Update on Pulmonary Management in Spinal Muscular Atrophy type 1
|
|
- Jody Anabel Kelley
- 5 years ago
- Views:
Transcription
1 Update on Pulmonary Management in Spinal Muscular Atrophy type 1 David Zielinski, MD FRCPC, FCCP Associate Professor McGill University Montreal Children s Hospital
2 None Disclosures
3
4 Objectives Review genetic basis of Spinal Muscular Atrophy Review underlying pulmonary natural history of SMA type 1 Review management options for SMA type 1 Review the impact of disease modifying treatments on disease
5 Spinal Muscular Atrophy 2 nd most common fatal autosomal recessive disease after Cystic Fibrosis 1 in births 1/40-1/60 carrier status Degenerative disorder of alpha motor neurons in spinal cord Proximal muscle weakness/paralysis
6 Spinal Muscular Atrophy Onset Highest Function Life Expectancy Type 1 < 6 months Never sit <2 years of age Type months Never walk 70% alive at 25 years Type 3 >18 months Stand and walk ~Normal Type 4.>21 years Stand and walk Normal
7 Natural History Cobben; Neuromuscular Disorders 2008
8 Clinical subdivisions SMA1 SMA 1A present at birth, joint contractures, respiratory compromise SMA 1B: onset of symptoms before 3 months SMA 1C: onset of symptoms after 3 months
9 Survival and SMA 1 type Death or 16 hrs/day ventilation Finkel; Neurology 2014
10 Genetics Buchtbach; Front Mol BioScie 2016
11 Survival and SMN2 Death or 16 hrs/day ventilation Finkel; Neurology 2014
12
13 Options of Care Palliative Care Non-Invasive respiratory support Tracheostomy and ventilation
14 CTS Pediatric HMV Statement The option of invasive and noninvasive HMV support as well as the differences between therapeutic and palliative NIV should be presented to all parents of children with SMA. (Grade 1C) Children with SMA whose parents would like a respiratory opinion should be referred to an experienced center to discuss the treatment options. (Grade 1C) Amin et al 2017
15 CTS Pediatric HMV Statement The decision to recommend or not recommend ventilatory support to children with Type 1 and Type 2 SMA by the treating physician should be made on a case-by case basis after discussion with family and other caregivers, and a careful assessment of medical benefit as well as the effect on quality of life. (Grade 1C) Amin et al 2017
16 HMV and SMA1 in Canada Oskoui; Ped Pulm 2017
17 HMV and SMA1 in Canada Oskoui; Ped Pulm 2017
18 Palliative Care Palliative care options needs to be available as a real choice and option for families with new diagnosis of SMA type 1 Canada : 38% at time of diagnosis 15% at time of acute respiratory decompensation 8% based on family request 36% at variable times Oskoui; Ped Pulm 2017
19 Non-Invasive Support
20 Survival YOB YOB Death Death or Ventilation Oskoui; Neurology 2007
21 CTS Pediatric HMV Statement Assessment for sleep disordered breathing (ideally with a polysomnogram conducted as per the American Academy of Sleep Medicine standards) should be considered for children with SMA if this is in accordance with the child and family s goals of care. (Grade 1C) Amin et al 2017
22 Natural History
23 Chest Wall
24 Natural History
25 Respiratory Infections
26 Indications Non-Invasive Ventilation Early prophylactic Hospitalizations with Respiratory Failure Evidence of Sleep Disordered Breathing
27 Non-Invasive Ventilation 6 mths 18 mths Titrate Bipap for adequate chest wall expansion Usually needs ΔP >12 cm H20 From A. Simonds, Royal Brompton Hospital, UK / M. Schroth Wisonsin
28 High Span NIV 12 months: IPAP: 10, EPAP: 4 5 years: IPAP 16, EPAP: 4
29 High Span NIV 24/7 Ventilation; ΔP 17 cm H20 IPAP 20, EPAP 4; 11.4 hrs/day
30 High Span NIV 24/7 Ventilation; ΔP 17 cm H20 IPAP 20, EPAP 4; 11.4 hrs/day
31 Mechanical Cough Assist Pressures: +/- 40 cmh2o Ti/Te: 1-2 seconds 3-5 cycles CoughTrak Oscillations Use with percussion/other airway clearance techniques Recommend early initiation Habituation when the child is well
32 Gastrostomy Allows optimization of nutrition Prevent periods >4 hours of NPO +/- fundoplication
33 Gastrostomy Fundoplication Prevents aspiration risk, especially as non-invasive respiratory supports increase Extubation risk is minimal Durkin; J Pediatr Surg, 2008 No Fundoplication Can avoid general anesthesia Avoid complications of fundoplication Aspirations are rare
34 Acute Decompensations Management Principles Volume recruitment with effective ventilation Airway secretion mobilization and clearance Assessments SaO2 <95% is a marker of atelectasis/mucous plugging Resting heart rate >100 may be sign of increased distress Oxygen should not be the primary treatment of a low saturation
35 CTS Pediatric HMV Statement When and how to augment ventilatory support during acute illnesses should be made on a case-by-case basis for each patient by the treating physician, as there is no validated protocol at present. (Grade 1C) Amin et al 2017
36 Acute Decompensations Increased respiratory support needs Increased time on BiPAP Increased Pressures Increased airway clearance Cough assist q1-4 hours Increased nutritional demands Bach; Paed Resp Rev; 2008
37 CTS Pediatric HMV Statement If children are intubated, a protocol-led extubation, including the use of a mechanical in-exsufflation device and NIV, is recommended. (Grade 1C) Amin et al 2017
38 Bach; Chest 2010 Schroth, FMSA.org Extubation Protocol Extubation Criteria Afebrile SpO2 >94% on FiO2 of 0.21 Alert and Co-operative No atelectasis or collapse on chest radiograph Minimal airway suctioning required Extubation Protocol Do not wean to low pressure CPAP trials Avoid hypercapnia Avoid derecruitment Extubate to high span Bipap Aim for FiO2 of 0.21 with SaO2>94% Aim for normal CO2
39 SMA type 1 - Intubations 28 episodes of respiratory failure (24 for acute infectious) 48 intubation episodes
