Clinical UM Guideline

 

Subject: Vertical Expandable Prosthetic Titanium Rib
Guideline #:  CG-SURG-07 Publish Date:    08/29/2018
Status: Reviewed Last Review Date:    07/26/2018

Description

This document addresses the use of a vertical expandable prosthetic titanium rib (VEPTR®) device as a treatment of severe progressive spinal deformities associated with thoracic insufficiency syndrome (TIS), which includes severe forms of scoliosis. TIS is defined as the inability of the thorax to support normal respiration or lung growth. The VEPTR device consists of a curved rod that conforms to the shape of the thoracic cage. This device, with clearance from the U.S. Food and Drug Administration (FDA), is surgically implanted and vertically affixed to two ribs above and below expansion thoracostomies, which are placed at the site of the vertebral deformity.

Clinical Indications

Medically Necessary:

The implantation, expansion (lengthening), exchange (replacement), or conversion (rib to spine) of an FDA cleared vertical expandable prosthetic titanium rib device is considered medically necessary in skeletally immature individuals for the treatment of severe, progressive spinal deformities and three-dimensional deformity of the thorax associated with, or at risk for, thoracic insufficiency syndrome including severe progressive scoliosis for ANY of the following conditions (A through D):

  1. Congenital scoliosis; or
  2. Neuromuscular scoliosis; or
  3. Infantile and juvenile idiopathic scoliosis; or
  4. Syndromic scoliosis.

Not Medically Necessary:

Use of the vertical expandable prosthetic titanium rib device is considered not medically necessary when the above criteria are not met and for all other indications.

Coding

The following codes for treatments and procedures applicable to this document are included below for informational purposes. Inclusion or exclusion of a procedure, diagnosis or device code(s) does not constitute or imply member coverage or provider reimbursement policy. Please refer to the member's contract benefits in effect at the time of service to determine coverage or non-coverage of these services as it applies to an individual member.

CPT

 

21899

Unlisted procedure, neck or thorax [when specified as implantation of vertical expandable prosthetic titanium rib]

 

 

ICD-10 Diagnosis

 

 

All diagnoses

   
Discussion/General Information

TIS is a rare, life threatening condition that involves the inability of the thorax to support normal respiration and lung growth (Campbell, 2003). The condition results from serious congenital defects that affect the ribs or chest wall including, but not limited to, flail chest syndrome, severe scoliosis, rib fusion (which may accompany scoliosis), and various hypoplastic thorax syndromes, which may include Jeune’s syndrome, achondroplasia, Jarcho-Levin syndrome, and Ellis-van Creveld syndrome. Children with TIS show visible signs of the disease in a variety of ways. The syndrome is frequently terminal without surgical treatment, and each afflicted child has the potential to develop pulmonary hypertension, respiratory failure, right ventricular failure, cor pulmonale, and death.

The VEPTR device (DePuy Synthes® Spine, Raynham, MA) obtained U.S. Food and Drug Administration (FDA) clearance under a Humanitarian Device Exemption (HDE) in 2004. The data submitted to the FDA as part of the approval process consisted of a single-site feasibility study of 33 subjects and a prospective, multi-center, single treatment arm trial of 247 children, ranging from ages 6 months to 15 years. The children had various serious defects affecting their ribs or chest wall and their ability to breathe, including progressive scoliosis, flail chest syndrome, rib fusion and various hypoplastic thorax syndromes. The investigators reported study results for both trials that demonstrated probable benefit by enabling some of the children to breathe unassisted, or be less dependent on ventilators. Treatment-emergent events were reported for the entire length of follow-up for both trials, (with a mean follow-up for the feasibility study of 84.8 months and 22.6 months for the multi-center trial). A total of 29 (88%) of 33 subjects in the feasibility study reported a total of 182 adverse effects, while a total of 68 adverse effects were considered to be related to the device in 21 subjects (64%). A total of 11 adverse events that were considered potentially life-threatening were reported for 2 subjects, and 13 adverse effects of fatal intensity were reported for 4 trial participants, none of which were considered to be device-related.

In the multi-center trial, a total of 119 (56%) of 214 subjects reported a total of 356 adverse effects, while a total of 141 adverse effects, considered to be related to the device, were reported in 71 subjects (33%). The investigators reported that the majority of adverse events were related to device migrations which occurred after the subjects had been implanted with the VEPTR for 2 or more years. A total of 12 deaths were reported, none of which were related to a problem with the device.

