IN POSTMENOPAUSAL WOMEN

Osteoporosis and Lupus. Andrew Ruthberg, MD University Rheumatologists

WHAT KEEPS OUR BONES STRONG?

Pharmacy Management Drug Policy

Overview. Bone Biology Osteoporosis Osteomalacia Paget s Disease Cases. People Centred Positive Compassion Excellence

Pharmacy Management Drug Policy

Whole Body Dual X-Ray Absorptiometry to Determine Body Composition

Factors related to bone forming inadequate response to treatment (teriparatide/pth 1-84) in patients with severe osteoporosis. Preliminary results

Additional Research is Needed to Determine the Effects of Soy Protein on Calcium Binding and Absorption NDFS 435 3/26/2015. Dr.

Correlation between Thyroid Function and Bone Mineral Density in Elderly People

Forteo (teriparatide) Prior Authorization Program Summary

Collagen Crosslinks, Any Method

Oral Alendronate Vs. Three-Monthly Iv Ibandronate In The Treatment Of Postmenopausal Osteoporosis

Collagen Crosslinks, Any Method

Metabolic Bone Disease Related to Chronic Kidney Disease

Breast Cancer and Bone Loss. One in seven women will develop breast cancer during a lifetime

O. Bruyère M. Fossi B. Zegels L. Leonori M. Hiligsmann A. Neuprez J.-Y. Reginster

sad EFFECTIVE DATE: POLICY LAST UPDATED:

Comparison of Bone Density of Distal Radius With Hip and Spine Using DXA

Interpreting DEXA Scan and. the New Fracture Risk. Assessment. Algorithm

Name of Policy: Boniva (Ibandronate Sodium) Infusion

Bone strength is proportional to bone mass, measured with DXA. Bone turnover markers indicate the status of bone quality.

Osteoporosis. Open Access. John A. Kanis. Diseases, University of Sheffield, UK

Pharmacy Management Drug Policy

Osteoporosis/Fracture Prevention

Cross-reference: MP Whole Body Dual X-Ray Absorptiometry (DEXA) to Determine Body Composition MP Bone Mineral Density

Monitoring hormone replacement therapy by biochemical markers of bone metabolism in menopausal women

Assessment and Treatment of Osteoporosis Professor T.Masud

ASJ. How Many High Risk Korean Patients with Osteopenia Could Overlook Treatment Eligibility? Asian Spine Journal. Introduction

Product: Denosumab (AMG 162) Clinical Study Report: month Primary Analysis Date: 21 November 2016 Page 1

FORTEO (teriparatide) INJECTION

OSTEOPOROSIS AND WHAT TO DO AFTER A VERTEBRAL FRACTURE. Lydia Au Geriatrics Ng Teng Fong Hospital

Osteoporosis: An Overview. Carolyn J. Crandall, MD, MS

TYMLOS (abaloparatide)

Response of several markers of bone collagen degradation to calcium supplementation in postmenopausal women with low calcium intake

Alendronate sodium in the management of osteoporosis

Bone Mass Measurement BONE MASS MEASUREMENT HS-042. Policy Number: HS-042. Original Effective Date: 8/25/2008

[If no, skip to question 10.] Y N. 2. Does the member have a diagnosis of Paget s disease of bone? Y N. [If no, skip to question 4.

3. Has bone specific alkaline phosphatase level increased OR does the member have symptoms related to active Paget s?

Coordinator of Post Professional Programs Texas Woman's University 1

denosumab (Prolia ) Policy # Original Effective Date: 07/21/2011 Current Effective Date: 04/19/2017

Based on review of available data, the Company may consider the use of denosumab (Prolia) for the

OSTEOPOROSIS IN MEN. Nelson B. Watts, MD OSTEOPOROSIS AND BONE HEALTH SERVICES CINCINNATI, OHIO

Bone and Mineral. Comprehensive Menu for the Management of Bone and Mineral Related Diseases

Download slides:

Chau Nguyen, D.O. Rheumatologist Clinical Assistant Professor of Internal Medicine at Western University of Health Sciences

Vitamin D and calcium are required at the time of denosumab administration during osteoporosis treatment

