1、Designation: F3037 15Standard Guide forClinical Trial Design for Hip Replacement Systems (HRSs)1This standard is issued under the fixed designation F3037; the number immediately following the designation indicates the year oforiginal adoption or, in the case of revision, the year of last revision. A
2、 number in parentheses indicates the year of last reapproval. Asuperscript epsilon () indicates an editorial change since the last revision or reapproval.1. Scope1.1 This guide is intended as a resource for individuals andorganizations involved in designing clinical trials of hipreplacement systems
3、(HRSs) including metal/polymer, metal/metal, metal/composite, metal/ceramic/polymer, metal/polymer/metal, and ceramic/ceramic bearing surfaces; semi-constrained and constrained designs; and cemented, nonporousuncemented, and porous-coated uncemented fixation.1.2 In this guide, methods to measure the
4、 efficacy,effectiveness, and safety of HRS devices through standardizingoutcomes measures are provided for designing, reviewing, andaccepting human clinical trial protocols.1.3 This guide is intended to provide consistency in studydesign, review, regulatory approval, and coverage approval forhip rep
5、lacement systems to the health care market.1.4 For the purpose of this guide, an HRS is any device thatis intended to replace the hip joint, in part or in total, as atreatment for joint disease, trauma, or dysfunction, wherelong-term functional restoration and pain relief without majoradverse events
6、 are the desired outcomes.1.5 This standard does not purport to address all of thesafety concerns, if any, associated with its use. It is theresponsibility of the user of this standard to establish appro-priate safety and health practices and determine the applica-bility of regulatory limitations pr
7、ior to use.2. Referenced Documents2.1 ASTM Standards:2F561 Practice for Retrieval and Analysis of MedicalDevices, and Associated Tissues and FluidsF2809 Terminology Relating to Medical and Surgical Mate-rials and DevicesF2978 Guide to Optimize Scan Sequences for Clinical Di-agnostic Evaluation of Me
8、tal-on-Metal Hip ArthroplastyDevices using Magnetic Resonance ImagingF2979 Guide for Characterization of Wear from the Articu-lating Surfaces in Retrieved Metal-on-Metal and otherHard-on-Hard Hip Prostheses2.2 ISO Standards3ISO 12891-1 Retrieval and analysis of surgical implants Part1: Retrieval and
9、 handlingISO 12891-2 Retrieval and analysis of surgical implants Part 2: Analysis of retrieved surgical implantsISO 14155 Clinical investigation of medical devices forhuman subjects Good clinical practiceISO 14971 Medical devices Application of risk manage-ment to medical devices3. Terminology3.1 Un
10、less provided in 3.2.1 3.2.5, definitions shall be inconformance with Terminology F2809.3.2 Definitions:3.2.1 coverage, ninsurance decision to reimburse for adevice and/or procedure.3.2.2 effectiveness, nextent to which medical interven-tions achieve health improvements in real practice settings.3.2
11、.3 effcacy, nextent to which medical interventionsachieve health improvements under ideal circumstances.3.2.4 level of evidencestrength of clinical evidence forevidence-based medicine (1)4.3.2.5 safetythe condition of being protected from orunlikely to cause risk or injury. See Appendix X1 for atabu
12、lated list of adverse events reported for hip replacementsystems (2).3.3 Acronyms:AJRRAmerican Joint Replacement RegistryASAAmerican Society of AnesthesiologistsDVTDeep Vein ThrombosisEQ-5DEuropean Quality of Life 5 DomainsFDAFood and Drug AdministrationHHSHarris Hip Score1This test method is under
13、the jurisdiction of ASTM Committee F04 on Medicaland Surgical Materials and Devices and is the direct responsibility of SubcommitteeF04.39 on Human Clinical Trials.Current edition approved June 1, 2015. Published August 2015. DOI: 10.1520/F3037-15.2For referenced ASTM standards, visit the ASTM websi
14、te, www.astm.org, orcontact ASTM Customer Service at serviceastm.org. For Annual Book of ASTMStandards volume information, refer to the standards Document Summary page onthe ASTM website.3Available from American National Standards Institute (ANSI), 25 W. 43rd St.,4th Floor, New York, NY 10036, http:
15、/www.ansi.org.4The boldface numbers in parentheses refer to a list of references at the end ofthis standard.Copyright ASTM International, 100 Barr Harbor Drive, PO Box C700, West Conshohocken, PA 19428-2959. United States1HOOSHip dysfunction and Osteoarthritis OutcomeScoreHRQLHealth-related quality
16、of lifeHRSHip Replacement SystemICDInternational Classification of DiseasesLEASLower Extremity Activity ScaleMCIDMinimal clinically important differenceMRIMagnetic Resonance ImagingOHSOxford Hip ScorePROPatient-reported outcomePROMISPatient-Reported Outcomes MeasurementInformation SystemQALYQuality
17、adjusted life yearRSARadiostereometric analysisSAESerious adverse eventSF-36Short Form (36 questions)SF-12Short Form (12 questions)SF-6DShort Form (6 dimensions)THATotal hip arthroplastyTUGTimed up and goUCLAUniversity of California at Los AngelesUTIUrinary tract infectionWOMACWestern Ontario McMast
