Correction: Dagenais et al. Real-World Safety of CFTR Modulators in the Treatment of Cystic Fibrosis: A Systematic Review. J. Clin. Med. 2021, 10, 23

In the original article [1], there was a mistake in Table 1 as published. Reference citation [34] was wrong. The corrected

Table 1. Summary of characteristics and results of cohort or survey studies with full manuscript.

Ref	Study Design & Location	Population a	n	Recruitment Period & Follow-Up Duration	Overall Adverse Events (AE) b,c			Dose Modification, Interruption, or Discontinuation Due to AE b,c
Ivacaftor
[29]	Prospective Cohort d   United States	Baseline Age Pediatric and Adult - Mean: 33 yr - Range: 10–61 yr   CFTR Genotype ≥1 copy G551D   Baseline ppFEV1 Mean: 30%	44	Recruitment Period Prior to commercial availability   Follow-Up Duration NS	AE in n = 38 (86%):	n	%	Discontinuation:	n	%
					- Pulmonary exacerbation - Hemoptysis - Increased sputum - Increased cough - URTI - Dyspnea - Abnormal respiration - Respiratory tract congestion - Headache - Rash	20 7 7 6 6 3 3 3 5 4	45 16 16 14 14 7 7 7 11 9	- Severe abdominal pain - Dizziness/tinnitus	1 1	2 2
					SAE in n = 14 (32%):
					- Pulmonary exacerbation - Hemoptysis - Pneumothorax - Acute respiratory failure - URTI - Abdominal pain - Gastroenteritis - Abnormal LFTs - Syncope - Secondary adrenocortical insufficiency	NS NS NS NS NS NS NS NS NS NS	- - - - - - - - - -
[55]	Prospective Cohort   United States Canada Italy	Baseline Age Pediatric and Adult - Mean: 17 yr - Range: 5–61 yr   CFTR Genotype ≥1 gating mutation   Baseline ppFEV1 Mean: 86%	23	Recruitment Period   Mar 2014 to Aug 2015   Follow-Up Duration 3 mo	49 AE in n = 21 (91%):	n	%	None reported
					- Respiratory, unspecified - Gastrointestinal, unspecified - Infection, unspecified - Headache - Weakness - Dizziness - Fatigue	NS NS NS NS NS NS NS	- - - - - - -
					5 SAE in n = 3 (13%):	n	%
					- Respiratory infection - Acute changes in metabolic and liver status	4 1	17 4
[56]	Retrospective Cohort   United Kingdom (1 center)	Baseline Age Pediatric - Mean: 9 yr - Range: 6–14 yr   CFTR Genotype 1 copy G551D   Baseline ppFEV1 Mean: 68% e	4	Recruitment Period Jan 2013 to Jun 2015   Follow-Up Duration Mean: 24 mo	- Transaminitis (<3 x ULN)	n 1	% 25	None reported
[57]	Retrospective Cohort   Scotland (11 centers)	Baseline Age Pediatric - Median: 9 yr   CFTR Genotype ≥1 copy G551D   Baseline ppFEV1 Mean: 85%	26	Recruitment Period NS (Jan 2013 to Mar 2013 for 85%)   Follow-Up Duration Mean: 17 mo		n	%	None reported
					- Headache - Swollen ear - Cataracts	1 1 2	4 4 17 f
[58]	Retrospective Cohort   France (25 centers)	Baseline Age Pediatric and Adult - Median: 18 yr - Range: 6–52 yr   CFTR Genotype ≥1 copy G551D   Baseline ppFEV1 Mean: 72%	57	Recruitment Period Pre-1 Jun 2013 up to 30 Sep 2014   Follow-Up Duration Up to 2 yr	34 AE in n = 21 (37%):	n	%	Interruption in n = 7 (12%):	n	%
					- Transaminitis - Rhinopharyngitis - Asthma - Fever - Chest pain - Abdominal pain - Nausea or vomiting - Intestinal dysmotility - Headache - Fatigue - Rash or eczema - Depression - Myalgia - Arthritis - Breast hypertrophy - Orchitis - Atrial fibrillation	3 NS NS NS NS NS NS NS NS NS NS NS NS NS NS NS NS	5 - - - - - - - - - - - - - - - -	- Hepatitis - Rhinopharyngitis - Abdominal pain - Vomiting - Headache - Rash - Severe depression	NS NS NS NS NS NS NS	- - - - - - -
