CPAP for Hypoxemic Acute Chest Syndrome in Sickle Cell Disease

Trial statusNot yet recruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age18+
SponsorAssistance Publique - Hôpitaux de Paris

About this trial

Sickle cell disease (SCD) is a severe hemoglobinopathy, considered the first monogenic disease in the world. Acute chest syndrome (ACS), one of the most frequent and serious complications of SCD, is defined by the association of fever and/or acute respiratory symptoms with a new pulmonary infiltrate on chest imaging. ACS is characterized by lung consolidation, severe pulmonary vascular dysfunction, with potential role for regional alveolar hypoxia. Therefore, improving alveolar oxygenation and limiting lung consolidation are key objectives of the treatment of ACS, in addition to ensuring pain relief and giving blood transfusions and antibiotics. Bilevel non-invasive ventilation failed in improving outcomes during ACS (Fartoukh 2010). These results are in accordance with those reported in other forms of acute lung injury (Frat 2015), with conflicting results. Among other explanations, NIV may favour high tidal volume ventilation leading to patient self-inflicted lung injury (P-SILI) (Carteaux 2016). Continuous positive airway pressure (CPAP) is a simple to use and affordable technique for non-invasive ventilatory support, that theoretically exposes to a lower risk of P-SILI (Carteaux 2021). In patients with acute hypoxemic respiratory failure (AHRF), applying a positive pressure to the airway opening has been shown to mitigate the reduction in functional residual capacity and to improve respiratory mechanics and gas exchange. In a randomized controlled trial (RCT) conducted in patients with AHRF, CPAP achieved early physiologic improvement (Delclaux 2000). Recent results also suggest that CPAP reduces the composite outcome of intubation or death in adults with AHRF due to COVID-19 in a large multicentre study (RECOVERY-R) (Perkins 2022). In addition, CPAP can be safely used at early stages in the wards, with a frugal approach, using virtual valves (Carteaux 2021). In patients with SCD, CPAP has shown benefits when used at night in children with sleep apnea (Marshall 2009), or for the peri-operative management (Leff 2007). CPAP is also used in clinical practice for hypoxemic ACS (Heilbronner 2021), but it has not been formally assessed in this setting.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

SCD patient of all genotypes (SS, SC, S/β0 and S/β+)

Age ≥ 18 years old

Hospitalised for ACS (defined as the association of fever and/or acute respiratory symptoms with a new pulmonary infiltrate on chest imaging)

Requiring supplemental O2 ≥ 2 L/min for SpO2 ≥ 95%

Disqualifiers

Patient having both ACS criteria and need for supplemental O2 ≥ 2 L/min for SpO2 ≥ 95% since more than 48 hours

Requirement for home supplemental O2 or home CPAP / NIV.

Signs of worsening respiratory failure mandating intubation (as defined in (Helms et al., 2024))

Current enrolment in another interventional research concerning a respiratory support during ACS

Trial design

Design model

Parallel

Treatments tested in this trial

  • O2+CPAP group

    Other intervention

    ACS episodes assigned to this group will receive supplemental O2 in addition to periods of CPAP. CPAP will target a positive pressure between 5 and 10 cmH2O. CPAP will be given discontinuously (≥6 hours/day) based on patient tolerance . CPAP sessions will be stopped when the patient achieves the criteria for cessation of supplemental O2. These criteria will be the same as in the O2 group. No sedation will be used for CPAP tolerance.

Treatment groups

140 Participants
are divided into 2 treatment groups
Group A: O2+CPAP groupExperimental treatment 1 intervention
Group B: O2 groupNo intervention 0 interventions

Trial outcomes

Primary outcomes

1

Time to résolution of acute chest syndrome (ACS)

time to resolution of ACS, defined as the time from randomization to the joint resolution of fever (body temperature \< 38°C), chest pain (visual analog scale, VAS ≤ 3 cm, morphine ≤ 40mg/24h), dyspnea (VAS ≤ 3 cm, respiratory rate \< 25/min, no ventilatory support), and hypoxemia (SpO2 \> 92% on room air) (Mekontso Dessap, Habibi, et al., 2025). If a VAS is unavailable, a verbal rating scale will be used. Resolution of ACS will be assessed every 8 to 12 hours and will be considered achieved if sustained across 2 to 3 consecutive evaluations (i.e., over a 24-hour period).

Time frame
Up to randomization

Secondary outcomes

1

Mortality

All-cause mortality

Time frame
up to hospital discharge or day-28 after randomisation (whichever occurs first), and at 3 months after randomisation.
2

Length of hospital stay

Time frame
up to hospital discharge or day-28 after randomisation (whichever occurs first), and at 3 months after randomisation.
3

Length of ICU stay

Time frame
up to hospital discharge or day-28 after randomisation (whichever occurs first), and at 3 months after randomisation.
4

Need for catecholamine infusion

dobutamine, dopamine, adrenaline or noradrenaline

Time frame
From randomisation to discharge or Day-28

Other outcomes

Locations

This trial has no locations.

Sponsors and contacts

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