The document discusses amyotrophic lateral sclerosis (ALS) and focuses on respiratory assessment and ventilatory management of patients with ALS. It highlights the importance of a multidisciplinary team approach and early systematic respiratory evaluation to improve outcomes. Prognosis is difficult to predict for individual patients but median survival time is around 3-5 years from time of diagnosis.

1. Neuromuscular disorders: R espiratory assessment and ventilatory management . In patients with Amiotrophic Lateral Sclerosis Joan Escarrabill MD Master Plan of Respiratory Diseases (PDMAR) Institut d’Estudis de la Salut Barcelona [email_address] Stressa, April 4th 2009

3. ALS: Prognosis D ifficult to predict in an individual patient 3-4 yrs 50% > 5 yrs 20% > 10 yrs 10% > 20 yrs Occasional L ife expectancy

4. ALS: 3 key words Precocious Team Package

5. ALS: 3 key words Precocious Team Package

8. ALS: Natural History End of life Diagnosis Symptoms Non Invasive Ventilation

9. ALS: Natural History End of life Diagnosis Symptoms Early indication of NIV Natural History

10. ALS: Natural History End of life Diagnosis Symptoms Natural History Palliative NIV

11. ALS: Natural History End of life Diagnosis Symptoms Natural History Palliative NIV Early indication of NIV

12. Chest 2007;127:2132-8 Early systematic respiratory evaluation is necessary to improve the results o f HMV in ALS Survival in patients without bulbar involvement Protocol Pre-Protocol

13. M uscle strength vs Vital capacity M. Estenne, 1991

14. Lung function VC (lying, and sitting/standing) 20% Sniff-Nasal Inspiratory force Nocturnal pulsioxymetry

15. Eur Respir J 2008; 31: 93–98 M any patients with neuromuscular disease find the PImax manoeuvre difficult to perform -70 cmH2O in males or ,-60 cmH2O in females is unlikely to be associated with inspiratory muscle weakness Sniff-Nasal Inspiratory force (SNIP)

16. Am J Respir Crit Care Med 2005; 171 : 269–274 Kaplan-Meier survival curves based on the SNIF categories

17. Lung & bulbar function: Signs and symptoms Weight loss > 10% Dysarthria Dysphagia Syalorrhea Aspiration I ntolerance to supine position I neffective cough Lung function Bulbar function

18. ALS: 3 key words Precocious Team Package

20. The team produces more than the individual contributions of members.

21. Patient care team Wagner. BMJ 2000;320:569-72. R. Casas & P Romeu (1897)

23. Mitsumoto H & Rabkin JG. JAMA. 2007;298:207-216 C are in multidisciplinary clinics is associated with enhanced quality of life by alleviating symptoms and may extend survival

24. J Neurol Neurosurg Psychiatry 2006;77:948-50 Tertiary center Neurology clinic 1080 days 775 days The median survival from onset was 10 months longer in ALS centers 4 yrs younger PEG & NIV more often Less hospital admissions

25. www.has-sante.fr/ 2003 17 Reference centers Coodination Working groups Local organization

26. Evalutaion of ALS reference centers

27. Survival of Irish ALS patients One year mortality was decreased by 29.7% Prognosis of bulbar onset patients was extended by 9.6 months

28. ALS patients who received their care at a multidisciplinary clinic had a better prognosis Recruitment bias ALS clinic treated a group of fitter ALS patients General neurologists saw all ALS patients Living further from ALS clinic More disabled Increased age Bulbar onset Shorter duration of illness Hutchinson M. J Neurol Neurosurg Psychiatry 2004;75:1208-12

29. Effect of referral bias Sorenson EJ et al. N eurology 2007;68:600-602 132 subjects Tertiary center 3 years. Survival p = 0.007 18 months 29 months local population referral population

30. Zoccolella S et al. J Neurol 2007;254:1107-12 No improvements in survival: Low rate of interventions?

31. Home care organized through reference centers has many limitations Complex organization, D istance, R esponse to emergenc ies In most cases the reference center coordinates care but it can not assume direct care

32. Bias in the care of patients Accessibility Low rate of interventions Distance Referral Bias

34. Conclusions: 3 key words Precocious Team Package

36. Lancet Neurol 2006;5:140-7

37. Are NIV trials necessaries in ALS with non-bulbar impairement? Servera E. Sancho S. Lancet Neurol 2006;5:140-7 Non-bulbar patients in control group Stop studies according the results Assessment efficacy of NIV Pressure vs volume ventilators Secretion management It’s mandatory to evaluate therapy “package” Ethical issues Technical issues

38. “ Therapy package” in ALS Mobility Swallowing & Speaking Coping with changes Breathing changes Symptoms Caregiver Adapted from www.alsa.org/

40. Chest 2000;118:1390-6

42. Chest 2004;125:1400-5 PCF MIC > 4 L/s MI-E not generate greater PCF than manually assisted coughing PCF MIC < 2.7 L/s Dynamic collapse of the upper airway during the exsufflation

43. Lung function Sancho J. Chest 2004;125:1400-5

45. www.patientslikeme.com/

46. Sweden 1965 - 2004 6642 patients 40 years 3 years

47. Mitsumoto H & Rabkin JG. JAMA. 2007;298:207-216

48. ALS: 3 key words Precocious Team Package Especially in regard to lung function Respiratory & Non-respiratory Since the beginning of the disease