								Discontinuation:
								- Transaminitis - Liver cirrhosis diagnosis	1 1	2 2
[30]	Retrospective Cohort d   Germany (multicenter)	Baseline Age Adult - Mean: 34 yr   CFTR Genotype ≥1 copy G551D   Baseline ppFEV1 Mean: 25%	14	Recruitment Period Sep 2012 to Apr 2013   Follow-Up Duration Mean: 235 days	- Increased bronchial and nasal secretions - Headache - Worsening RLS - Abdominal pain - Hyperbilirubinemia (mild) - Transaminitis (<3 to 4x ULN)	n 3 1 1 1 1 1	% 21 7 7 7 7 7	Discontinuation: - Increased bronchial and nasal secretions *   * Trial of reduced dose before discontinuation	n 1	% 7
Lumacaftor/Ivacaftor
[41]	Prospective Cohort   France (1 center)	Baseline Age Pediatric - Mean: 16 yr   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean: 87%	32	Recruitment Period Mar 2016 to Dec 2016   Follow-Up Duration 4 h post-first dose	- Acute drop in ppFEV1 - Wheeze	n 32 3	% 100 9	None reported
[42]	Prospective Cohort   France (47 centers)	Baseline Age Pediatric and Adult - Mean: 22 yr   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean: 65%	845	Recruitment Period 1 Jan 2016 to 31 Dec 2016   Follow-Up Duration 12 mo	AE in n = 494 (59%):	n	%	Interruption:	n	%
					- Respiratory - Digestive - Menstrual abnormality - Fatigue - Headache - CK > 5xULN - Transaminitis (> 3xULN)	316 181 53 37 19 20 5	37 21 6 4 2 2 0.6	- Respiratory - ‘Non-respiratory’   Discontinuation: Respiratory - Chest tightness/dyspnea - Bronchospasm - Increased cough/sputum - Hemoptysis - Pneumothorax   Non-respiratory - Diarrhea, abdominal pain - CK >10xULN + myalgia - Fatigue - Headache - Depression - Metrorrhagia - Transaminitis (>6xULN) - Cutaneous rash - Tachycardia	16 8   n  38 24 9 2 1     18 5 5 4 4 3 2 1 1	2 1   %   5 3 1 0.2 0.1     2 0.6 0.6 0.5 0.5 0.4 0.2 0.1 0.1
[59]	Prospective Cohort   United States (1 center)	Baseline Age Pediatric and Adult - Mean: 23 yr - Range: 12–48 yr   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean: 70%	26	Recruitment Period NS   Follow-Up Duration 6 mo	See Discontinuation			Discontinuation: - Transaminitis - Unspecified AE	n 1 4	% 4 15
[31]	Prospective Cohort d   Switzerland (1 center)	Baseline Age Adult - Mean NR   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Median: 30%	20	Recruitment Period Jan 2016 to Jan 2017   Follow-Up Duration 1 mo		n	%	Reduced dose:	n	%
					- Dyspnea  - 3 h  - 24 h  - 1 mo - Chest tightness  - 3 h  - 24 h  - 1 mo - Increased sputum  - 3 h  - 24 h  - 1 mo - Pulmonary exacerbation  - 1 mo	0 1 1   1 10 1   1 8 3   2	- 5 5   5 50 5   5 40 15   10	- Respiratory intolerance   Discontinuation: - Chest tightness (at 24 h)	3     1	15     5
[32]	Prospective Cohort   Australia (1 center)	Baseline Age Adult - Mean: 27 yr   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Median: 36%	12	Recruitment Period Jan 2016 to Oct 2016   Follow-Up Duration 1 mo		n	%	Discontinuation:	n	%
					- Acute drop in ppFEV1 - Respiratory AE overall  - 4 h  - 24 h  - 1 mo - Dyspnea  - 4 h  - 24 h  - 1 mo - Chest tightness  - 4 h  - 24 h  - 1 mo - Increased sputum  - 4 h  - 24 h  - 1 mo - Pulmonary exacerbation	12   5 10 8   2 6 7   4 8 5   0 2 1 6	100   42 83 67   17 50 58   33 67 42   - 17 8 50	- Chest tightness/dyspnea *   * n = 2 discontinued after 1mo follow-up (5 wk and 9 wk)	3	25