Advanced DXA Using TBS insight

Skeletal Manifestations

Parathyroid Hormone Analogs

Effects of Anti RANK ligand Denosumab on Beta Thalassemia induced osteoporosis

Outline. Osteoporosis Definition DXA* Osteoporosis is Common. Brittle bones: Pitfalls in the evaluation and management of osteoporosis

9/26/2016. The Impact of Dietary Protein on the Musculoskeletal System. Research in dietary protein, musculoskeletal health and calcium economy

Cancer. Description. Section: Surgery Effective Date: October 15, 2016 Subsection: Original Policy Date: September 9, 2011 Subject:

Use of DXA / Bone Density in the Care of Your Patients. Brenda Lee Holbert, M.D. Associate Professor Senior Staff Radiologist

NICE SCOOP OF THE DAY FRAX with NOGG. Eugene McCloskey Professor of Adult Bone Diseases University of Sheffield

Task Force Co-Chairs. Members

Bone turnover markers and prediction of bone loss in elderly women

Osteoporosis challenges

Disclosure and Conflicts of Interest Steven T Harris MD Osteoporosis Diagnosis: BMD, FRAX and Assessment of Secondary Osteoporosis

An audit of osteoporotic patients in an Australian general practice

BMD: A Continuum of Risk WHO Bone Density Criteria

Contractor Number 03201

Treatments for Osteoporosis Expected Benefits, Potential Harms and Drug Holidays. Suzanne Morin MD FRCP FACP McGill University May 2014

New Therapeutic Directions: Osteoanabolic and Antiresorptive Therapy in Combination Therapy and in Sequence

Osteoporosis Update. Greg Summers Consultant Rheumatologist

Page 1. New Developments in Osteoporosis. What s New in Osteoporosis

Chapter 5: Evaluation and treatment of kidney transplant bone disease Kidney International (2009) 76 (Suppl 113), S100 S110; doi: /ki.2009.

Purpose. Methods and Materials

Medical Policy. MP Vertebral Fracture Assessment With Densitometry

What is Osteoporosis?

Efficacy of risedronate in men with primary and secondary osteoporosis: results of a 1-year study

Transcription:

Last Review Status/Date: December 2014 Page: 1 of 12 Management of Osteoporosis and Diseases Description Bone turnover markers are biochemical markers of either bone formation or bone resorption. Commercially available tests are used to assess some of these markers in urine and/or serum by high performance liquid chromatography (HPLC) or immunoassay. Assessment of bone turnover markers is proposed to supplement bone mineral density (BMD) measurement in the diagnosis of osteoporosis and aid in treatment decisions. Bone turnover markers could also potentially be used to evaluate treatment effectiveness before changes in BMD can be observed. Background After cessation of growth, bone is in a constant state of remodeling (or turnover), with initial absorption of bone by osteoclasts followed by deposition of new bone matrix by osteoblasts. This constant bone turnover is critical to the overall health of the bone, by repairing microfractures and remodeling the bony architecture in response to stress. Normally, the action of osteoblasts and osteoclasts is balanced, but bone loss occurs if the two processes become uncoupled. Bone-turnover markers (BTMs) can be categorized as bone-formation markers or bone-resorption markers, and can be identified in serum and/or urine. Table 1 summarizes the various bone-turnover markers. Table 1 Formation Markers Serum osteocalcin (OC) Serum total alkaline phosphatase (ALP) Serum bone specific alkaline phosphatase (B-ALP) Serum procollagen I carboxyterminal propeptide (PICP) Resorption Markers Serum and urinary hydroxyproline (Hyp) Urinary total pyridinoline (Pyr) Urinary total deoxypyridinoline (dpyr) Urinary-free pyridinoline (f-pyr, also known as Pyrilinks )