According to the original FDA approved product label, the VEPTR is indicated for:

The treatment of thoracic insufficiency syndrome in skeletally immature patients and it should not be used under the following conditions:

According to the FDA summary of safety and probable benefit data, during the course of these 14 years of study, as the children experienced normal growth or as the spine and thorax required further correction, the device would require expansions or replacement of the components to increase the overall size of the device. As a guideline, children with scoliosis or flail chest syndrome were scheduled for expansion of the device when the Cobb angle increased by 5 degrees or greater. Studies have typically involved expansion (lengthening) of the device at 4 to 6 month intervals depending on growth rates. In some cases, a device exchange was needed for growth, and device conversion from rib-rib to rib-spine was also reported. In one series, the mean number of lengthenings performed per individual was 3.5 ± 2.6 (Emans, 2005). The FDA concluded from the submitted trial results that the probable benefits associated with VEPTR implantation outweighed the risks for the high-risk population of skeletally immature individuals with TIS.

In 2014, the FDA Center for Devices and Radiological Health (CDRH) cleared the VEPTR®-VEPTR II device which was reclassified as a Class II device that is considered substantially equivalent to other similar predicate devices through the 510(k) approval process for the following indications:

For skeletally immature patients with severe, progressive spinal deformities and/or three dimensional deformity of the thorax associated with, or at risk of, Thoracic Insufficiency Syndrome (TIS). TIS is defined as the inability of the thorax to support normal respiration or lung growth. This would include patients with progressive congenital, neuromuscular, idiopathic, or syndromic scoliosis (FDA, 2014).

Due to the rarity of TIS, there have been few published clinical trials of VEPTR device implantation with outcomes data. In 2007, Waldhausen and colleagues performed a retrospective chart review to analyze the cases of 22 children who received 36 VEPTR devices between October 2001 and December 2005; 2 of the implanted trial subjects had Jeune syndrome, 19 had scoliosis, and 1 had a chest wall resection for a tumor. The majority of trial participants had pulmonary restrictive disease, carbon dioxide retention or respiratory failure. A total of 11 had multiple fused ribs requiring an opening wedge thoracostomy. All but the most recently treated cases had undergone sequential VEPTR expansion; seven cases required revision for erosion through the bone or device dislodgment with three devices requiring removal. A total of five devices were outgrown and removed or replaced, and one eroded the soft tissue causing superficial infections that resolved with operative revision. The postoperative ventilation/perfusion scans showed the most improvement in the younger treated children with 2 of the 3 children with carbon dioxide retention pre-VEPTR showed reductions in carbon dioxide levels post-VEPTR. The authors concluded that the VEPTR may decrease carbon dioxide retention in some cases and may be most beneficial in younger children.

The 2014 expansion of the FDA labeling for VEPTR was based on results of post marketing follow-up data from the original multi-center trial previously described. Additional trial results have consisted of small case series with outcomes data for 2 to 3 years that demonstrated some improvement in pulmonary function, thoracic height and the degree of Cobb angle deformity associated with severe scoliosis. Multiple additional expansion surgical procedures were necessary in the majority of cases to accommodate normal growth patterns, and high incidences of adverse events were seen in the reported outcomes. The investigators of these small studies concluded that the benefits outweighed the risks for the high-risk population with severe progressive scoliosis associated with TIS (Campbell, 2004; Farley, 2014; Flynn, 2013).

The results of a prospective cohort study involving 63 subjects with early-onset scoliosis (EOS) and no rib abnormalities treated with VEPTR and followed for a mean of 2.2 years was reported by El-Hawary and others (2017). The mean age at time of implantation was 6.1 years and involved subjects with congenital (n=6), neuromuscular (n=36), syndromic (n=4), and idiopathic (n=17) scoliosis. The authors reported that the scoliosis major curve improved from a mean of 72 ± 18 degrees prior to treatment to 47 ± 17 degrees after surgery (p<0.0001). At 2 years, the mean measurement increased to 57 ± 18 degrees but still remained significant (p<0.0001). Similar results were reported for the secondary curve, with a mean of 42.8 degrees at baseline, 35 degrees postoperatively, and 39.6 degrees at 2 years (p=0.009). Subjects with syndromic or neuromuscular EOS had a higher percentage of change in sagittal spine length (SSL) and a decrease in kyphosis after 2 years. The authors noted that there was no correlation between pre-treatment severity and percentage of correction of the major curve. However, they did note that there was a larger percent correction of the secondary scoliosis in subjects with severe (>90 degrees) preoperative scoliosis. Successful treatment with VEPTR, defined as either the magnitude of scoliosis or truncal height as greater than or equal to preoperative measures, was noted in 54 subjects. SSL increased during the distraction phase of treatment in 82% of subjects for T1-T12 and 84% of subjects in T1-S1. Spine height growth during the 2 year follow-up represented 144% of expected age-matched coronal T1-T12 growth and 193% of expected age-matched coronal T1-S1 growth. Complications were reported in 31 (49%) of subjects, with a total of 58 complications reported. Device-related causes were identified for 39 of the complications. The highest number of complications were due to device migration (n=15). Overall, 86% of subjects had an improvement in scoliosis severity and 94% had an increased spinal height vs. preoperative measures. The authors concluded that, “This study proved that the spine continues to grow after VEPTR instrumentation during the distraction phase” and “We find this growth important as it proves continuous spine growth with VEPTR treatment.”

