Criteria for the appropriate treatment of osteoporotic vertebral compression fractures : RAND Study
 
 
 
 
 Pr Charles Court, Dr Hendrik Fransen§ Service chirurgie orthopédique Bicêtre §Departement of radiology, UZ Antwerp Belgium

Disclosure ! This study was supported by an unrestricted educational grant from Medtronic Spinal & Biologics Europe ! The sponsor was not involved in the panel process and preparation of the slides ! Panel members received honoraria from the sponsor for the rating activities and their participation in the panel meetings ! The study was coordinated by Ismar Healthcare, Lier, Belgium

Background ● Vertebral compression fractures (VCFs) are frequently seen complications of osteoporosis and can have a high impact on QoL1 ● Common treatment options are non-surgical management (NSM), vertebroplasty (VP) and balloon kyphoplasty (BKP) ● High-quality evidence to support treatment choice is limited and inconclusive ● As a consequence, most guidelines are non very specific 1 J Bone Miner Res 2002;17:716-24.

● To establish / refine criteria for the appropriate treatment of VCF at the patient-specific level, by combining evidence from clinical studies and expert opinion ! ● To translate these criteria into clinically useful recommendations Aims

● Modified Delphi method that combines: ̶ Best available scientific evidence ̶ Collective judgement of experts to assess the appropriateness of performing a procedure at the level of a patient’s symptoms, medical history and test results ● A procedure is considered appropriate if its expected benefits outweigh its potential negative consequences by a sufficient margin that the procedure is worth doing RAND/UCLA Appropriateness Method1 1 Brook RH et al Int J Technol Assess Health Care 1986;2:53-63.

European multidisciplinary panel ● Giovanni Anselmetti Radiologist Turin (IT) ● Jason Bernard Orthopaedic surgeon London (UK) ● Thomas Blattert Orthop./trauma surgeon Schwarzach (DE) ● Charles Court Orthopaedic surgeon Paris (FR) ● Daniel Fagan Spinal surgeon Middlesbrough (UK) ● Hendrik Fransen Radiologist Ghent (BE) ● Patrick Fransen Neurosurgeon Brussels (BE) ● Christian Kasperk Internist Heidelberg (DE) ● Tarun Sabharwal Radiologist London (UK) ● Frédéric Schils Neurosurgeon Liège (BE) ● Rupert Schupfner Trauma surgeon Bayreuth (DE) ● Mashood Ali Siddiqi Bone metabolic specialist Liverpool (UK)

First rating round Feedback and panel discussion Second rating round Panel criteria: ! ▪ Appropriate ▪ Uncertain ▪ Inappropriate First panel meeting ! ● Patient population ● Treatments included ● Clinical variables Decision model & rating program Research steps Recommendations

Patient population : VCF due to osteoporosis Patients had to fulfil the following criteria: ● At least moderate symptoms (VAS ≥ 5) correlating with the fracture ● Absence of neurological symptoms ● Age ≥ 18 years ● Absence of absolute contra-indications for active treatment: ̶ Not fit enough to undergo surgery ̶ Pregnancy ̶ Spine infection ̶ Coagulation disorder 1. Magerl F et al. Eur Spine J 1994;3:184-201

Clinical variables relevant to treatment choice Variables Categories Time since fracture < 6 wk 6 wk-3 mo > 3 mo MRI findings Negative Positive Impact of VCF on daily functioning Moderate Severe Evolution of symptoms Stable Has worsened Spinal deformity No Yes Proof of ongoing fracture process No Yes Pulmonary dysfunction No Yes

1 2 3 4 5 6 7 8 9 1 Extremely inappropriate 5 Uncertain / equivocal 9 Extremely appropriate Rating the appropriateness Disregarding financial costs, waiting lists, and other potential constraints...

Agreement 1 2 3 4 5 6 7 8 9 ! ! ! Disagreement 1 2 3 4 5 6 7 8 9 ! ! ! ! ! ! ! ! ! ! ! ! Indeterminate 1 2 3 4 5 6 7 8 9 ! ! ! ! ! ! ! ! ! ! ! ! !! ! ! !! ! ! ! Agreement = Accord/Désacord ≥ 9/12 ≥ 4/12 ≥ 4/12 (All other situations)

RELEVANT PATIENT CHARACTERISTICS Patient profiles: mutually exclusive combinations of relevant patient characteristics were rated using an electronic program

First rating round ● 192 cases (patient profiles) for which 3 treatment options (NSM, VP, BKP) had to be rated ● Substantial disagreement : 31% due to: ― Different ways of rating ― Different interpretation of definitions and cases ― Variations in opinion between specialties à Adaptation of rating structure, exclusion of unrealistic cases, refinement of instructions à Second rating round: 128 cases