Page: 2 of 12 Serum procollagen type 1 N-terminal propeptide (PINP) Bone sialoprotein Urinary-free deoxypyridinoline (f-dpyr, also known as Pyrilinks-D ) Serum and urinary collagen type I cross-linked N-telopeptide (NTx, also referred to as Osteomark) Serum and urinary collagen type I cross-linked C-telopeptide (CTx, also referred to as Cross Laps ) Serum carboxyterminal telopeptide of type I collagen (ITCP) Tartrate-resistant acid phosphatase (TRAP or TRACP) There is interest in the use of bone turnover markers to evaluate age-related osteoporosis, a disease characterized by slow, prolonged bone loss, resulting in an increased risk of fractures at the hip, spine, or wrist. Currently, fracture risk is primarily based on measurements of BMD in conjunction with other genetic and environmental factors, such as family history of osteoporosis, history of smoking, and weight. It is thought that the level of bone turnover markers may also predict fracture risk, possibly through a different mechanism than that associated with BMD. However, it must be emphasized that the presence of bone turnover markers in the serum or urine is not necessarily related to bone loss. For example, even if bone turnover is high, if resorption is balanced with formation, there will be no net bone loss. Bone loss will only occur if resorption exceeds formation. Therefore, bone turnover markers have been primarily studied as an adjunct, not an alternative, to measurements of BMD to estimate fracture risk and document the need for preventive or therapeutic strategies for osteoporosis. In addition, bone turnover markers might provide a more immediate assessment of treatment response and predict change in BMD in response to treatment. Treatment-related changes in BMD occur very slowly. This fact, coupled with the precision of BMD technologies, suggested that clinically significant changes in BMD could not be reliably detected until at least 2 years. In contrast, changes in bone turnover markers could be anticipated after 3 months of therapy. Bone-turnover markers have been researched as markers of diseases associated with markedly high levels of bone turnover, such as Paget s disease, primary hyperparathyroidism, and renal osteodystrophy. Regulatory Status Several tests for bone turnover markers have been cleared by the U.S. Food and Drug Administration (FDA) using the 510(k) process. 1995: Pyrilinks test (Metra Biosystems, Santa Clara, CA) measures collagen type 1 cross-link, pyridinium 1996: Osteomark test (Ostex International, Seattle, WA) measures cross-linked N-telopeptides of type 1 collagen (NTx) 1999: Serum Crosslaps One-step ELISA test measures hydroxyproline

Page: 3 of 12 2000: Ostase (Beckman Coulter) measures bone-specific alkaline phosphatase (B-ALP) 2001: N-MID Osteocalcin One-Step ELISA (Osteometer Bio Tech) measures osteocalcin (OC) Related Policies 6.01.01 Bone Mineral Density Studies Policy *This policy statement applies to clinical review performed for pre-service (Prior Approval, Precertification, Advanced Benefit Determination, etc.) and/or post-service claims. Measurement of bone turnover markers is considered not medically necessary in the diagnosis and management of age-related osteoporosis. Measurement of bone turnover markers is considered investigational in the management of patients with conditions associated with high rates of bone turnover, including but not limited to Paget disease, primary hyperparathyroidism and renal osteodystrophy. Rationale In general, to be considered clinically useful, studies need to demonstrate that tests for bone turnover markers are accurate and reliable and that their use can result in improved health outcomes. For example, to evaluate their utility for diagnosing osteoporosis as an adjunct to BMD measurements with dual energy x-ray absorptiometry (DXA), studies would moreover need to show that bone turnover markers independently predict fracture risk beyond BMD and that the additional information provided by information on bone turnover has the potential to influence treatment decisions and clinical outcomes. Similarly, to be considered useful for monitoring osteoporosis treatment beyond follow-up BMD measurements, bone turnover test results would need to impact the decision to continue or change treatment in a way that leads to improved patient outcomes. Following is a summary of key literature on bone turnover markers published to date: Diagnosis and management of osteoporosis Do bone turnover markers independently predict fracture risk beyond BMD measurements? Few studies have directly addressed the question of whether any bone turnover markers are independent predictors of fracture risk. One study conducted in men and one conducted in women are described next. A 2013 analysis of population-based data in Japan included postmenopausal women and adjusted for BMD. (1) The study involved baseline surveys, bone turnover marker assessment and BMD measurements, and 3 follow-ups over 10 years. At baseline, 851 women who participated were aged

