scholarly journals Characteristics, Treatment Patterns, Healthcare Resource Use, and Costs among Pediatric Patients Diagnosed with Neurofibromatosis Type 1 and Plexiform Neurofibromas: A Retrospective Database Analysis of a Medicaid Population

2020 ◽  
Author(s):  
Xiaoqin Yang ◽  
Kaushal Desai ◽  
Neha Agrawal ◽  
Kirti Mirchandani ◽  
Sagnik Chatterjee ◽  
...  
Keyword(s):  
Resource Use ◽  
Neurofibromatosis Type 1 ◽  
Pediatric Patients ◽  
Healthcare Resource ◽  
Neurofibromatosis Type ◽  
Treatment Patterns ◽  
Healthcare Resource Use ◽  
The Mean

Abstract Background: Neurofibromatosis type 1 (NF1)-related plexiform neurofibromas (PN) can cause substantial morbidity by disfigurement and compression of vital structures. Real-world data on the burden and cost of disease among pediatric patients with NF1 and PN is limited. The objectives of this study were to describe the characteristics, treatment patterns, healthcare resource use (HCRU), and costs of these patients.Results: A total of 383 patients were included in the retrospective analysis of patients aged ≤18 with at least 1 ICD-10-CM diagnosis code for both NF1 and PN enrolled in the MarketScan® Multistate Medicaid database from October 1, 2014 to December 31, 2017. The mean follow-up was 448 days. The mean age was 11.4 years and 52.0% of patients were male. Most patients were diagnosed by a specialist (63.5%). During the follow-up period, pain medications were used by 58.5% of patients, 25.1% were treated with chemotherapy, 7.1% received surgery for PN, 1.6% received MEK inhibitors, and 0.8% received radiation. Mean per patient per year inpatient, outpatient, emergency room, pharmacy, and other visits were 1.4, 17.3, 1.6, 13.6, and 25.8, respectively. Mean ±SD (median) total per patient per year healthcare costs (2018 USD) were $17,275 ±$61,903 ($2,889), with total medical costs of $14,628 ±$56,203 ($2,334) and pharmacy costs of $2,646 ±$13,303 ($26). Inpatient costs were the largest drivers of medical cost, with a mean per patient per year cost of $6,739.Conclusions: This study showed that many pediatric patients diagnosed with NF1 and PN were treated with supportive care only, highlighting a substantial unmet medical need. This study also highlights the considerable economic burden among patients with NF1 and PN.

scholarly journals Treatment, Resource Use and Costs Among Pediatric Patients with Neurofibromatosis Type 1 and Plexiform Neurofibromas

2020 ◽  
Vol Volume 11 ◽  
pp. 421-428
Author(s):  
Xiaoqin Yang ◽  
Kaushal Desai ◽  
Neha Agrawal ◽  
Kirti Mirchandani ◽  
Sagnik Chatterjee ◽  
...  
Keyword(s):  
Resource Use ◽  
Neurofibromatosis Type 1 ◽  
Pediatric Patients ◽  
Neurofibromatosis Type ◽  
Plexiform Neurofibromas

scholarly journals Outcomes of growing rods in a series of early-onset scoliosis patients with neurofibromatosis type 1

2020 ◽  
Vol 33 (3) ◽  
pp. 373-380
Author(s):  
Charlie Bouthors ◽  
Ruben Dukan ◽  
Christophe Glorion ◽  
Lotfi Miladi
Keyword(s):  
Neurofibromatosis Type 1 ◽  
Early Onset ◽  
Neurofibromatosis Type ◽  
Posterior Spinal Fusion ◽  
Early Onset Scoliosis ◽  
Mechanical Complication ◽  
Growing Rods ◽  
The Mean

OBJECTIVEEarly-onset scoliosis (EOS) is not uncommon in patients with neurofibromatosis type 1 (NF1). Despite conservative treatment, spinal deformities progress and require early surgical intervention. To avoid potential interference with chest and trunk growth, growing rods (GRs) have been used effectively in EOS of various etiologies. In this study the authors sought to analyze the outcomes of GRs in EOS patients with NF1.METHODSThis was a retrospective single-center cohort study that included consecutive EOS patients with NF1 who were treated with GRs and were followed up for a minimum of 2 years. Clinical and radiological analyses were performed preoperatively and until the last follow-up.RESULTSFrom to 2008 to 2017, 18 patients (6 male, 12 female) underwent GR surgery (14 single GRs, 4 dual GRs) at a mean age of 8 ± 2.1 years. Mean follow-up was 5 ± 2.4 years. Fifty-five lengthenings were performed at a mean rate of 3 lengthenings per patient (range 0–7). Ten of 14 single GRs (71%) were converted into dual GRs during treatment. No patient underwent definitive posterior spinal fusion (PSF) at GR treatment completion. The mean initial and last follow-up major curves were 57° and 36°, respectively (p < 0.001, 37% correction). The average T1–S1 increase was 13 mm/yr. Six of 9 hyperkyphotic patients had normal kyphosis at last follow-up. There were 26 complications involving 13 patients (72%), with 1 patient who required unplanned revision. The primary complications were instrumentation related, consisting of 17 proximal hook dislodgments, 6 distal pedicle screw pullouts, and 2 rod fractures. Only 1 patient experienced a mechanical complication after dual GR implantation. There were no wound infections.CONCLUSIONSThe GR technique provided satisfactory spinal deformity control in EOS patients with NF1 while allowing substantial spinal growth. Adequately contoured dual GRs with proximal hooks placed in nondystrophic regions should be used to minimize implant-related complications. Surgeons should not attempt to correct kyphosis at GR implantation.

