A survey of cystic fibrosis physicians' views on lung transplant referral in the era of elexacaftor/tezacaftor/ivacaftor

Pediatr Pulmonol. 2024 Dec;59(12):3641-3649. doi: 10.1002/ppul.27273. Epub 2024 Sep 18.

Abstract

Rationale: In 2015, a survey of cystic fibrosis (CF) physicians showed significant gaps in lung transplant (LTx) referral knowledge. Subsequently, LTx referral guidelines for people with CF were published, and elexacaftor/tezacaftor/ivacaftor (ETI) became available for >80% of people in the United States (US). We sought to assess physicians' LTx referral knowledge and self-reported referral practices.

Methods: CF center directors in the US were surveyed about LTx. Questions addressed transplant referral indications, contraindications, testing, and the impact of ETI on referral timing. Thematic analysis was used to assess responses to open-ended questions.

Results: There were 110/309 (36%) responses. Respondents identified several referral indications, including rapid decline in FEV1 (93%), recurrent hemoptysis (80%), hypoxemia (79%), and pulmonary hypertension (75%). Over 70% of respondents reported using oximetry, echocardiogram, and blood gas to assess disease severity. Respondents were more likely to find early LTx discussions useful for patients not on modulators versus on modulators (87% vs. 63%, p < .005). Most respondents (66%) reported delaying LTx referral for some patients with FEV1 30%-40% who met criteria, while 26% had delayed referral for patients with FEV1 < 30%. Uncertainty regarding optimal LTx referral timing for patients on ETI was a prominent theme of the qualitative analysis.

Conclusions: While physician knowledge about LTx referral indications appears improved since the CF referral guidelines were published, uncertainty about referral timing is pervasive, and the guidelines will need to be updated as more data become available about the long-term effectiveness of ETI in advanced lung disease.

Keywords: cystic fibrosis; elexacaftor/tezacaftor/ivacaftor; lung transplantation; physician survey; referral.

MeSH terms

  • Aminophenols* / therapeutic use
  • Attitude of Health Personnel
  • Benzodioxoles* / therapeutic use
  • Cystic Fibrosis* / drug therapy
  • Cystic Fibrosis* / surgery
  • Drug Combinations*
  • Female
  • Humans
  • Indoles* / therapeutic use
  • Lung Transplantation*
  • Male
  • Physicians / statistics & numerical data
  • Practice Patterns, Physicians' / statistics & numerical data
  • Pyrazoles* / therapeutic use
  • Pyridines* / therapeutic use
  • Pyrrolidines / therapeutic use
  • Quinolines
  • Quinolones* / therapeutic use
  • Referral and Consultation* / statistics & numerical data
  • Surveys and Questionnaires
  • United States

Substances

  • Benzodioxoles
  • Drug Combinations
  • Pyridines
  • Pyrazoles
  • Indoles
  • Aminophenols
  • Quinolones
  • Pyrrolidines
  • elexacaftor, ivacaftor, tezacaftor drug combination
  • Quinolines