Page: 4 of 12 50 years or older and were eligible for vertebral fracture assessment. Of these, 730 women had BMD measurements taken at the initial examination and at 1 or more follow-ups. Women with early menopause (ie, <40 years old), with a history or illness or medication known to affect bone metabolism and with incomplete data were excluded. After exclusions, 522 women included in the analysis. Over a median follow-up period of 10 years, 81 of 522 women (15.5%) were found on imaging to have an incident vertebral fracture. Seventy-eight of the 81 women with radiographically detected vertebral fractures were more than 5 years from menopause at baseline. Risk of incident vertebral fractures adjusted for BMD T-scores was significantly associated with several bone turnover markers, specifically ALP, urinary total deoxypyridinoline (tdpd) and urinary free deoxypyridinoline (IPPD). For example, in a multivariate model adjusting for a variety of covariates including femoral neck BMD, the risk of developing a fracture per SD of change in ALP was increased by 33% (risk ratio, 1.33; 95% CI, 1.06 to 1.66). Risk of incident vertebral fracture was not significantly associated with other bone turnover markers including osteocalcin (OC) and crosslinked C-telopeptide (CTx). It is not clear how generalizable findings from this study are; that is, the association between subsequent fracture risk and certain bone turnover markers, and the lack of association between fracture risk and other bone turnover markers. This study is also limited by the large number of women excluded from analysis due to incomplete data. In men, a subanalysis of prospectively-collected data from the Osteoporotic Fractures in Men (MrOS) study also included adjustment of BMD. (2) Baseline levels of bone turnover markers were compared in 384 men, age 65 years or older, who had nonspine fractures over an average follow-up of 5 years with 885 men without nonspine fracture. A second analysis compared 72 hip-fracture cases and 993 controls without hip fracture. After adjusting for age and recruitment site, the association between nonspine fracture and quartile of the bone turnover marker procollagen type 1 N-terminal propeptide (PINP) was statistically significant (for each analysis, p<0.05 was used). The associations between nonspine fracture and quartiles of the 2 other bone turnover markers, beta C-terminal cross-linked telopeptide of type 1 collagen (b-ctx) and tartrate-resistant acid phosphatase 5b (TRACP5b) were not statistically significant. Moreover, in the analysis adjusting only for age and recruitment site, when the highest quartile of bone turnover markers was compared with the lower 3 quartiles, the risk of nonspine and hip fractures was significantly increased for PINP and c-ctx but not TRACP5b. After additional adjustment for baseline BMD, or baseline BMD and other potential confounders, there were no statistically significant relationships between any bone turnover marker and fracture risk. The authors concluded that their results do not support the routine use of bone turnover markers to assess fracture risk in older men when there is the option of measuring hip BMD. Systematic reviews have examined the association between bone turnover markers and fracture risk, but have not included analyses on the additional predictive value beyond BMD. For example, a 2014 meta-analysis by Johansson et al focused on the markers PINP and CTx and examined their ability to predict future fracture risk. (3) The review included 10 prospective cohort studies in which bone turnover markers were measured at baseline and incident fractures were recorded. Pooled analyses were performed on a subset of these studies. A meta-analysis of 3 studies found a statistically significant association between baseline PINP and subsequent fracture risk (hazard ratio [HR]=95%