Changes between 1st and 2nd rating round 0 18 35 53 70 Disagreement Uncertain outcomes Round 1 Round 2 % of ratings

Appropriate treatments % None 4 NSM only VP only BKP only 25 5 45 NSM and VP NSM and BKP VP and BKP - - 21 NSM and VP and BKP - Total 100 Is there an appropriate treatment for each case? 75%

Appropriate treatments (%) by clinical variables Variable Value NSM VP BKP Time since fracture < 6 weeks 6 weeks – 3 months > 3 months 3 31 33 9 31 31 94 60 54 MRI findings Negative Positive 94 2 - 34 - 89 Impact on daily functioning Moderate Severe 28 22 19 33 64 69 Evolution of symptoms Stable Has worsened 28 22 23 28 63 70 Spinal deformity No Yes 28 22 44 8 58 75 Ongoing fracture process No Yes 40 0 16 42 46 100 Pulmonary dysfunction No Yes 27 23 23 28 64 69 Total 25 26 66

Appropriate treatments (%) by clinical variables Variable Value NSM VP BKP Time since fracture < 6 weeks 6 weeks – 3 months > 3 months 3 31 33 9 31 31 94 60 54 MRI findings Negative Positive 94 2 - 34 - 89 Impact on daily functioning Moderate Severe 28 22 19 33 64 69 Evolution of symptoms Stable Has worsened 28 22 23 28 63 70 Spinal deformity No Yes 28 22 44 8 58 75 Ongoing fracture process No Yes 40 0 16 42 46 100 Pulmonary dysfunction No Yes 27 23 23 28 64 69 Total 25 26 66

Appropriate treatments (%) by clinical variables Variable Value NSM VP BKP Time since fracture < 6 weeks 6 weeks – 3 months > 3 months 3 31 33 9 31 31 94 60 54 MRI findings Negative Positive 94 2 - 34 - 89 Impact on daily functioning Moderate Severe 28 22 19 33 64 69 Evolution of symptoms Stable Has worsened 28 22 23 28 63 70 Spinal deformity No Yes 28 22 44 8 58 75 Ongoing fracture process No Yes 40 0 16 42 46 100 Pulmonary dysfunction No Yes 27 23 23 28 64 69 Total 25 26 66

Variable Value VP BKP Time since fracture < 6 weeks 6 weeks – 3 months > 3 months 9 31 31 94 60 54 MRI findings Negative Positive - 34 - 89 Impact on daily functioning Moderate Severe 19 33 64 69 Evolution of symptoms Stable Has worsened 23 28 63 70 Spinal deformity No Yes 44 8 58 75 Ongoing fracture process No Yes 16 42 46 100 Pulmonary dysfunction No Yes 23 28 64 69 Total 26 66 Appropriateness of VP and BKP (4)

Guideline recommendations ● NICE 1,2 : VP and BKP limited to patients refractory to conservative treatment ● CIRSE3 : VP indicated in painful osteoporotic VCFs refractory to conservative treatment (insufficient response and/or intolerance following 3 weeks of medical treatment) ● AAOS4 : against VP in osteoporotic VCFs (strong recommendation), BKP is an option (weak recommendation) ● Position paper5 : VP and BKP for painful VCFs refractory to (optimised) medical therapy ● ACR6 : 1st line: conservative management; 2nd line: VP/BKP (BKP similar to VP, better angular and fracture correction) ● DVO7 : VP and BKP indicated in osteoporotic VCFs after ● 3 week conservative treatment attempt ● Exclusion of degenerative changes of the spine ● Interdisciplinary discussion of the case 1. NICE http://guidance.nice.org.uk/IPG166/ 2. NICE http://guidance.nice.org.uk/IPG12/ 3. CIRSE http://www.cirse.org/files/File/07_qig.pdf 4. AAOS http://www.aaos.org/research/guidelines/ ! 5. Jensen ME et al. J Neurointerv Surg 2009;1:181-5. 6. ACR http://www.acr.org/AC. 7. DVO_http://www.schattauer.de/fileadmin/assets/ zeitschriften/osteologie/0111_OST_01_2011.pdf

Conclusions ● This multidisciplinary RAND study provides recommendations for treatment of osteoporotic VCFs at the patient-specific level ! ! ! ! ! ● In 75% of the profiles 1 treatment option was preferred, in only 4% of the profiles no specific recommendation could be given ● Validity and feasibility in daily clinical practice should be determined in prospective studies ̶ Not in contradiction with current guidelines and RCT outcomes (except for VP in AAOS guideline) ̶ More tailored than current guidelines

Further study : 80% population covered by 20% profiles (128), inclusion : 25x20= 500 pat