A paper published by Nossov (2017) reported the results of a retrospective cohort study investigating the impact of VEPTR on the need for respiratory assistance in 77 subjects with TIS and EOS who underwent VEPTR implantation while less than 10 years of age. All subjects had greater than 2 years follow-up data available and the mean was 5.6 years. The assisted ventilation rate (AVR) changed in 28 subjects (36%), with 29 (24%) improving and 90 (12%) deteriorating. The remaining 49 (64%) subjects had no change in AVR. Age was a significant factor in improvement, with the average age of subjects with improvement being 4, and the average age of subjects deteriorating was 6.7 (p<0.01). In total, 16 subjects improved to a normal AVR at last follow-up.

Waldenhausen and colleagues (2016) published the results of a prospective cohort study involving 65 subjects being treated for TIS with VEPTR. Several versions of the VEPTR device were used, including VEPTR I (n=35), VEPTR II (n=30) rib-to-rib and rib-to-spine devices, as well as modified VEPTR devices. The average age at time of implantation was 9.6 years (range 1.3-24.8) and the mean follow-up was 6.9 years (range 0.4-14.8). The overall average number of expansions was 5.5. Complication rates for those subjects classifiable using the early onset scoliosis classification system (C-EOS) were 21.7% for subjects with congenital scoliosis, 58.3% for those with neuromuscular scoliosis, 28.5% for those with syndromic scoliosis, and 50% for those with idiopathic scoliosis. Due to the small numbers, no significant differences were reported. For those not classifiable with C-EOS, the complication rate was 35.7%. Complications were experienced by 22 subjects with 37 total complications reported, 35 of which were deemed to have been device-associated. Most complications required at least 1 unplanned surgery, with 2 subjects needing device removal and one subject proceeding to a fusion procedure. Migration of the device was reported in 12 subjects who needed additional procedures to address. One subject experienced post-operative radiculopathy that spontaneously resolved. Implant fracture occurred in 3 subjects. The authors concluded that, “Use of the VEPTR for TIS was associated with significant complications.” and that, “VEPTR is usually salvaged and abandonment of a growth-friendly strategy is unusual.”

No definitive recommendations specific to VEPTR implantation have been issued by any of the specialty medical societies, to date. However, there is general consensus in the practice community that surgery is warranted to correct a spinal curvature with a Cobb angle greater than 45 degrees in skeletally immature individuals, in order to prevent further progression of the spinal curvature and the associated potential for pulmonary compromise.  

Definitions

Cobb angle: A measurement of the degree of spinal curvature. This is considered the standard measurement used in the practice community to quantify a scoliosis for the purpose of measuring curve progression over time. A curve is considered to be scoliosis at a Cobb angle of 10º or more. Any increase greater than 5º is considered a significant change indicative of curvature progression with scoliosis considered mild at 10º-24º, moderate at 25º-50º and severe at greater than 50º in skeletally mature individuals. Cobb angles greater than 45º are considered severe in skeletally immature persons.

Ellis-van Creveld syndrome: Autosomal recessive genetic disorder characterized by skeletal dysplasia.

Jarcho-Levin syndrome: Heritable axial skeleton growth disorder associated with malformation of the vertebral column and ribs.

Jeune syndrome: Congenital dwarfism associated with asphyxiating thoracic dystrophy.

Scoliosis: A musculoskeletal condition that involves abnormal lateral curvature of the spine. There are several different types of scoliosis that affect children and adolescents. The most common type is considered idiopathic but additional types of scoliosis include congenital, neuromuscular and syndromic scoliosis.

Thoracic insufficiency syndrome (TIS): This rare condition is defined by the FDA as, “The inability of the thorax to support normal respiration or lung growth. This would include patients with progressive congenital, neuromuscular, idiopathic, or syndromic scoliosis” (FDA, 2014).