40 SMA type 1 - Intubations Oskoui; Ped Pulm 2017
41 Malik
42 MCH SMA 1 Experience At diagnosis families meet with Complex Care Pediatrics, Respirology, Neurology and Palliative Care Families who chose pro-active care Arrangements made to start NIV Cough assist and saturation monitor introduced and teaching done Swallowing assessment and NG tube Once NIV established -> gastrostomy and fundoplication
43 MCH SMA 1 Experience P7 P6 P5 P4 P3 P2 P1 SMN2 Copies 2 copies 6 (86%) 3 copies 1 (14%) SMA type SMA 1a 1 (14%) SMA 1b 4 (57%) SMA 1c 2 (28%) Onset of: Symptoms: 3 (0-5) months Age (Years) 1 st Resp Failure: 6.5 (2-17) months Disease modifying therapy trial (57%)
44 MCH SMA 1 Experience P7 P6 P5 P4 P3 P2 P1 Age (Years) Mean age 5.6 years NIV use (100%) 7 (2-12) months NIV ΔP 12 cm H20 (12-17) NIV hrs/day 13 hrs ( ) Ever Intubated (86%)
45 MCH SMA 1 Experience Respiratory Failure Admissions per year Year 1 Year 2 Year 3 Year 4 Year 5 Age (years) Admissions/year
46 MCH SMA 1 Experience 20 LOS days for Respiratory Admissions per year LOS/year 0 Year 1 Year 2 Year 3 Year 4 Year 5 Age (years)
47 MCH SMA 1 Experience P7 P6 P5 P4 Mortality or >16 hours/day ventilation 1 (17%) P3 P2 P1 Age (Years)
48
49 Tracheostomy Pro No facial interface Easier control of airway Easier suctioning Easier to treat intercurrent illnesses Prolongs survival Improves quality of life CON?loss of speaking Increased secretions locked in syndrome Progression of disease Worsens quality of life Requires Experienced/trained personnel
50 Tracheostomy n=194 Gregoretti; Pediatrics 2013
51 Tracheostomy
52 Nusinersen Anitsense oligonucleotide Modifies pre-mrna slicing of SMN2 to increase functional protein International multicenter RCT with Sham injection as placebo 80 infants in active arm 40 infants in sham arm Inthratecal injections on days 1, 15, 29, 64, 183 and 302 Eligible: 2 SMN2 copies Diagnosed <6 months of age Normal room air gas exchange Nocturnal NIV permitted Singh 2017 Finkel; NEJM 2017
53 Nusinersen Intern analysis: Motor-milestone response: 41% vs 0% (p<0.001) Study was stopped prematurely Final analysis: Motor-milestone response: 51% vs 0% (p<0.001) 22% full head control 10% roll over 8% sit independently 1% stand Death or >16 hour/day ventilation: 39% vs 68% (P=0.005) Finkel; NEJM 2017
54 Disease Modification
55 Disease Modification
56
57 Single vial: $118,000 Nusinersen Price Year 1: $708,000 Subsequent years: $354,000 CDTAH Common Drug Review December 2017
58 Nusinersen in Canada CADTH Recognize value for SMA type 1 with: 2 SMN2 copies <7 months of age Not on invasive ventilation Show a clinical response within 6 doses to continue Suggested price too high Ongoing price negotiation and province each will decide Did not recommend for SMA type 2 or 3 INESSS Recognize therapeutic value for SMA type 1 Recommended not to list due to high cost Ministry of Health to decide Did not recommend for SMA type 2 or 3 Ministry agreed with recommendation
59 Gene Replacement One time IV injection delivery of adenoassociated viral serotype 9 to deliver copy of SMN Crosses blood-brain barrier 1 mg/kg of Prednisone for 30 days Mendell; NEJM 2017
60 Gene Replacement 11 sat 9 sat >30 sec 9 rolled 2 walked 4 feeding orally (hand to mouth) Mendell; NEJM 2017
61 Summary SMA type 1 has a varying but severe natural history Treatment options to prolong survival Early high span NIV, airway clearance, nutrition Having an approach to sick management is essential New disease modifying treatments are available for SMA type 1 They are changing the progression of disease Significant resource challenges are awaiting us
62
63 Jessika is 3 and a half years old, she has spent approximately 30 days, sick, in hospital care since her diagnosis three years ago. Not a lot considering the bleak outlook that was painted for us of her not living past her second birthday. In our opinion that is due partly to our proactive use of NIV care. We wanted Jessika on the BiPap as soon as we were aware of NIV protocol, to keep her lungs as strong as possible, she uses it everyday during her nap and every night when she sleeps. We wanted to start a chest physio routine right away as well, which included breath-stacking, cough assist, suctioning and chest tapping. We did this everyday since we have had the machines, and now do her chest physio routine 3 times a day when Jessika is healthy and, on the odd occasions when she is sick, at least every 4 hours. Jessika is amazingly happy and intelligent and we appreciate every moment with her as does she. Not only has she surpassed our expectations for longevity but her speech is extremely advanced and clear, something that was not expected from an SMA type 1 child. And Jessika is a world away from being a type 2. She speaks and sings clearly and loudly even when she is wearing her BiPap. Every parent has the right to choose their own path, some choose proactive care, others choose palliative, some are totally lost. These parents should be able to make informed decisions though, based on real world knowledge of this disease and not just the textbook diagnosis. Due to NIV protocols, recent technological advances, and the hospital's approach to treating our children with SMA, we are seeing longer lifespans and greater quality of life. We should also mention that even though taking care of a child with SMA is a monumental task, think full time job with lots of overtime, it is made so much easier knowing that we have a great support network behind us which made up mostly of dedicated, open minded, caring health care professionals, such as the fantastic resp. team from the Mtl Children's. Anyone who wants to know more about Jessika is welcome to visit either her website or her facebook page.