18、er OsteoarthritisIndex4. Summary of Guide4.1 It is the intent of this guide to provide an overview ofappropriate outcomes that are to be addressed in human clinicaltrials of hip replacement systems (HRSs). Depending on therequirements of the clinical trial, the outcomes to be addressedinclude hip-sp
19、ecific patient-reported outcomes, health-relatedquality-of-life patient-reported outcomes, activity level scales,gait speed, symptom relief (pain visual analog scales), andfrequency of adverse events.4.2 In general and in accordance with evidence-based medi-cine principles, patient-reported outcomes
20、 should be givenpreference over mixed outcome measures (surgeon and patientcompletion), intermediate outcomes (physical examinationfindings), or radiographic outcomes. However, the U.S. De-partment of Health and Human Services and/or local require-ments may require mixed outcomes measures.4.3 Becaus
21、e of the broad range of indications for HRSs,patient comorbidities, and functional/activity levels, it is im-possible to identify or specify a single instrument score thatmeasures the “success” of HRSs. Instead, a clinically signifi-cant improvement (minimum clinically important differenceMCID) in a
22、 joint-specific, disease-specific, or quality-of-lifeinstrument should be used as a measure of clinical “success”(30). A practical guide for determining MCIDs is that theMCID equals one half of the standard deviation of the changein the instrument score, MCID = /2 (3). This distributionmethod of det
23、ermining MCID for a validated PRO instrumentallows the calculation of the MCID for specific patient sub-groups and/or interventions/treatments because the MCID mayvary by patient subgroup and/or intervention/treatment.4.4 The application of this guide does not guarantee clinicalsuccess of a finished
24、 product but will help to ensure consis-tency and adequacy in the clinical data of the clinical trialprotocol.4.5 The coverage criteria for medical treatments include: (1)that a net health outcome is achieved, (2) the clinical trialresults are applicable (generalizable) to the patient population,and
25、 (3) the clinical trial results are applicable (generalizable) tomedical providers (effectiveness versus efficacy). Therefore,clinical trials should be able to perform subgroup analysesbased on patient characteristics (age, sex) and provider char-acteristics (community providers).4.6 This guide does
26、 not suggest that all outcome instrumentsbe used for each HRS. However, inclusion of an outcomemeasure from each section will provide a thorough descriptionof the benefits of an HRS, including hip function/pain relief,health-related quality of life including a health utility measurewith the ability
27、to calculate Quality Adjusted Life Years(QALYs) (4), activity level, and mobility.5. Significance and Use5.1 Approximately 300,000 primary total hip arthroplasties(THAs) and 50,000 revision THAs are performed in the UnitedStates annually (5, 6). In addition, approximately 50 % of the300,000 hip frac
28、tures in the United States annually are femoralneck fractures. The majority of femoral neck fractures aretreated with hip hemiarthroplasties (femoral head replacementonly).6. Use (Outcome Measures)6.1 Patient-Reported Outcomes (PROs):6.1.1 Patient-reported outcomes (PROs) are vital to under-standing
29、 the value patients receive from health care. Value canbe defined as the change in quality of life and function dividedby the total cost of care. Improvement in quality of life is mostcommonly measured by Quality Adjusted Life Years (QALYs)(4). QALYs are required for cost-effectiveness analyses andc
30、omparative effectiveness analyses used in coverage decisions.Standardization of PRO measures is necessary to compareoutcomes of procedures (7). Standardizing PRO measures forimplant and outcome registries will make comparative effec-tiveness data available to the clinical and regulatory commu-nities
31、.6.1.2 PRO Measure SelectionPRO measure selectionshall be pragmatic. High-respondent burden (too many ques-tions) will result in poor rates of patient completion. Highlicensing fees make it difficult for not-for-profit registries tolicense the measure.6.1.3 Hip-Specific or Disease-Specific OutcomeIn
32、strumentsThe hip-specific PRO measures most frequentlyused are the Oxford Hip Score (OHS) (8) and Hip dysfunctionand Osteoarthritis Outcome Score (HOOS) (9). The OHS isused in the New Zealand Joint Registry (10) and the NationalJoint Registry of England, Wales, and Northern Ireland. TheOxford Hip Sc
33、ore and Oxford Knee Score have also beenadopted for use in the United States. The American JointReplacement Registry (AJRR) accepts the Oxford Hip Scoreand Oxford Knee Score as Level 3 data on patient-reportedF3037 152outcomes. The Oxford Knee Score is the PRO knee instrumentmandated by the Minnesot