Page: 5 of 12 confidence interval [CI], 1.09 to 1.39). Similarly, a meta-analysis of 6 studies found an association between CTx and fracture risk (HR=1.18, 95%, 1.09 to 1.29). None of the individual studies adjusted for BMD, and consequently the pooled analyses do not reflect the ability of bone turnover markers to predict fracture risk beyond BMD. A previous systematic review, published in 2012 by Biver et al, did not find a statistically significant association between another bone turnover marker, OC and fracture risk. (4) When findings from 3 studies were pooled, the mean difference in OC levels in patients with and without vertebral fractures was 1.61 ng/ml (95% CI, -0.59 to 3.81). Both systematic reviews noted a high degree of heterogeneity among the published studies identified. Section Summary: Some studies have found statistically significant associations between bone turnover markers and fracture risk, but there is insufficient literature on any specific marker. For example, an analysis of MrOS data found a significant association between PINP and risk of nonspine fracture in men, and the JPOS study from Japan found a significant association between ALP, tdpd and fdpd and risk of incident vertebral fracture in women. Moreover, there is insufficient evidence that any bone turnover marker is an independent predictor of fracture risk, beyond BMD. Do bone turnover markers independently predict response to osteoporosis treatment? Studies have also examined the ability of bone turnover markers to evaluate response to osteoporosis treatment. For example, a subanalysis of the randomized Fracture Intervention Trial (n=6184) by Bauer et al found that pretreatment levels of the bone turnover marker PINP significantly predicted the anti-fracture efficacy of alendronate. (5) Over a mean follow-up of 3.2 years, there were 492 non-spine and 294 vertebral fractures. Compared to those in the placebo group, the efficacy of alendronate for reducing non-spine fractures was significantly greater in women who were in the highest tercile of PINP (>56.8 ng/ml) than those in the lowest tercile (<41.6 ng/ml). Baseline bone turnover rates were not associated with alendronate efficacy in reducing vertebral fractures. The authors indicated that this result needed confirmation in additional studies, and, even if verified, the impact on treatment recommendations is not clear. A small randomized trial of an osteoporosis treatment (n=43) found that urinary cross-linked N-terminal telopeptides provided a more sensitive measure of treatment response than serum levels. (6) Another small randomized trial from Japan measured levels of OC in response to osteoporosis treatment in 109 postmenopausal women. (7) The authors found that undercarboxylated osteocalcin (uc-oc) levels in serum were significantly lower at 1 month in the group receiving active treatment for osteoporosis compared to the control intervention; the implication for fracture prevention was not studied. A 2011 systematic review by Funck-Brentano et al addressed the issue of whether early changes in serum biochemical bone turnover markers predict the efficacy of osteoporosis therapy. (8) Their review included 24 studies that presented correlations between bone turnover markers and the outcomes of fracture risk reduction or change in BMD. Five studies (including the Bauer study, previously described) reported on fracture risk and 20 studies reported on BMD changes. The review authors discussed study findings qualitatively but did not pool study results. The evidence did not

Page: 6 of 12 support a correlation between short-term changes in bone turnover markers and fracture risk reduction. In addition, few studies were available on this topic, leading to the conclusion that bone turnover markers have shown limited value as a technique to monitor osteoporosis therapy. An additional study on this topic was published by Baxter et al in 2013. (9) This was a retrospective review of data on 200 patients commencing treatment with bisphosphonates for osteoporosis or osteopenia requiring treatment. The investigators found statistically significant inverse correlation between change in urine NTx at 4 months and change in spine BMD at 18 months (Pearson productmoment correlation coefficient r=0.33, p<0.001). There was not a significant association between change in urine NTx and hip BMD. Section Summary: The available evidence on the association between any specific bone turnover marker and response to osteoporosis treatment is limited in quantity and quality. While some individual studies have reported positive correlations for markers, such as PINP in the Fracture Intervention Trial, a body of evidence in support of any specific marker is lacking. As a result, the evidence is insufficient to conclude whether bone turnover markers are an independent predictor of treatment response. Does information provided by bone turnover markers improve treatment decisions and/or improve health outcomes? To provide clinical utility, bone turnover markers would need to provide information beyond that offered by BMD measurements that has an impact on treatment decisions and/or leads to improved health outcomes. Bone turnover markers can be measured more frequently than BMD and thus could potentially provide information with clinical utility. For example, the 2013 guideline from the National Osteoporosis Foundation states that biochemical markers of bone turnover can be used to predict the extent of fracture risk reduction when measured 3-6 months after starting FDA-approved osteoporosis treatments. (10) Several randomized controlled trials (RCTs) have addressed the issue of whether measurement of bone turnover markers can improve adherence to oral bisphosphonate treatment. A 2014 systematic review identified 5 RCTs and did not find significant differences in compliance rates between groups that did and did not receive feedback on bone turnover marker test results. (11) Study data were not pooled. The authors noted a high baseline compliance rate that limited the studies ability to detect an impact of feedback. As an example, a 2011 industry-sponsored study by Roux et al from France randomized physicians to manage patients on oral monthly ibandronate with a collagen crosslinks test (CTx) or usual care. (12) In the CTx group, bone marker assessment was done at baseline and week 5 for the week 6 visit, a standardized message was delivered to patients regarding change in CTX since baseline. If the decrease in CTX was more than 30% of the baseline value, they were told that the treatment effect was optimal. If not, they were told that the treatment effect was suboptimal, and they were given additional advice. Patients told they had a suboptimal response were retested with CTx at week 13 for the week 14 visit.