What is the next best step?
Noninvasive Ventilation William Janssen, M.D. Assistant Professor of Medicine National Jewish Health University of Colorado Denver Health Sciences Center What is the next best step? 65 year old female
More informationPreventing Respiratory Complications of Muscular Dystrophy
Preventing Respiratory Complications of Muscular Dystrophy Jonathan D. Finder, MD Professor of Pediatrics University of Pittsburgh School of Medicine Children s Hospital of Pittsburgh Introduction Respiratory
More informationNON INVASIVE LIFE SAVERS. Non Invasive Ventilation (NIV)
Table 1. NIV: Mechanisms Of Action Decreases work of breathing Increases functional residual capacity Recruits collapsed alveoli Improves respiratory gas exchange Reverses hypoventilation Maintains upper
More informationClinical Policy Bulletin: Nusinersen (Spinraza)
Clinical Policy Bulletin: Nusinersen (Spinraza) Number: 0915 Policy *Pleasesee amendment forpennsylvaniamedicaidattheendofthiscpb. Note: REQUIRES PRECERTIFICATION.Footnotes for Precertification of nusinersen
More informationPOLICY. Number: Title: APPLICATION OF NON INVASIVE VENTILATION FOR ACUTE RESPIRATORY FAILURE. Authorization
POLICY Number: 7311-60-024 Title: APPLICATION OF NON INVASIVE VENTILATION FOR ACUTE RESPIRATORY FAILURE Authorization [ ] President and CEO [ x ] Vice President, Finance and Corporate Services Source:
More informationSpinal Muscular Atrophy: Case Study. Spinal muscular atrophy (SMA) is a fairly common genetic disorder, affecting
Spinal Muscular Atrophy: Case Study Spinal muscular atrophy (SMA) is a fairly common genetic disorder, affecting approximately one in 6,000 babies. It is estimated that one in every 40 Americans carries
More informationAFCH NEUROMUSCULAR DISORDERS (NMD) PROTOCOL
AFCH NEUROMUSCULAR DISORDERS (NMD) PROTOCOL A. Definition of Therapy: 1. Cough machine: 4 sets of 5 breaths with a goal of I:E pressures approximately the same of 30-40. Inhale time = 1 second, exhale
More informationDiagnosis, management and new treatments for Spinal Muscular Atrophy Special Focus: SMA Type 1
Diagnosis, management and new treatments for Spinal Muscular Atrophy Special Focus: SMA Type 1 17 th April 2018 Adnan Manzur Consultant Paediatric Neurologist Dubowitz Neuromuscular Centre, GOSH & ICH,
More informationPediatric Patients. Neuromuscular Disease. Teera Kijmassuwan, MD Phetcharat Netmuy, B.N.S., MA Oranee Sanmaneechai, MD : Preceptor
Patient Management Pediatric Patients with Neuromuscular Disease Teera Kijmassuwan, MD Phetcharat Netmuy, B.N.S., MA Oranee Sanmaneechai, MD : Preceptor Case Thai boy 1 year old Present with Respiratory
More informationMechanical Ventilation of the Patient with Neuromuscular Disease
Mechanical Ventilation of the Patient with Neuromuscular Disease Dean Hess PhD RRT Associate Professor of Anesthesia, Harvard Medical School Assistant Director of Respiratory Care, Massachusetts General
More informationNON-INVASIVE VENTILATION. Lijun Ding 23 Jan 2018
NON-INVASIVE VENTILATION Lijun Ding 23 Jan 2018 Learning objectives What is NIV The difference between CPAP and BiPAP The indication of the use of NIV Complication of NIV application Patient monitoring
More informationOXYGEN USE IN PHYSICAL THERAPY PRACTICE. Rebecca H. Crouch, PT,DPT,MS,CCS,FAACVPR
OXYGEN USE IN PHYSICAL THERAPY PRACTICE Rebecca H. Crouch, PT,DPT,MS,CCS,FAACVPR Supplemental Oxygen Advantages British Medical Research Council Clinical Trial Improved survival using oxygen 15 hrs/day
More informationUnderstanding Breathing Muscle Weakness
Understanding Breathing Muscle Weakness A N D R E A L. K L E I N P R E S I D E N T / F O U N D E R B R E A T H E W I T H M D w w w.facebook.com/ b r e a t h e w i t h m d h t t p : / / w w w. b r e a t
More informationNIV - BI-LEVEL POSITIVE AIRWAY PRESSURE (BIPAP)
Introduction NIV - BI-LEVEL POSITIVE AIRWAY PRESSURE (BIPAP) Noninvasive ventilation (NIV) is a method of delivering oxygen by positive pressure mask that allows for the prevention or postponement of invasive
More informationApproach to type 2 Respiratory Failure
Approach to type 2 Respiratory Failure Changing Nature of NIV Not longer just the traditional COPD patients Increasingly Obesity Neuromuscular Pneumonias 3 fold increase in patients with Ph 7.25 and below
More informationSpinal Muscular Atrophy: What s new in the management of pediatric neuromuscular disease