[33]	Prospective Cohort   France (11 centers)	Baseline Age Adult - Mean: 31 yr   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean: 32%	53	Recruitment Period Jan 2016 to Jun 2016   Follow-Up Duration 3 mo	AE in n = 34 (63%):	n	%	Discontinuation:	n	%
					- Abnormal respiration - Dyspnea - Increased cough - Abdominal pain, nausea, diarrhea, or vomiting - Fatigue - Rash - Pruritus - Breast tension	13 11 3 9   2 1 1 1	25 21 6 17   4 2 2 2	- Respiratory intolerance - Vomiting - Fatigue	13 1 1	25 2 2
[25]	Prospective Cohort d,g   Australia (1 center)	Baseline Age Adult - Mean: 27 yr   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean: 36%	10	Recruitment Period NS   Follow-Up Duration 52 wk	AE in n = 6 (60%):	n	%	None reported
					- Chest tightness/dyspnea - Headache	6 2	60 20
[43]	Retrospective Cohort Ireland   (1 center)	Baseline Age Pediatric - Mean: 14 yr   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean: 77%	15	Recruitment Period Sep 2016 to Aug 2017   Follow-Up Duration NS		n	%	None reported
					- Acute drop in ppFEV1 - Chest tightness - Increased sputum	14 2 2	93 13 13
[44]	Retrospective Cohort   United States (1 center)	Baseline Age Pediatric and Adult - Mean: 25 yr - Range: 12–59 yr   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean: 67%	116	Recruitment Period NS   Follow-Up Duration Up to 11 mo	AE in n = 46 (40%):	n	%	Reduced dose:	n	%
					- Chest tightness - Dyspnea - Increased cough - Diarrhea - Nausea - Decreased appetite - Rash	23 12 10 5 3 2 2	20 10 9 4 3 2 2	- AE not specified   Discontinuation: - Reasons not specified h	10     20	9     17
[60]	Retrospective Cohort   Greece (1 center)	Baseline Age Pediatric and Adult - Mean: 16 yr i - Range: 12–23 yr   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean 92% i	62	Recruitment Period Mar 2016 to Aug 2017   Follow-Up Duration 12 mo	- Chest tightness	n 2	% 3	Discontinuation: - Transaminitis - Cataract	n 1 1	% 2 2
[34]	Retrospective Cohort d   Spain (multicenter)	Baseline Age Pediatric and Adult - Mean: 27 yr - Range: 10–45 yr   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean: 32%	20	Recruitment Period 2016   Follow-Up Duration 6 mo	AE in n = 15 (75%):	n	%	Discontinuation:	n	%
					- Chest tightness - Dyspnea - Headache - Weight loss - ‘Sickness’ (not defined) - Asthenia - Abdominal pain - Transaminitis	9 8 5 5 3 3 2 2	45 40 25 25 15 15 10 10	- Decreased ppFEV1 - AE not specified	1 6	5 30
[45]	Retrospective Cohort   Canada (1 center)	Baseline Age Adult - Median: 32 yr   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Median: 40%	22	Recruitment Period Apr 2016 to Jun 2018   Follow-Up Duration Median: 10 mo	AE in n = 19 (86%):	n	%	Discontinuation:	n	%
					- Chest tightness - Wheeze - Dyspnea - Increased sputum - Increased cough - Flu-like symptoms - Elevated blood pressure - Headache - Nausea - Elevated AST - Anxiety - Bradycardia - Pleuritic chest pain	14 4 3 3 2 1 5 4 2 1 1 1 1	64 18 14 14 9 5 23 18 9 5 5 5 5	- Respiratory symptoms - Asymptomatic hypertension - Symptomatic hypertension  - Headache  - Hypertensive emergency - Anxiety	3 2   1 1 1	14 9   5 5 5