Page: 7 of 12 The primary outcome was the proportion of patients who were adherent at 1 year. After 1 year, rates of adherence to ibandronate were 74.8% in the CTx group and 75.1% in the usual care group; the difference between groups was not statistically significant (p=0.93). There was also not a statistically significant difference in the proportion of patients having taken at least 10 out of 12 pills; 82.4% in the CTx group and 80.0% in the usual care group. In this study, monitoring bone markers and providing this information to patients did not improve adherence to oral osteoporosis medication. Section Summary: There is a limited amount of evidence on the impact of bone turnover markers on management of osteoporosis. Individual RCTs and a meta-analysis of these RCTs have not found that feedback on bone turnover marker results improves adherence rates. No studies were identified that evaluate whether the use of bone turnover markers lead to management changes that are expected to improve outcomes. Management of other conditions associated with high rates of bone turnover There is little published literature on use of bone turnover markers in the management of conditions associated with high rates of bone turnover, such as Paget disease, primary hyperparathyroidism, and renal osteodystrophy. Moreover, very few studies on this topic have been published since 2000. One recent study, published in 2012 by Rainon et al, reported on 198 patients with primary hyperparathyroidism who underwent parathyroidectomy. (13) The authors found a statistically significant association (p<0.05) between pre-operative serum OC levels and persistent postoperative elevation of parathyroid hormone 6 months after the surgery. In addition, several studies published 2000 or later were identified that tested bone turnover levels in patients with Paget disease before and after treatment with bisphosphonates. (14-16) For example, Alvarez et al found that the mean values of bone markers decreased significantly after bisphosphonate treatment in 31 of 38 patients who completed a 3-month course of oral bisphosphonates. Bone markers measured in the Alvarez study included serum total alkaline phosphatase (ALP), serum bone-specific alkaline phosphatase (B-ALP), and PINP and urinary hydroxyproline (Hyp), CTx and NTx. No studies were identified that addressed whether bone turnover markers for these conditions associated with high bone turnover resulted in improved patient management decisions or health outcomes. Practice Guidelines and Position Statements In 2014, the National Osteoporosis Foundation updated their guideline for prevention and treatment of osteoporosis. (10) Regarding biochemical markers of bone turnover, the guideline states: Biochemical markers of bone turnover may: Predict risk of fracture independently of bone density Predict extent of fracture risk reduction when repeated after 3-6 months of treatment with FDAapproved therapies. Predict magnitude of BMD increases with FDA-approved therapies. Predict rapidity of bone loss.

Page: 8 of 12 Help determine adequacy of patient compliance and persistence with osteoporosis therapy. Help determine duration of 'drug holiday' and when and if medication should be restarted (Data are quite limited to support this use, but studies are underway). In 2010, the North American Menopause Society issued an updated position statement on management of osteoporosis in postmenopausal women. The statement included the recommendation, routine use of biochemical markers of bone turnover in clinical practice is not generally recommended. (17) In 2011, the International Osteoporosis Foundation (IOF) and the International Federation of Clinical Chemistry and Laboratory Medicine (IFCC) published a position statement by a joint IOF IFCC Bone Marker Standards Working Group. (18) The aim of the group was to evaluate evidence on using bone turnover markers for fracture risk assessment and monitoring of treatment. The group s overall conclusion was, In summary, the available studies relating bone turnover marker changes to fracture risk reduction with osteoporosis treatments are promising. Further studies are needed that take care of sample handling, ensure that bone turnover markers are measured in all available patients, and use the appropriate statistical methods, including an assessment of whether the final bone turnover marker level is a guide to fracture risk. In 2011, the Joint Official Positions Development Conference of the International Society for Clinical Densitometry and the IOF on the FRAX fracture risk prediction algorithms published the following statement (19): Evidence that bone turnover markers predict fracture risk independent of BMD is inconclusive. Therefore, bone turnover markers are not included as risk factors in FRAX. U.S. Preventive Service Task Force Recommendations The U.S. Preventive Services Task Force 2011 recommendations on osteoporosis screening address DXA testing but do not mention bone turnover markers. (20) Summary The literature suggests that bone turnover marker levels may be independently associated with osteoporosis and fracture risk in some groups, but there is insufficient evidence reporting an association for any specific marker. Questions remain about whether bone turnover markers are sufficiently sensitive to reliably determine individual treatment responses. In addition, there is insufficient evidence from controlled studies that bone turnover marker measurement improves adherence to treatment, impacts management decisions, and/or improves health outcomes such as reducing fracture rates. Thus, the use of bone turnover markers for the diagnosis and management of osteoporosis is considered not medically necessary. There is insufficient evidence that measurement of bone turnover markers improves patient management or health outcomes in patients with conditions associated with high bone turnover