Spinal Muscular Atrophy: What s new in the management of pediatric neuromuscular disease Mary Schroth MD Professor, Pediatric Pulmonology University of Wisconsin School of Medicine and Public Health, Madison
More informationNeuromuscular diseases (NMDs) include both hereditary and acquired diseases of the peripheral neuromuscular system. They are diseases of the
Neuromuscular diseases (NMDs) include both hereditary and acquired diseases of the peripheral neuromuscular system. They are diseases of the peripheral nerves (neuropathies and anterior horn cell diseases),
More informationNoninvasive Mechanical Ventilation in Children ศ.พญ.อร ณวรรณ พฤทธ พ นธ หน วยโรคระบบหายใจเด ก ภาคว ชาก มารเวชศาสตร คณะแพทยศาสตร โรงพยาบาลรามาธ บด
Noninvasive Mechanical Ventilation in Children ศ.พญ.อร ณวรรณ พฤทธ พ นธ หน วยโรคระบบหายใจเด ก ภาคว ชาก มารเวชศาสตร คณะแพทยศาสตร โรงพยาบาลรามาธ บด Noninvasive Mechanical Ventilation Provide support without
More informationNon-invasive Positive Pressure Mechanical Ventilation: NIPPV: CPAP BPAP IPAP EPAP. My Real Goals. What s new in 2018? OMG PAP?
Non-invasive Positive Pressure Mechanical Ventilation: What s new in 2018? Geoffrey R. Connors, MD, FACP Associate Professor of Medicine University of Colorado School of Medicine Division of Pulmonary
More informationRespiratory Management of Facioscapulohumeral Muscular Dystrophy. Nicholas S. Hill, MD Tufts Medical Center Boston, MA
Respiratory Management of Facioscapulohumeral Muscular Dystrophy Nicholas S. Hill, MD Tufts Medical Center Boston, MA Respiratory Involvement in FSHD Very variable time of onset rate of progression Muscles
More informationSMA Treatments and Clinical Trials. Kenneth Hobby, President Mary Schroth, MD, Chief Medical Officer Jill Jarecki, PhD, Chief Scientific Officer
SMA Treatments and Clinical Trials Kenneth Hobby, President Mary Schroth, MD, Chief Medical Officer Jill Jarecki, PhD, Chief Scientific Officer Agenda Introduction Kenneth Hobby, President Spinraza Update
More information11/20/2015. Beyond CPAP. No relevant financial conflicts of interest. Kristie R Ross, M.D. November 12, Describe advanced ventilation options
Beyond CPAP Kristie R Ross, M.D. November 12, 2015 No relevant financial conflicts of interest Sponsored by The Warren Alpert Medical School of Brown University Describe advanced ventilation options Compare
More informationKENNEDY DISEASE PULMONARY CONSIDERATIONS: SCIENCE & MANAGEMENT STRATEGIES
KENNEDY DISEASE PULMONARY CONSIDERATIONS: SCIENCE & MANAGEMENT STRATEGIES When you can t breathe nothing else matters American Lung Association Noah Lechtzin, MD; MHS Associate Professor of Medicine Johns
More informationLetter of Medical Necessity The Use of SPINRAZA (nusinersen) for Spinal Muscular Atrophy
TEMPLATE Letter of Medical Necessity The Use of SPINRAZA (nusinersen) for Spinal Muscular Atrophy Date: [Insert Name of Medical Director] RE: Patient Name [ ] [Insurance Company] Policy Number [ ] [Address]
More informationRespiratory Management- Your Questions Answered! Michelle Chatwin, PhD Consultant Physiotherapist
Respiratory Management- Your Questions Answered! Michelle Chatwin, PhD Consultant Physiotherapist Why Are People Affected Differently Neuromuscular Disease; A Spectrum Its severity varies widely within
More informationAdapting to the Worsening of the LTMV Patient
14 èmes Journées Internationales de Ventilation à Domicile LYON, 26-28 mars 2015 Adapting to the Worsening of the LTMV Patient Andrea Vianello Fisiopatologia e Terapia Intensiva Respiratoria Ospedale-Università
More informationPrepared by : Bayan Kaddourah RN,MHM. GICU Clinical Instructor
Mechanical Ventilation Prepared by : Bayan Kaddourah RN,MHM. GICU Clinical Instructor 1 Definition Is a supportive therapy to facilitate gas exchange. Most ventilatory support requires an artificial airway.
More informationWhere do we come from?
Interdisciplinary Decision-Making with Patients Requiring Tracheostomy and Carrie Windhorst MS CCC-SLP Cheryl Wagoner MS CCC-SLP Ricque Harth MEd CCC-SLP Where do we come from? Welcome to Madonna Rehabilitation
More informationCystic Fibrosis Complications ANDRES ZIRLINGER, MD STANFORD UNIVERSITY MEDICAL CENTER MARCH 3, 2012
Cystic Fibrosis Complications ANDRES ZIRLINGER, MD STANFORD UNIVERSITY MEDICAL CENTER MARCH 3, 2012 INTRODUCTION PNEUMOTHORAX HEMOPTYSIS RESPIRATORY FAILURE Cystic Fibrosis Autosomal Recessive Genetically
More informationBy Mark Bachand, RRT-NPS, RPFT. I have no actual or potential conflict of interest in relation to this presentation.