scholarly journals Cohort study of healthcare use, costs and diagnoses from onset to 6 months after discharge for takotsubo syndrome in Sweden

BMJ Open ◽  
2019 ◽  
Vol 9 (2) ◽  
pp. e027814 ◽  
Author(s):  
Sara Wallström ◽  
Inger Ekman ◽  
Elmir Omerovic ◽  
Kerstin Ulin ◽  
Hanna Gyllensten
Keyword(s):  
Primary Care ◽  
Resource Use ◽  
Healthcare Costs ◽  
Healthcare Resource ◽  
Takotsubo Syndrome ◽  
Healthcare Resource Use ◽  
Direct Healthcare Costs ◽  
The Mean ◽  
Discharge From Hospital

ObjectiveLittle is known about the economic impact of takotsubo syndrome (TS) for patients and the health system after initial discharge from hospital. Therefore, the aim of this study was to describe the healthcare resource use and calculate direct healthcare costs for TS, from hospitalisation to 6 months after discharge, and explore the distribution of costs between TS and other diagnoses among patients with TS.Method, participants and settingCohort study investigating direct healthcare costs from hospitalisation, open specialised outpatient and primary care. Healthcare resource use during 6 months after diagnosis with TS was collected for 58 consecutive patients from the Regional Patient Register. Incidence-based direct healthcare costs, in 2015 values, were calculated using diagnosis-related group weights and unit costs from national statistics on healthcare costs.ResultsThe mean length of hospital stay was 10.2 days, index 6.4 and re-admissions 3.8 days. The mean number of follow-up encounters per patient was 15.6, of which two-thirds was specialised outpatient and one-third was primary care. This resulted in an average cost of €10 360. Of this, costs of €8026 (77.5%) occurred during encounters for which at least one of the registered conditions was cardiovascular. Costs differed little according to background characteristics.ConclusionThis study shows that patients utilise hospital, specialised outpatient and primary care after discharge for TS. Most direct healthcare costs relate to cardiac diagnoses. Patients with TS would probably benefit from a supportive follow-up programme after discharge from hospital.

scholarly journals Posterior-only surgical correction of dystrophic scoliosis in 31 patients with neurofibromatosis Type 1 using the multiple anchor point method

2017 ◽  
Vol 19 (1) ◽  
pp. 96-101 ◽  
Author(s):  
Ang Deng ◽  
Hong-Qi Zhang ◽  
Ming-Xing Tang ◽  
Shao-Hua Liu ◽  
Yu-Xiang Wang ◽  
...  
Keyword(s):  
Cobb Angle ◽  
Clinical Efficacy ◽  
Neurofibromatosis Type 1 ◽  
Neurofibromatosis Type ◽  
Point Method ◽  
Surgical Correction ◽  
Anchor Point ◽  
The Mean

OBJECTIVE The objective of this study was to evaluate the clinical efficacy of posterior-only surgical correction of dystrophic scoliosis in patients with neurofibromatosis Type 1 (NF1) using a multiple anchor point method (MAPM). METHODS From 2005 to 2014, 31 patients (mean age 13.5 years old, range 10–22 years old) suffering from dystrophic scoliosis associated with NF1 underwent posterior-only surgical correction using a MAPM. The apex of the deformity was thoracic (n = 25), thoracolumbar (n = 4), and lumbar (n = 2). The mean preoperative coronal Cobb angle was 69.1° (range 48.9°–91.4°). The mean Cobb angle on the side-bending radiograph of the convex side was 58.2° (range 40°–79.8°). The mean flexibility and apical vertebral rotation (AVR) were 15.6% (range 8.3%–28.2%) and 2.5° (range 2°–3°), respectively. The mean angle of sagittal kyphosis was 58.3° (range 34.1°–79.6°). RESULTS The mean follow-up period was 53 months (range 12–96 months). The mean postoperative coronal Cobb angle was 27.4° (range 16.3°–46.7°). Postoperatively, the mean AVR and angle of sagittal kyphosis were 1.2° (range 1°–2°) and 22.4° (range 4.2°–36.3°), respectively. All patients showed good correction of all indices postoperatively. The mean postoperative correction rate was 58.7% (range 46.3%–74.1%). At the final follow-up evaluation, the corrective loss rate of the Cobb angle was only 2.3%. Only 1 patient required revision surgery. No severe complications such as spinal cord, neural, or large vascular injury occurred during the operation. CONCLUSIONS Posterior-only surgical correction of dystrophic scoliosis in patients with NF1 using a MAPM could yield satisfactory clinical efficacy of correction and fusion.

