A lung transplant can be lifesaving for children with cystic fibrosis (CF) admitted for respiratory failure to a pediatric intensive care unit (PICU). Only 35% of these young patients went on to be discharged without a transplant, according to an analysis of children with CF treated at a French hospital.
Over the years following discharge, a lung transplant also associated with the best odds of survival for these patients. Factors influencing a risk of death or likely transplant outcomes included female sex and disease severity.
Children with CF who need critical care because of endstage respiratory failure need to be transplanted in [a] short-term period, the researchers wrote, calling a transplant their main chance to survive after leaving a hospital.
The study, Acute respiratory failure due to pulmonary exacerbation in children with cystic fibrosis admitted in a pediatric intensive care unit: outcomes and factors associated with mortality, was published in Respiratory Research.
The buildup with CF of thick and sticky mucus in the lungs causes inflammation and damage, leading to disease symptoms like wheezing, shortness or breath, or cough. Should the lungs become severely compromised, patients can go into acute respiratory failure and need to be hospitalized.
With the emergence of more effective disease treatments and better overall care, mortality rates for adult CF patients hospitalized for acute respiratory failure have significantly declined in recent decades.
Outcomes among young CF patients in a similar situation, however, have been poorly studied, the scientists wrote.
Paradoxically, short and longer-term outcomes at discharge from PICU have not been reported, they added.
A team at Hpital Necker Enfants Malades in Paris explored outcomes among 29 children with CF, ages 6-17 (median age of 13.5), admitted to its PICU due to acute respiratory failure between 2000 and 2020.
These patients had severe respiratory disease at hospital admission, and about three-quarters received nutritional support through a feeding tube and required at-home ventilation support. Nearly half (48%) were on a waiting list for a lung transplant.
All needed oxygen therapy in the hospital, with 92% being given non-invasive ventilation (i.e., an oxygen mask) and 10.3% invasive ventilation (i.e., a breathing tube). None were using CFTR modulator therapies, either because these effective treatments werent yet available or the childs disease-causing mutation wasnt eligible for their use.
Overall, five children (17%) died while in the ICU of respiratory failure, 14 (48%) received a lung transplant, and 10 (35%) were discharged without a transplant. Of those 10, three required a transplant within three years.
Mortality rates for the entire group were 31% at three months of follow-up, 34% at one year, and 42% at three years. Seventeen of the 29 children (58%) were alive at three years of follow-up, 12 (70%) of whom underwent a lung transplant. Of a total of 17 patients given a transplant, five died within three years of that surgery.
Most importantly, our results show very low rates of survival without lung transplantation during the 36 months following PICU admission for children with chronic respiratory insufficiency, the scientists wrote.
Lung transplantation should therefore be considered early after PICU admission and children referred to lung transplant centers immediately after their discharge, they added.
Statistical analyses uncovered risk factors associated with mortality or the need for a lung transplant. At the time of hospital admission, female sex, and higher carbon dioxide and lower chloride levels associated with a greater likelihood of poorer outcomes.
The probability of death was significantly increased in females, the scientists wrote.
A need for respiratory or nutritional support at the home, prior registration on a lung transplant list, or a lung infection with Stenotrophomonas Maltophilia bacteria prior to hospitalization also linked with a mortality or transplant risk.
Each of these factors could be considered markers of more severe underlying diseases, highlighting the importance of longitudinal disease control and proactive early care, they noted.
Study limitations include a retrospective design that precludes conclusions regarding causality between the factors identified and the risk of mortality or lung transplantation, the scientists wrote.
They added that study findings need to be confirmed in children at other centers, as their hospital typically treats severely ill patients.
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Lung transplant best chance for severely ill pediatric CF... - Cystic Fibrosis News Today
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