[61]	Retrospective Cohort   United States (1 center)	Baseline Age Adult - Mean NR   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean NR	82	Recruitment Period Jul 2015 to Jun 2016   Follow-Up Duration 12 mo	See Discontinuation			Discontinuation: Total overall: - Chest tightness * - Diarrhea ** - Abdominal pain - Nausea ** - Dysphagia - Elevated LFTs - Pericarditis - Allergic reaction ** - Suspected Stevens–Johnson syndrome   * n = 3 also had significant drop in ppFEV1 ** n = 1 also discontinued due to chest tightness	n 17 11 2 1 1 1 1 1 1 1	% 21 13 2 1 1 1 1 1 1 1
[26]	Retrospective Cohort d,g   Australia (7 centers)	Baseline Age Adult - Mean: 31 yr   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean: 37%	72	Recruitment Period Nov 2015 to Mar 2017   Follow-Up Duration 12 mo		n	%	Discontinuation:	n	%
					- Chest tightness/dyspnea - Increased sputum - Decrease in ppFEV1 - Headache - Fatigue - Nausea - Rash	40 4 2 2 5 1 2	56 6 3 3 7 1 3	- Chest tightness/dyspnea	22	31
[27]	Case Series (Survey) j   International (31 centers)	Baseline Age Adult - Mean: 30 yr   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean: 59%	26	Recruitment Period Questionnaire sent in 2018–2019   Follow-Up Duration NS		n	%	Discontinuation:	n	%
					- Pulmonary exacerbation - Post-partum acute myelocytic leukemia	1 1	4 4	- Chest tightness	2	8

^a When adult and pediatric patients both included, age range reported when possible; ^b Rates not reported for all AE, as indicated by ‘NS’; ^c To avoid redundancy, if AE only reported in context of dose modification, interruption, and/or discontinuation of therapy, it was not listed in overall AE; ^d Study population part of a compassionate, ‘expanded access’, ‘managed access’, or ‘named patient’ program; ^e Mean calculated from n = 3 (75%) of study subjects, as baseline not reported for n = 1 (25%); ^f Frequency of 17% based on n = 12 screened; 8% frequency for overall cohort of n = 26; ^g Study was case-control, but only LUM/IVA-treated participants included in systematic review; therefore, assessed as cohort study; ^h Reason for discontinuation was not consistently assessed, and may include reasons unrelated to AE; ⁱ Mean baseline age and ppFEV₁ based on n = 52 in final analysis of outcomes assessing effectiveness; n = 10 excluded from this analysis; ^j This case series is included in Table 1 due to results being presented in aggregate. AST, aspartate aminotransferase; CFTR, cystic fibrosis transmembrane conductance regulator; CK, creatine kinase; h, hour(s); LFT, liver function test; mo, month(s); NR, not reported; NS, not specified; ppFEV₁, percent predicted Forced Expiratory Volume in 1 sec; RLS, restless leg syndrome; SAE, serious adverse events; ULN, upper limit of normal; URTI, upper respiratory tract infection; wk, week(s); yr, year(s).

appears below. The authors state that the scientific conclusions are unaffected. The original article has been updated.

In the original article [1], there was a mistake in Table 2 as published. Subtitle “Lumacaftor/Ivacaftor” was wrong. The corrected

Table 2. Summary of characteristics and results of cohort or survey studies in abstract form.