scholarly journals Primary Hyperoxaluria Type 1 Disease Manifestations and Healthcare Utilization: A Multi-Country, Online, Chart Review Study

2021 ◽  
Vol 8 ◽  
Author(s):  
Xiangling Wang ◽  
David Danese ◽  
Thomas Brown ◽  
Jessica Baldwin ◽  
Gautam Sajeev ◽  
...  
Keyword(s):  
Kidney Disease ◽  
Resource Use ◽  
Kidney Stones ◽  
Healthcare Resource ◽  
Primary Hyperoxaluria ◽  
Healthcare Resource Use ◽  
Primary Hyperoxaluria Type 1 ◽  
Hyperoxaluria Type

Background: Primary hyperoxaluria type 1 (PH1) is a rare genetic disease that can result in irreversible damage to the kidneys and, eventually, extrarenal organs. While kidney failure is a known consequence of PH1, few studies to date have characterized clinical consequences of PH1 prior to kidney failure, and data on healthcare resource use outcomes across different stages of disease severity in PH1 are also limited. To help fill this knowledge gap, this study characterized the clinical and healthcare resource use (HRU) burden in patients with PH1 with varying stages of kidney disease.Methods: Nephrologists in the United States, Canada, United Kingdom, France, Germany, and Italy abstracted chart data from patients with PH1 under their care via an online questionnaire. Eligible patients had confirmed PH1 and ≥2 office visits from 2016 to 2019.Results: A total of 120 patients were analyzed (median age at diagnosis, 17.4 years old, median age at index 19.5 years old, median eGFR at index 45 ml/min/1.73 m2; median follow-up 1.7 years). During follow-up, the most common PH1 manifestations were kidney stones and urinary tract infections (UTIs, both 56.8%), and the most common symptoms were fatigue/weakness (71.7%) and pain (64.6%). With regard to HRU during follow-up, 37.4% required lithotripsy, 31.3% required ureteroscopy, and 9.6% required nephrolithotomy. PH1-related hospitalizations and emergency/urgent care visits were noted for 84.0 and 81.6% of patients, respectively.Conclusions: The current study demonstrated that patients with PH1 across various stages of kidney disease exhibited a substantial clinical burden, including kidney stones, UTIs, fatigue/weakness, and pain, and required frequent HRU, including kidney stone procedures, hospitalizations, and emergency visits. These findings highlight the significant morbidity and HRU burden in patients with PH1.

scholarly journals Neurofibromatosis Type 1 in a Pediatric Population: Ste-Justine's Experience

2005 ◽  
Vol 32 (2) ◽  
pp. 225-231 ◽  
Author(s):  
J.M. Boulanger ◽  
A. Larbrisseau
Keyword(s):  
Neurofibromatosis Type 1 ◽  
Attention Deficit Disorder ◽  
Pediatric Population ◽  
Laboratory Data ◽  
Neurofibromatosis Type ◽  
Optic Glioma ◽  
Increased Risk ◽  
The Mean

ABSTRACT:Background:To date, few pediatric series of neurofibromatosis type 1 (NF-1) have been described in the literature even though it is the most frequently encountered phakomatosis.Methods:We reviewed 987 charts of pediatric patients with a presumptive diagnosis of NF-1 who were evaluated at Ste-Justine hospital from January 1, 1991 to July 31, 2002. Patients who presented with two or more cardinal criteria were diagnosed with NF-1. Clinical and laboratory data were retrospectively collected, including: demographics, neuroimaging and presence or absence of associated symptoms or signs of NF-1.Results:A total of 279 patients were diagnosed with NF-1. The mean age at diagnosis was 3.4 years. Ninety-nine percent of the patients had café au lait spots and 47% had a first degree relative with NF-1. Almost 60 percent (59.6%) of those seen by an ophthalmologist had Lisch nodules. Optic glioma was found in in 14.7%, cutaneous neurofibromas in 38.4%, plexiform neurofibromas in 24.7%, neurofibrosarcoma in 1.8%, learning disabilities in 39%, attention deficit disorder in 40.5%, osseous dysplasias in 7.2%, pseudoarthrosis in 3.6%, precocious puberty in 3.2% and short stature in 17.9%. Magnetic resonance, when performed, showed hyperintense T2 lesions in 87.1% of cases. The mean period of follow-up was 7.4 years.Conclusion:Neurofibromatosis type 1 is a multisystemic disorder associated with increased risk of malignancy. It can be diagnosed at a very young age and clinical follow-up is advised. To our knowledge, this is the largest single center study of NF-1 in a pediatric population.

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