References

Peer Reviewed Publications:

  1. Abol Oyoun N, Stuecker R. Bilateral rib-to-pelvis Eiffel Tower VEPTR construct for children with neuromuscular scoliosis: A preliminary report. Spine J. 2014; 14(7):1183-1191.
  2. Balioglu MB, Albayrak A, Akman YE, et al. The effect of vertical expandable prosthetic titanium rib on growth in congenital scoliosis. J Craniovertebr Junction Spine. 2015; 6(4):200-205.
  3. Campbell RM Jr. VEPTR: past experience and the future of VEPTR principles. Eur Spine J. 2013; 22 Suppl 2:S106-S117.
  4. Campbell RM Jr, Smith MD, Mayes TC, et al. The characteristics of thoracic insufficiency syndrome associated with fused ribs and congenital scoliosis. J Bone Joint Surg Am. 2003; 85(3):399-408.
  5. Campbell RM Jr, Smith MD, Mayes TC, et al. The effect of opening wedge thoracostomy on thoracic insufficiency syndrome associated with fused ribs and congenital scoliosis. J Bone Joint Surg Am. 2004; 86-A(8):1659-1674.
  6. El-Hawary R, Kadhim M, Vitale M, et al. VEPTR implantation to treat children with early-onset scoliosis without rib abnormalities: early results from a prospective multicenter study. J Pediatr Orthop. 2017; 37(8):e599-e605.
  7. Emans JB, Caubet JF, Ordonez CL, et al. The treatment of spine and chest wall deformities with fused ribs by expansion thoracostomy and insertion of vertical expandable prosthetic titanium rib: growth of thoracic spine and improvement of lung volumes. Spine. 2005; 30(17 Suppl):S58-68.
  8. Farley FA, Li Y, Jong N, et al. Congenital scoliosis SRS-22 outcomes in children treated with observation, surgery, and VEPTR. Spine (Phila Pa 1976). 2014; 39(22):1868-1874.
  9. Flynn JM, Emans JB, Smith JT, et al. VEPTR to treat nonsyndromic congenital scoliosis: a multicenter, mid-term follow-up study. J Pediatr Orthop. 2013; 33(7):679-684.
  10. Gadepalli SK, Hirschl RB, Tsai WC, et al. Vertical expandable prosthetic titanium rib device insertion: does it improve pulmonary function? J Pediatr Surg. 2011; 46(1):77-80.
  11. Hell AK, Campbell RM, Hefti F. The vertical expandable prosthetic titanium rib implant for the treatment of thoracic insufficiency syndrome associated with congenital and neuromuscular scoliosis in young children. J Pediatr Orthop B. 2005; 14(4):287-293.
  12. Karlin JG, Roth MK, Patil V, et al. Management of thoracic insufficiency syndrome in patients with Jarcho-Levin syndrome using VEPTRs (vertical expandable prosthetic titanium ribs). J Bone Joint Surg Am. 2014; 96(21):e181.
  13. LaGreca J, Flynn T, Cahill PJ, et al; Children’s Spine Study Group. Patients without intraoperative neuromonitoring (IONM) alerts during VEPTR implantation did not sustain neurological injury during subsequent routine expansions: A retrospective multicenter cohort study. J Pediatr Orthop. 2017; 37(8):e619-e624.
  14. Maruyama T, Takeshita K. Surgical treatment of scoliosis: a review of techniques currently applied. Scoliosis. 2008; 3:6.
  15. Motoyama EK, Deeney VF, Fine GF, et al. Effects on lung function of multiple expansion thoracoplasty in children with thoracic insufficiency syndrome: a longitudinal study. Spine. 2006; 31(3):284-290.
  16. Murphy RF, Moisan A, Kelly DM, et al. Use of vertical expandable prosthetic titanium rib (VEPTR) in the treatment of congenital scoliosis without fused ribs. J Pediatr Orthop. 2016; 36(4):329-335.
  17. Nossov SB, Curatolo E, Campbell RM, et al.; Children’s Spine Study Group. VEPTR: Are we reducing respiratory assistance requirements? J Pediatr Orthop. 2017 Apr 10. [Epub ahead of print].
  18. O'Brien A, Roth MK, Athreya H, et al. Management of thoracic insufficiency syndrome in patients with Jeune syndrome using the 70 mm radius vertical expandable prosthetic titanium rib. J Pediatr Orthop. 2015; 35(8):783-797.
  19. Waldhausen JH, Redding GJ, Song KM. Vertical expandable prosthetic titanium rib for thoracic insufficiency syndrome: a new method to treat an old problem. J Pediatr Surg. 2007; 42(1):76-80.
  20. Waldhausen JH, Redding G, White K, Song K. Complications in using the vertical expandable prosthetic titanium rib (VEPTR) in children. J Pediatr Surg. 2016; 51(11):1747-1750.
  21. White KK, Song KM, Frost N, et al. VEPTR growing rods for early-onset neuromuscular scoliosis: feasible and effective. Clin Orthop Relat Res. 2011; 469(5):1335-1341.