Page: 9 of 12 including Paget disease, primary hyperparathyroidism, and renal osteodystrophy. Thus, bone turnover marker testing for these other conditions is considered investigational. Medicare National Coverage On November 25, 2002, the Centers for Medicare and Medicaid Services (CMS) issued a National Coverage Determination (NCD) on collagen cross-links. (21) The CMS NCD identifies a set of clinical conditions for which collagen cross-links would be considered eligible for coverage. The CMS NCD is limited to urine-based collagen cross-link tests and does not address serum-based collagen cross-link tests. The Federal Register (22) notes that Medicare carriers have discretion to make their own determinations on the medical necessity of serum-based collagen cross-link tests for assessing or monitoring bone loss therapy. The Federal Register also notes that the FDA approved the serumbased collagen cross-link tests under 510(k) review, as substantially equivalent to the urine-based collagen cross-link test. It should be noted that the serum-based collagen cross-link tests are more commonly performed than urine collagen cross-link tests. The Medicare NCD analysis focused on the technical feasibility of collagen cross-links and anticipated outcomes. The discussion above focused on the impact on health outcomes as documented in controlled studies. References 1. Tamaki J, Iki M, Kadowaki E, et al. Biochemical markers for bone turnover predict risk of vertebral fractures in postmenopausal women over 10 years: the Japanese Population-based Osteoporosis (JPOS) Cohort Study. Osteoporos Int. Mar 2013; 24(3):887-897. PMID 22885773 2. Bauer DC, Garnero P, Harrison SL, et al. Biochemical markers of bone turnover, hip bone loss and fracture in older men: the MrOS Study. J Bone Mineral Res. 2009; 24(12):2032-2038. PMID 3. Johansson H, Oden A, Kanis JA, et al. A meta-analysis of reference markers of bone turnover for prediction of fracture. Calcif Tissue Int. May 2014; 94(5):560-567. PMID 24590144 4. Biver E, Chopin F, Coiffier G et al. Bone turnover markers for osteoporotic status assessment? A systematic review of their diagnosis value at baseline in osteoporosis. Joint Bone Spine 2012; 79(1):20-25. PMID 21724445 5. Bauer DC, Garnero P, Hochberg MC, et al. Pretreatment levels of bone turnover and the antifracture efficacy of alendronate: the Fracture Intervention trial. J Bone Miner Res. 2006; 21(2):292-299. PMID 6. Abe Y, Ishikawa H, Fukao A. Higher efficacy of urinary bone resorption marker measurements in assessing response to treatment for osteoporosis in postmenopausal women. Tohoku J Exp Med. 2008; 214(1):51-59. PMID