By Mark Bachand, RRT-NPS, RPFT I have no actual or potential conflict of interest in relation to this presentation. Objectives Review state protocols regarding CPAP use. Touch on the different modes that
More informationAlternative title: Confessions of a Mucus Enthusiast. Mechanical Insufflation Exsufflation for airway secretion clearance and lung expansion therapy
Mechanical Insufflation Exsufflation for airway secretion clearance and lung expansion therapy Alternative title: Confessions of a Mucus Enthusiast Marty Davig, RRT RCP Philips Respironics Inc. Objectives
More informationSpinal Muscular Atrophy in 2017
Spinal Muscular Atrophy in 2017 Leigh Maria Ramos-Platt, MD Children s Hospital of Los Angeles University of Southern California Keck School of Medicine Disclosures MDA Care Center Grant In this presentation,
More informationSpinraza (Nusinersen) Drug Prior Authorization Protocol (Medical Benefit & Part B Benefit)
Line of Business: All Lines of Business Effective Date: August 16, 2017 Spinraza (Nusinersen) Drug Prior Authorization Protocol (Medical Benefit & Part B Benefit) This policy has been developed through
More informationNIV indications for children with rare diseases
NIV indications for children with rare diseases Renato Cutrera Dir. U.O.C. Broncopneumologie Département de Médecine Pédiatrique Hôpital Bambino Gesù, IRCCS, Rome, Italie R. Cutrera, 2015 - cutrera@opbg.net
More informationCigna Drug and Biologic Coverage Policy
Cigna Drug and Biologic Coverage Policy Subject Nusinersen Table of Contents Coverage Policy... 1 General Background... 2 Coding/Billing Information... 5 References... 5 Effective Date... 10/15/2017 Next
More informationIndex. sleep.theclinics.com. Note: Page numbers of article titles are in boldface type.
Index Note: Page numbers of article titles are in boldface type. A Acquired central hypoventilation syndrome, NPPV in children with, 475 Acute cardiogenic pulmonary edema, PAP therapy in, 394 395 Adaptive
More informationAcute NIV in COPD and what happens next. Dr Rachael Evans PhD Associate Professor, Respiratory Medicine, Glenfield Hospital
Acute NIV in COPD and what happens next Dr Rachael Evans PhD Associate Professor, Respiratory Medicine, Glenfield Hospital Content Scenarios Evidence based medicine for the first 24 hrs Who should we refer
More informationHome Mechanical Ventilation. Anthony Bateman
Home Mechanical Ventilation Anthony Bateman What is Long Term Ventilation? LTV is the provision of respiratory support to individuals with non-acute respiratory failure Progression of expected disease
More informationRESPIRATORY PROBLEMS IN MND RICHARD HARRISON MND RESPIRATORY ASSESSMENT SERVICE LUNG HEALTH UNIVERSITY HOSPITAL OF NORTH TEES
RESPIRATORY PROBLEMS IN MND RICHARD HARRISON MND RESPIRATORY ASSESSMENT SERVICE LUNG HEALTH UNIVERSITY HOSPITAL OF NORTH TEES A TYPICAL HISTORY: NON BULBAR ONSET Difficulty walking Weak hands and arms
More informationCADTH Canadian Drug Expert Committee Recommendation
CADTH COMMON DRUG REVIEW CADTH Canadian Drug Expert Committee Recommendation (FINAL) Nusinersen (Spinraza Biogen Canada Inc.) Indication: Treatment of 5q Spinal Muscular Atrophy RECOMMENDATION: The CADTH
More informationPedsCases Podcast Scripts
PedsCases Podcast Scripts This is a text version of a podcast from Pedscases.com on Non-Invasive Ventilation in Pediatric Medicine. These podcasts are designed to give medical students an overview of key
More informationMotor Neurone Disease NICE to manage Management of ineffective cough. Alex Long Specialist NIV/Respiratory physiotherapist June 2016
Motor Neurone Disease NICE to manage Management of ineffective cough Alex Long Specialist NIV/Respiratory physiotherapist June 2016 Content NICE guideline recommendations Respiratory involvement in MND
More informationSCOTTISH MUSCLE NETWORK DUCHENNE MUSCULAR DYSTROPHY TRANSITION SOME USEFUL THINGS TO KNOW ABOUT HEALTH AROUND ADOLESCENCE
SCOTTISH MUSCLE NETWORK DUCHENNE MUSCULAR DYSTROPHY TRANSITION SOME USEFUL THINGS TO KNOW ABOUT HEALTH AROUND ADOLESCENCE 02 Changes in our lives can be stressful and leaving school and moving into the
More information9/30/2017. SMA: Spinal Muscular Atrophy Learning Outcomes: SMA. Prevalence. Review of the diagnosis. Here are the odds
SMA: Spinal Muscular Atrophy Learning Outcomes: 1 in 6,000 to 1 in 10,000 but 100% to CRT Kay Koch, OTR/L, ATP The van Halem Group, A Division of VGM kkotrchoa@yahoo.com The participant will be able to
More informationTracheostomy and Ventilator Education Program Module 10: Communication Supplies
Tracheostomy and Ventilator Education Program Module 10: Communication Supplies Disclaimer This material is intended for use by trained family members and caregivers of children with tracheostomies who
More informationSpinal Muscular Atrophy Newborn Screening