Government Agency, Medical Society, and Other Authoritative Publications:

  1. U.S. Food and Drug Administration. 510(k) Premarket Notification Database. Vertical Expandable Prosthetic Titanium Rib (VEPTR®-VEPTR II, DePuy Synthes® Spine, Raynham, MA). Summary of Safety and Effectiveness. No. K142587. Rockville, MD: FDA. November 18, 2014. Available at: http://www.accessdata.fda.gov/cdrh_docs/pdf14/k142587.pdf. Accessed on May 17, 2018.
  2. Vanderbilt University Medical Center. Vertical Expandable Prosthetic Titanium Rib (VEPTR) for Thoracic Insufficiency Syndrome (VEPTR). NLM Identifier: NCT02241954. Last updated April 3, 2018. Available at: https://clinicaltrials.gov/ct2/show/NCT02241954?term=NCT02241954&rank=1. Accessed on May 17, 2018.
Websites for Additional Information
  1. American Academy of Orthopedic Surgeons. Idiopathic Scoliosis in Children and Adolescents. Your Orthopedic Connection. March 2015. Available at: http://orthoinfo.aaos.org/topic.cfm?topic=A00353. Accessed on May 17, 2018.
  2. National Institute of Arthritis and Musculoskeletal and Skin Diseases. Questions and Answers about Scoliosis in Children and Adolescents. December 30, 2015. Available at: http://www.niams.nih.gov/Health_Info/Scoliosis/default.asp. Accessed on May 17, 2018.
  3. Scoliosis Research Society. Information about Thoracic Insufficiency Syndrome Treatment. Available at: https://www.srs.org/patients-and-families/conditions-and-treatments/parents/scoliosis/early-onset-scoliosis/thoracic-insufficiency-syndrome. Accessed on May 17, 2018.
Index

Scoliosis
Thoracic insufficiency syndrome, TIS
VEPTR
Vertical expandable prosthetic titanium rib

The use of specific product names is illustrative only. It is not intended to be a recommendation of one product over another, and is not intended to represent a complete listing of all products available.

History

Status

Date

Action

Reviewed

07/26/2018

Medical Policy & Technology Assessment Committee (MPTAC) review. References were updated.

 

05/03/2018

The document header wording updated from “Current Effective Date” to “Publish Date.”

Reviewed

08/03/2017

MPTAC review. Removed acronym from title. Updated Discussion and References sections.

Revised

08/04/2016

MPTAC review. Acronyms removed from medically necessary criteria. References were updated. Removed ICD-9 codes from Coding section.

Revised

08/06/2015

MPTAC review. The medically necessary indications and criteria for the VEPTR device were expanded to include certain severe, progressive spinal deformities, associated with, or at risk for, TIS including scoliosis in skeletally immature individuals. The Discussion section, Definitions and References were updated.

Reviewed

08/14/2014

MPTAC review. Discussion and References sections updated.

Reviewed

08/08/2013

MPTAC review. Discussion and References sections updated.

Reviewed

08/09/2012

MPTAC review. Definition and References sections updated.

Reviewed

08/18/2011

MPTAC review. Discussion, Definition, References and Index sections updated.

Reviewed

08/19/2010

MPTAC review. Discussion and reference link updated.

Reviewed

08/27/2009

MPTAC review. Discussion and references updated. Definition section added.

Reviewed

08/28/2008

MPTAC review. Description, discussion and references updated.

Reviewed

08/23/2007

MPTAC review. Addition of Not Medically Necessary statement for clarification. References updated. 

Revised

09/14/2006

MPTAC review. Updated language in criteria, description and discussion sections, references and coding. Added language about expansion, exchange and conversion procedures as medically necessary.

Revised

09/22/2005

MPTAC review. Revision based on Pre-merger Anthem and Pre-merger WellPoint Harmonization.

Pre-Merger Organizations

Last Review Date

Guideline Number

Title

Anthem, Inc.

 

 

No document

WellPoint Health Networks, Inc.

09/23/2004

3.07.20

Vertical Expandable Prosthetic Titanium Rib (VEPTR)