34、a Department of Health for all kneearthroplasty procedures in the State of Minnesota effectiveJanuary 1, 2012 (11, 12). The Western Ontario McMasterOsteoarthritis Index (WOMAC) (13) is a lower extremityosteoarthritis disease-specific outcome instrument used forlower extremity osteoarthritis. The Har
35、ris Hip Score (HHS) isa surgeon-reported outcome completed with patient input andsubject to surgeon bias. However, because of the clinical andregulatory experience with the HHS, the HHS may be used asan outcome measure, but is not a preferred outcome measuredue to potential surgeon bias. Pynsent et
36、al (14) reviewedvalidated hip PRO instruments. A more recent validated PROfor osteoarthritis is the Patient-Reported Outcomes Measure-ment Information System (PROMIS) Physical Function instru-ment (Broderick JE, 2013).6.1.4 General Health-Related Quality of Life (HRQL) Out-come InstrumentsThe Europe
37、an Quality of Life (EQ-5D) isused by the British National Health Service and National JointRegistry of England, Wales, and Northern Ireland to assess theHRQL change after THA (15). EQ-5D is used by the SwedishHip Registry (16, 17), the Norwegian Arthroplasty Register(18), and the Norwegian Hip Fract
38、ure Register (19). TheEQ-5D has become the standard outcome instrument for hipfracture outcome studies looking at femoral neck fractures(20), intertrochanteric fractures (21), and subtrochanteric frac-tures (22). EQ-5D is used for musculoskeletal disease researchin Japan (23), Denmark (24), the Neth
39、erlands (25), and Finland(26). SF-36 and SF-12 are frequently used as HRQL outcomesinstruments. However, the quality-of-life summary measure(SF-6D) is a calculated summary score and does not allowpatient preference weighting for calculation of change inHRQL Both the EQ-5D and SF-6D can be used to ca
40、lculateQALYs for cost-effectiveness or comparative-effectivenessanalyses. The PROMIS Global Health instrument may be usedto assess health-related quality of life (Amtmann D, 2011).6.1.5 Activity Level ScalesThe University of California atLos Angeles (UCLA) Activity Scale and Lower ExtremityActivity
41、Scale (LEAS) (27) were found to be the most validactivity scales for hip osteoarthritis (28).6.1.6 Gait Speed/Mobility MeasuresA significant percent-age of patients with hip fractures have cognitive impairmentand are unable to complete PRO instruments. However, gaitspeed can be measured independentl
42、y of cognitive function ifthe subject is ambulatory. Also, gait speed (29) and mobilitydisability (30) are strong predictors of overall mortality.Therefore, measurement of gait speed change is a functionaloutcome measure for HRS. A standardized test to measure gaitspeed is the Timed Up and Go (TUG)
43、test (14).6.2 Safety:6.2.1 Adverse event rates are a measure of safety and shouldbe defined by the study protocol. All adverse events shall berecorded. Adverse events directly related to the HRS orotherwise required by regulatory guidance shall be reported.The following types of adverse events have
44、been reported forHRS (2) and an example of how to report the data is includedin Table X1.1. Additional adverse events that should bereported are: pseudotumor, adverse local tissue reaction, noise(grinding, clicking, popping, squeaking), taper wear, andincrease in metal ion/corrosion products. Time w
45、indows foradverse event reporting should be based on regulatory guid-ance (Clinical Data Presentation for Orthopedic DeviceApplications, Food and Drug Administration, December 2,2004). Adverse event reporting may be reported and analyzedaccording to both: (1) regulatory requirements and (2) timewind
46、ows included in this guide in order to capture all adverseevents and determine if different time windows affect adverseevent rates.6.2.2 Adverse event collection, analyses, and reportingprotocols for a priori grading of adverse event severity andrelatedness shall be established. An independent Data
47、SafetyReview Board should be considered when appropriate. AClinical Events Committee should be considered when appro-priate.6.2.3 The following clinically expected events should bereported separately as hospital and/or surgeon quality mea-sures:6.2.3.1 Hip joint dislocation any time postoperatively;
48、6.2.3.2 Deep infection requiring re-operation within oneyear of surgery;6.2.3.3 Deep vein thrombosis or pulmonary emboli or bothwithin 90 days of surgery;6.2.3.4 All-cause non-elective 30-day hospital readmission;6.2.3.5 Intraoperative or postoperative femoral or acetabularfracture occurring within
49、one year of surgery;6.2.3.6 Hip Reoperation/Revision Surgery (No TiimeLimit)A revision surgery is defined as a procedure that isperformed on the replaced hip to remove and/or replace anyfemoral, acetabular, or both component(s) implanted at theindex operation; or reduction of a dislocated hip replacement;6.2.3.7 All Serious Adverse Events (SAEs), and6.2.3.8 Death within 30 days of surgery.6.2.3.9 For rare severe adverse events, consider increasingthe level of significance ().6.3 Radiographic Outcome:6.3.1 Radiographic analysis sho
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