10.2018 Spinal Muscular Atrophy Newborn Screening Melissa Gibbons, MS, CGC Erica Wright, MS, CGC Clinical Genetics and Metabolism Department of Pediatrics Children s Hospital Colorado/ University of Colorado
More informationMASTER SYLLABUS
MASTER SYLLABUS 2018-2019 A. Academic Division: Health Science B. Discipline: Respiratory Care C. Course Number and Title: RESP 2490 Practicum IV D. Course Coordinator: Tricia Winters, BBA, RRT, RCP Assistant
More informationOxygen and ABG. Dr Will Dooley
Oxygen and ABG G Dr Will Dooley Oxygen and ABGs Simply in 10 cases Recap of: ABG interpretation Oxygen management Some common concerns A-a gradient Base Excess Anion Gap COPD patients CPAP/BiPAP First
More informationCOMPANY OVERVIEW. June 2016
COMPANY OVERVIEW June 2016 Disclaimers Forward-Looking Statements This presentation contains forward-looking statements, including: statements about: the timing, progress and results of preclinical studies
More informationHyperinflation Therapy and the Tools to Accomplish It!! Bill Barnes, RN, RRT Good Shepherd Rehabilitation Network
Hyperinflation Therapy and the Tools to Accomplish It!! Bill Barnes, RN, RRT Good Shepherd Rehabilitation Network HYPERINFLATION THERAPY Challenges in Post Acute Care Deconditioning Malnutrition Hydration
More informationAEROSURF Phase 2 Program Update Investor Conference Call
AEROSURF Phase 2 Program Update Investor Conference Call November 12, 2015 Forward Looking Statement To the extent that statements in this presentation are not strictly historical, including statements
More informationI. Subject: Pressure Support Ventilation (PSV) with BiPAP Device/Nasal CPAP
I. Subject: Pressure Support Ventilation (PSV) with BiPAP Device/Nasal CPAP II. Policy: PSV with BiPAP device/nasal CPAP will be initiated upon a physician's order by Respiratory Therapy personnel trained
More informationRespiratory implications of motor neurone disease
Respiratory implications of motor neurone disease Dr Mark Elliott Mark.elliott2@nhs.net St. James s University Hospital Spontaneous breathing overnight -air Think of the diagnosis Respiratory muscle involvement
More informationCorporate Medical Policy
Corporate Medical Policy File Name: Origination: Last CAP Review: Next CAP Review: Last Review: nusinersen_spinraza 03/2017 10/2018 10/2019 10/2018 Description of Procedure or Service Spinal muscular atrophy
More informationTracheostomy and Ventilator Education Program Module 2: Respiratory Anatomy
Tracheostomy and Ventilator Education Program Module 2: Respiratory Anatomy Disclaimer This material is intended for use by trained family members and caregivers of children with tracheostomies who are
More informationSpinraza (nusinersen)
Spinraza (nusinersen) Policy Number: 5.02.540 Last Review: 04/2018 Origination: 03/2017 Next Review: 04/2019 Policy Blue Cross and Blue Shield of Kansas City (Blue KC) will provide coverage for Spinraza
More information(Non)-invasive ventilation: transition from PICU to home. Christian Dohna-Schwake
(Non)-invasive ventilation: transition from PICU to home Christian Dohna-Schwake Increased use of NIV in PICUs over last 15 years First choice of respiratory support in many diseases Common temporary indications:
More informationNeonatal Respiratory Physiotherapy. Nicky Hawkes Advanced Respiratory Physiotherapist Oct 2011
Neonatal Respiratory Physiotherapy Nicky Hawkes Advanced Respiratory Physiotherapist Oct 2011 Respiratory Physiotherapy?..Not just percussion! Assessment of baby s respiratory status and deciding what
More informationCondensed version.
I m Stu 3 Condensed version smcvicar@uwhealth.org Listen 1. Snoring 2. Gurgling 3. Hoarseness 4. Stridor (inspiratory/expiratory) 5. Wheezing 6. Grunting Listen Crackles Wheezing Stridor Absent Crackles
More informationHypoventilation? Obstructive Sleep Apnea? Different Tests, Different Treatment
Hypoventilation? Obstructive Sleep Apnea? Different Tests, Different Treatment Judith R. Fischer, MSLS, Editor, Ventilator-Assisted Living (fischer.judith@sbcglobal.net) Thanks to Josh Benditt, MD, University
More informationAirway Clearance Devices
Print Page 1 of 11 Wisconsin.gov home state agencies subject directory department of health services Search Welcome» August 2, 2018 5:18 PM Program Name: BadgerCare Plus and Medicaid Handbook Area: Durable
More informationRon Hosp, MS-HSA, RRT Regional Respiratory Specialist. This program has been approved for 1 hour of continuing education credit.
Ron Hosp, MS-HSA, RRT Regional Respiratory Specialist This program has been approved for 1 hour of continuing education credit. Course Objectives Identify at least four goals of home NIV Identify candidates
More informationSPINRAZA (NUSINERSEN)
SPINRAZA (NUSINERSEN) UnitedHealthcare Commercial Medical Benefit Drug Policy Policy Number: 2018D0059D Effective Date: April 1, 2018 Table of Contents Page INSTRUCTIONS FOR USE... 1 BENEFIT CONSIDERATIONS...
