Non Invasive Positive Pressure Ventilation for Prevention of Acute Chest Syndrome in Sickle Cell Disease (VPACS)

This study has been terminated.
(decision of the promoter)
Sponsor:
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier:
NCT01501643
First received: December 26, 2011
Last updated: June 4, 2013
Last verified: May 2013
  Purpose

Acute chest syndrome is a severe respiratory complication of sickle cell disease.

The standard prevention of this dangerous complication is spirometry in wich patient is required to take deep breaths trough a little device several times a day. This treatment is compromised when pain in important or when the patient is asleep and cannot participate.

The investigators hypothesised that non invasive ventilation in wich patient have no effort to take might be a better prevention than spirometry and may improve pain and quality of sleep.

Children with vaso-occlusive crisis necessitating morphinic treatment will be randomly assigned with either spirometry or ventilation and the investigators will monitor for occurrence of acute chest syndrome, pain and quality of sleep.


Condition Intervention
Sickle Cell-hemoglobin SS Disease
Vaso-occlusive Crisis
Device: Non invasive positive pressure ventilation
Device: Spirometry

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Prevention
Official Title: Non Invasive Positive Pressure Ventilation for Prevention of Acute Chest Syndrome in Sickle Cell Disease With Vaso-occlusive Crisis and Morphinic Treatment

Resource links provided by NLM:


Further study details as provided by Assistance Publique - Hôpitaux de Paris:

Primary Outcome Measures:
  • Occurence of acute chest syndrome [ Time Frame: up to 2 months at maximum (duration of hospitalization) ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Pain [ Time Frame: up to 2 months at maximum (duration of hospitalization) ] [ Designated as safety issue: No ]
  • Morphinic requirements [ Time Frame: up to 2 months at maximum (duration of hospitalization) ] [ Designated as safety issue: No ]
  • Length of hospital stay [ Time Frame: up to 2 months at maximum (duration of hospitalization) ] [ Designated as safety issue: No ]
  • Comfort [ Time Frame: up to 2 months at maximum (duration of hospitalization) ] [ Designated as safety issue: No ]
  • Quality of sleep [ Time Frame: up to 2 months at maximum (duration of hospitalization) ] [ Designated as safety issue: No ]

Estimated Enrollment: 120
Study Start Date: November 2011
Estimated Study Completion Date: May 2015
Estimated Primary Completion Date: November 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Spirometry
Spirometry
Device: Spirometry
Every two hours during the day with 10 maximum inspirations and at night if the patient is awake
Experimental: Non invasive positive pressure ventilation
Non invasive positive pressure ventilation
Device: Non invasive positive pressure ventilation
At least one hour every four hour but may be maintained more if the patient chooses to or is asleep with the machine on.

Detailed Description:

Acute chest syndrome is a severe complication of sickle cell disease. Several factors can contribute to this complication including infections, vaso-occlusive crisis, important pain that may give shortness of breath and morphinic treatments as they may cause hypoventilation.

The standard prevention of this dangerous complication is incentive spirometry in wich patient is required to take deep breaths trough a little device several times a day. This treatment is compromised when pain in important or when the patient is asleep and cannot participate.

In patients who have had surgery who have also pain and morphinic treatments, non invasive ventilation have proven a good option for preventing pulmonary complications.

Besides, our experience in non invasive ventilation for treatment of acute chest syndrome show a good tolerance and efficiency. It also seems to help with pain management.

The investigators hypothesised that non invasive ventilation in wich patient have no effort to take might be a better prevention than incentive spirometry and may improve pain and quality of sleep.

After informed consent, patients aged from 6 to 20 years old with SS or S-Beta sickle cell disease with vaso-occlusive crisis necessitating morphinic treatment hospitalised in intensive care unit in Necker Hospital for Sick Children (Paris) will be randomly assigned with either spirometry or ventilation. It is a monocentric study.

Patients with acute chest syndrome, oxygen needs, pneumonia or other condition requiring ventilation at presentation will not be included.

Spirometry will be realised through volumetric devices, every two hours during the day with 10 maximum inspirations and at night if the patient is awake.

Non invasive ventilation will be applied through a nasal mask with VS III machines from Resmed®, at least one hour every four hour but may be maintained more if the patient chooses to or is asleep with the machine on.

The investigators will monitor for occurrence of acute chest syndrome, pain, morphinic requirements, length of hospital stay, comfort, quality of sleep The treatment will be continued until morphinic treatment is stopped. The investigators intend to include 60 patients in each arm to show a difference of 25% in the occurrence of acute chest syndrome (primary judgement criteria).

Acute chest syndrome will be defined according to the criteria used by Vichinsky (Vichinsky, E. P. et al. Acute chest syndrome in sickle cell disease: clinical presentation and course. Cooperative Study of Sickle Cell Disease. Blood 89, 1787-1792 (1997) The investigators will aslo compare morphinic requirements, length of hospital stay and quality of sleep as secondary judgment criteria.

  Eligibility

Ages Eligible for Study:   6 Years to 20 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patient with SS or S-Beta sickle cell disease aged more than 6 years old and less then 20 years old
  • Hospitalised since less then 24 hours for severe vaso-occlusive crisis (either peripheric localisation or thoracic crisis) with morphinic requirements according to local pain management protocol
  • With a signed informed consent from the patient or his legal representative if the patient is under 18 years old
  • Benefiting of the French social security system

Exclusion Criteria:

  • Patient that has already participated in the study
  • Oxygen requirements, pneumonia, acute chest syndrome or other condition necessitating non invasive ventilation on admission
  • No understanding of spirometry or ventilation techniques
  • Other condition that forbid the use of non invasive ventilation or spirometry as defined by french consensus conference (2006) on non invasive ventilation
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01501643

Locations
France
Hôpital Necker - Enfants Malades Hospital
Paris, France, 75015
Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
Investigators
Principal Investigator: Claire Heilbronner, MD, PhD Necker - Enfants Malades Hospital
  More Information

No publications provided

Responsible Party: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT01501643     History of Changes
Other Study ID Numbers: P091114
Study First Received: December 26, 2011
Last Updated: June 4, 2013
Health Authority: France: Ministry of Health

Keywords provided by Assistance Publique - Hôpitaux de Paris:
Non invasive positive pressure ventilation
Spirometry
Acute chest syndrome
Sickle cell disease
Vaso-occlusive crisis

Additional relevant MeSH terms:
Acute Chest Syndrome
Anemia, Sickle Cell
Syndrome
Anemia
Anemia, Hemolytic
Anemia, Hemolytic, Congenital
Disease
Genetic Diseases, Inborn
Hematologic Diseases
Hemoglobinopathies
Lung Diseases
Pathologic Processes
Respiration Disorders
Respiratory Tract Diseases

ClinicalTrials.gov processed this record on October 30, 2014