Page: 10 of 12 7. Shiraki M, Itabashi A. Short-term menatetrenone therapy increases gamma-carboxylation of osteocalcin with a moderate increase of bone turnover in postmenopausal osteoporosis: a randomized prospective study. J Bone Miner Metab. 2009; 27(3):333-340. PMID 8. Funck-Brentano T, Biver E, Chopin F, et al. Clinical utility of serum bone turnover markers in postmenopausal osteoporosis therapy monitoring: a systematic review. Semin Arthritis Rheum. Oct 2011; 41(2):157-169. PMID 21507464 9. Baxter I, Rogers A, Eastell R, et al. Evaluation of urinary N-telopeptide of type I collagen measurements in the management of osteoporosis in clinical practice. Osteoporos Int. Mar 2013; 24(3):941-947. PMID 22872068 10. National Osteoporosis Foundation. 2014 Clinician's guide to prevention and treatment of osteoporosis. http://nof.org/files/nof/public/content/file/2610/upload/895.pdf. Accessed July 2014. 11. Burch J, Rice S, Yang H, et al. Systematic review of the use of bone turnover markers for monitoring the response to osteoporosis treatment: the secondary prevention of fractures, and primary prevention of fractures in high-risk groups. Health Technol Assess. Feb 2014; 18(11):1-180. PMID 24534414 12. Roux C, Giraudeau B, Rouanet S, et al. Monitoring of bone turnover markers does not improve persistence with ibandronate treatment. Joint Bone Spine 2011; 79(4):389-392. PMID 13. Rianon N, Alex G, Callender G, et al. Preoperative serum osteocalcin may predict postoperative elevated parathyroid hormone in patients with primary hyperparathyroidism. World J Surg. Jun 2012; 36(6):1320-1326. PMID 22278606 14. Woitge HW, Oberwittler H, Heichel S, et al. Short- and long-term effects of ibandronate treatment on bone turnover in Paget disease of bone. Clin Chem. May 2000; 46(5):684-690. PMID 10794751 15. Reid IR, Davidson JS, Wattie D, et al. Comparative responses of bone turnover markers to bisphosphonate therapy in Paget's disease of bone. Bone. Jul 2004; 35(1):224-230. PMID 15207761 16. Alvarez L, Guanabens N, Peris P, et al. Usefulness of biochemical markers of bone turnover in assessing response to the treatment of Paget's disease. Bone. Nov 2001;29(5):447-452. PMID 11704497 17. North American Menopause Society. Management of osteoporosis in postmenopausal women: 2010 position statement of the North American Menopause Society. 18. Vasikaran S, Cooper C, Eastell R, et al. International Osteoporosis Foundation and International Federation of Clinical Chemistry and Laboratory Medicine Position on bone marker standards in osteoporosis. Clin Chem Lab Med. 2011; 49(8):1271-1274. PMID 19. McCloskey EV, Vasikaran S, Cooper C. Official Positions for FRAX(R) clinical regarding biochemical markers from Joint Official Positions Development Conference of the International Society for Clinical Densitometry and International Osteoporosis Foundation on FRAX(R). J Clin Densitom. Jul-Sep 2011; 14(3):220-222. PMID 21810528

Page: 11 of 12 20. U.S. Preventive Services Task Force (USPSTF). Screening for Osteoporosis. 2011; http://www.uspreventiveservicestaskforce.org/uspstf10/osteoporosis/osteosum.htm. Accessed September 2014. 21. CMS National Coverage Determinations. 190.19 Collagen Crosslinks, Any Method. Available online at: http://www.cms.hhs.gov/coveragegeninfo/downloads/manual200907.pdf. Last accessed September 2014. 22. Federal Register, November 23, 2001. Rules and Regulations. 66(No. 226). PMID Policy History Date Action Reason September 2012 New Policy December 2013 Update Policy Policy updated with literature review, Policy statement added as investigational: Measurement of bone turnover markers in the management of conditions with high rates of bone turnover, including but not limited to Paget s disease, primary hyperparathyroidism and renal osteodystrophy. References added, others renumbered or removed. Title change to Bone Turnover Markers for the Diagnosis and Management of Osteoporosis and Diseases Associated with High Bone Turnover December 2014 Update Policy Policy updated with literature review. No change to policy statements. References 3, 11 and 20 added. Keywords Bone Turnover Markers Collagen Cross links, Osteoporosis Osteoporosis, Bone Turnover Markers Pyridinoline Deosypyridinoline Pyrilinks Pyrilinks-D N-telopeptide NTx Osteomark C-telopeptide CTx Cross Laps Telopeptide Popeptide

Page: 12 of 12 This policy was approved by the FEP Pharmacy and Medical Policy Committee on December 5, 2014 and is effective January 15, 2015. Signature on File Deborah M. Smith, MD, MPH