More informationI Need to Cough Ways to Keep Your Airways Clear
I Need to Cough Ways to Keep Your Airways Clear 2018 Annual Cure SMA Conference Richard Kravitz, MD Duke University School of Medicine Duke University Medical Center Durham, North Carolina Presenters Jane
More informationNon-invasive Ventilation protocol For COPD
NHS LANARKSHIRE MONKLANDS HOSPITAL Non-invasive Ventilation protocol For COPD April 2017 S Baird Review Date: Oct 2019 Approved by Medical Directorate Indications for Non-Invasive Ventilation (NIV) NIV
More informationMND Study Day. Martin Latham CNS Leeds Sleep Service
MND Study Day Martin Latham CNS Leeds Sleep Service Objectives: Identifying individuals at risk. Understand issues related to NIV. Understand issues related to secretion management Improve outcomes. Identifying
More informationBi-Level Therapy: Boosting Comfort & Compliance in Apnea Patients
Bi-Level Therapy: Boosting Comfort & Compliance in Apnea Patients Objectives Describe nocturnal ventilation characteristics that may indicate underlying conditions and benefits of bilevel therapy for specific
More informationDuchenne Muscular Dystrophy
Authors: Elia Gomez-Merino, MD John R. Bach, MD Affiliations: From the Department of Pulmonary Medicine, Hospital Clinico Universitario de San Juan, San Juan de Alicante, Spain (EGM), and the Department
More informationCrit Vent Bundle for Mechanical Ventilation (337) [337] Physician - Also, enter Critical Care Admission Orders
Crit Vent Bundle for Mechanical Ventilation (337) [337] Physician - Also, enter Critical Care Admission Orders Initial Vent Settings (Single Response) [6360] If no previous orders and no choice made by
More informationMechanical Ventilation Principles and Practices
Mechanical Ventilation Principles and Practices Dr LAU Chun Wing Arthur Department of Intensive Care Pamela Youde Nethersole Eastern Hospital 6 October 2009 In this lecture, you will learn Major concepts
More information(To be filled by the treating physician)
CERTIFICATE OF MEDICAL NECESSITY TO BE ISSUED TO CGHS BENEFICIAREIS BEING PRESCRIBED BILEVEL CONTINUOUS POSITIVE AIRWAY PRESSURE (BI-LEVEL CPAP) / BI-LEVEL VENTILATORY SUPPORT SYSTEM Certification Type
More informationRecent Advances in Respiratory Medicine
Recent Advances in Respiratory Medicine Dr. R KUMAR Pulmonologist Non Invasive Ventilation (NIV) NIV Noninvasive ventilation (NIV) refers to the administration of ventilatory support without using an invasive
More informationNIV use in ED. Dr. Khalfan AL Amrani Emergency Resuscitation Symposium 2 nd May 2016 SQUH
NIV use in ED Dr. Khalfan AL Amrani Emergency Resuscitation Symposium 2 nd May 2016 SQUH Outline History & Introduction Overview of NIV application Review of proven uses of NIV History of Ventilation 1940
More informationClinical Review Report
CADTH COMMON DRUG REVIEW Clinical Review Report Nusinersen (Spinraza) (Biogen Canada Inc.) Indication: Treatment of patients with 5q SMA Service Line: CADTH Common Drug Review Version: Version 1.0 Publication
More informationCourse Handouts & Disclosure
ALS: DISEASE TRAJECTORY AND HOSPICE ELIGIBILITY Terri L. Maxwell PhD, APRN VP, Strategic Initiatives Weatherbee Resources Inc Hospice Education Network Inc Course Handouts & Disclosure To download presentation
More informationThe objectives of this presentation are to
1 The objectives of this presentation are to 1. Review the mechanics of airway clearance 2. Understand the difference between secretion mobilization and secretion clearance 3. Identify conditions that
More informationLUNG VOLUME RECRUITMENT IN NEUROMUSCULAR DISEASE
LUNG VOLUME RECRUITMENT IN NEUROMUSCULAR DISEASE Sherri Katz, MDCM, MSc, FRCPC Pediatric Respirologist Children s Hospital of Eastern Ontario University of Ottawa Disclosures Research funding from CIHR,
More informationThe 2017 Update of the Standard of Care Recommendations for Spinal Muscular Atrophy
Richard Finkel, MD Nemours Children s Hospital Orlando, FL, USA Thomas Crawford, MD Johns Hopkins Hospital Baltimore, MD, USA The 2017 Update of the Standard of Care Recommendations for Spinal Muscular
More informationSample Case Study. The patient was a 77-year-old female who arrived to the emergency room on
Sample Case Study The patient was a 77-year-old female who arrived to the emergency room on February 25 th with a chief complaint of shortness of breath and a deteriorating pulmonary status along with
More informationNATIONAL INSTITUTE FOR HEALTH AND CLINICAL EXCELLENCE SCOPE
NATIONAL INSTITUTE FOR HEALTH AND CLINICAL EXCELLENCE 1 Guideline title SCOPE Motor neurone disease: the use of non-invasive ventilation in the management of motor neurone disease 1.1 Short title Motor
More informationNusinersen Use in Spinal Muscular Atrophy
Nusinersen Use in Spinal Muscular Atrophy Report by: Guideline Development, Dissemination, and Implementation Subcommittee of the American Academy of Neurology Slide 1 Evidence in Focus Endorsement and
More informationNoninvasive Ventilation: Non-COPD Applications
Noninvasive Ventilation: Non-COPD Applications NONINVASIVE MECHANICAL VENTILATION Why Noninvasive Ventilation? Avoids upper A respiratory airway trauma system lacerations, protective hemorrhage strategy
More informationMechanical Ventilation 1. Shari McKeown, RRT Respiratory Services - VGH
Mechanical Ventilation 1 Shari McKeown, RRT Respiratory Services - VGH Objectives Describe indications for mcvent Describe types of breaths and modes of ventilation Describe compliance and resistance and
More informationPain patient with sleep-disordered breathing
Pain patient with sleep-disordered breathing Clinical scenario: A 50-year old female with a history of acid reflux, fibromyalgia, spinal stenosis and degenerative disk disease s/p C3/C4 disk fusion, depression
More informationBTS/ICS Guidelines for the ventilatory management of acute hypercapnic respiratory failure in adults
BTS/ICS Guidelines for the ventilatory management of acute hypercapnic respiratory failure in adults British Thoracic Society Intensive Care Society Introduction Acute Hypercapnic Respiratory Failure (AHRF)
More informationSMA IS A SEVERE NEUROLOGICAL DISORDER [1]
SMA OVERVIEW SMA IS A SEVERE NEUROLOGICAL DISORDER [1] Autosomal recessive genetic inheritance 1 in 50 people (approximately 6 million Americans) are carriers [2] 1 in 6,000 to 1 in 10,000 children born
More informationCoughAssist E70. More than just a comfortable cough. Flexible therapy that brings more comfort to your patients airway clearance
CoughAssist E70 More than just a comfortable cough Flexible therapy that brings more comfort to your patients airway clearance Flexible, customisable loosening and clearing therapy An effective cough is
More informationCredential Maintenance Program
First Quarter of the Calendar 5 I. COMPETENCIES SHARED BETWEEN CRITICAL AND GENERAL CARE 3 7 0 A. Assess Patient Information 0 2 2. Patient history, for example, immunizations environmental pre-existing
More informationDr. CK NG Department of Medicine Queen Elizabeth Hospital Kowloon Central Cluster
Ng CK, O WH, Lit MPK, Lee KH, Chan HF, Chan YC, Cheung YF, Hui YT, Chu S, Chong HM, Yu David, Chan JHM and Chan JWM Dr. CK NG Department of Medicine Queen Elizabeth Hospital Kowloon Central Cluster Neuromuscular
More informationCorporate Medical Policy
Corporate Medical Policy File Name: Origination: Last CAP Review: Next CAP Review: Last Review: nusinersen_spinraza 03/2017 10/2017 10/2018 10/2017 Description of Procedure or Service Spinal muscular atrophy
More informationHome Mechanical Ventilation
The International Convention Centre (ICC), Birmingham 11 12 September 2017 Home Mechanical Ventilation Martin Latham Nurse Specialist in Sleep Disordered Breathing St James s University Hospital Leeds
More informationDAYTON CHILDREN S HOSPITAL CLINICAL PRACTICE GUIDELINES
DAYTON CHILDREN S HOSPITAL CLINICAL PRACTICE GUIDELINES DISCLAIMER: This Clinical Practice Guideline (CPG) generally describes a recommended course of treatment for patients with the identified health
More informationThe difference is clear. CoughAssist clears airways with the force of a natural cough
The difference is clear CoughAssist clears airways with the force of a natural cough When only a real cough will do CoughAssist is a noninvasive therapy that safely and consistently removes secretions
More informationBiLevel Pressure Device
PROCEDURE - Page 1 of 7 Purpose Scope Classes/ Goals Define indications and care settings for acute and chronic initiation of Noninvasive Positive Pressure Ventilation. Identify the role of Respiratory
More informationOxygenation Failure. Increase FiO2. Titrate end-expiratory pressure. Adjust duty cycle to increase MAP. Patient Positioning. Inhaled Vasodilators
Oxygenation Failure Increase FiO2 Titrate end-expiratory pressure Adjust duty cycle to increase MAP Patient Positioning Inhaled Vasodilators Extracorporeal Circulation ARDS Radiology Increasing Intensity
More informationNeonatal/Pediatric Specialty Examination Detailed Content Outline
I. COMPETENCIES SHARED BETWEEN CRITICAL AND GENERAL CARE 10 32 17 59 A. Assess Patient Information 1 5 7 13 1. Patient history, for example, immunizations environmental pre-existing conditions 2. Physical
More informationProvide guidelines for the management of mechanical ventilation in infants <34 weeks gestation.
Page 1 of 5 PURPOSE: Provide guidelines for the management of mechanical ventilation in infants
More informationOptimize vent weaning and SBT outcomes. Identify underlying causes for SBT failures. Role SBT and weaning protocol have in respiratory care
Optimize vent weaning and SBT outcomes Identify underlying causes for SBT failures Role SBT and weaning protocol have in respiratory care Lower risk of developing complications Lower risk of VAP, other
More informationNAVA. In Neonates. Howard Stein, M.D. Director Neonatology. Neurally Adjusted Ventilatory Assist. Toledo Children s Hospital Toledo, Ohio
NAVA Neurally Adjusted Ventilatory Assist In Neonates Howard Stein, M.D. Director Neonatology Toledo Children s Hospital Toledo, Ohio Disclaimers Dr Stein: Is discussing products made by Maquet Has no
More informationNon Invasive Ventilation In Preterm Infants. Manuel Sanchez Luna Hospital General Universitario Gregorio Marañón Complutense University Madrid
Non Invasive Ventilation In Preterm Infants Manuel Sanchez Luna Hospital General Universitario Gregorio Marañón Complutense University Madrid Summary Noninvasive ventilation begings in the delivery room
More information