Ref	Study Design	Population	n	Overall Adverse Events (AE) a,b			Dose Modification, Interruption, or Discontinuation Due to AE a,b
Ivacaftor
[62]	Prospective Cohort	Baseline Age Pediatric - Mean: 5 yr   CFTR Genotype ≥1 gating mutation   Baseline ppFEV1 Mean NR	4	AE in n = 2 (50%):	n	%	None reported
				- URTI - Nasal congestion - Headache	NS NS NS	- - -
[63]	Retrospective Cohort	Baseline Age Pediatric - Mean: 6 yr   CFTR Genotype ≥1 gating mutation   Baseline ppFEV1 Median: 87%	10	- Transient rash - Increased obesity	n 2 1	% 20 10	None reported
[64]	Prospective Cohort	Baseline Age Pediatric and Adult - Mean NR   CFTR Genotype ≥1 copy S549R   Baseline ppFEV1 Mean: 54%	15	- Liver enzyme derangement	n	%	None reported
					2	13
[65]	Cross-sectional Survey	Baseline Age Adult - Mean: 26 yr   CFTR Genotype ≥1G551D   Baseline ppFEV1 Mean: 62%	11 d	AE in n = 8 (73%) d: - Transient rash - Dizziness - Unspecified AE	n NS NS NS	% - - -	None reported
Lumacaftor/Ivacaftor
[66]	Prospective Cohort	Baseline Age Pediatric - Mean: 13 yr   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean: 91%	14		n	%	Reduced dose *:	n	%
				- Acute drop in ppFEV1 (asymptomatic) - Chest tightness, tachypnea (requiring oxygen)	1   1	7   7	- Chest tightness, tachypnea   * Eventual titration to full dose	1	7
[67]	Prospective Cohort	Baseline Age Pediatric - Mean: 14 yr   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean: 87%	13		n	%	None reported
				- Drop in ppFEV1 requiring salbutamol	7	54
[68]	Prospective Cohort	Baseline Age Pediatric and Adult - Mean: 23 yr e   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean 61% e	369		n	%	Discontinuation:	n	%
				- Bronchospasm - Dyspnea - Abnormal respiration - Unspecified respiratory AE - Unspecified AE	15 12 7 4 120	4 3 2 1 33	- Unspecified AE	16	4
[69]	Prospective Cohort	Baseline Age Pediatric and Adult - Mean: 25 yr   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean NR	311	379 AE in n = 213 (68%): - Dyspnea - Cough - GI discomfort (e.g., diarrhea, nausea, abdominal pain) - Headache - Fatigue - Unspecified	n f NS NS NS   NS NS NS	% 31 6 31   6 5 NR	Interruption (stop/restart): - Unspecified AE and other reasons g   Discontinuation: - Unspecified AE and other reasons g	n 12     42	% 4     14
[35]	Prospective Cohort c	Baseline Age Adult - Median: 31 yr   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Median: 28%	14		n	%	Discontinuation:	n	%
				- Chest tightness, breathless - Rash	7 1	50 7	- Respiratory AE and/or rash	4	29
[70]	Prospective Cohort	Baseline Age Adult - Mean NR   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean NR	29		n	%	Reduced dose:	n	%
				- Chest tightness *   * n = 4 cases severe, requiring hospitalization for IV steroids and antibiotics	13	45	- Chest tightness   Discontinuation: - Chest tightness	2     5	7     17
[36]	Prospective Cohort c	Baseline Age Mean NR h   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean NR	32	AE in 88%:	n f	%	Interruption (stop/restart):	n	%
				- Respiratory AE - Drop in ppFEV1	NS NS	87 -	- Unspecified AEA   Discontinuation: - Unspecified AE	1     8	3     25
[71]	Retrospective Cohort	Baseline Age Pediatric and Adult - Mean NR   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean NR	34	AE in n = 29 (85%): - Pulmonary exacerbation - Chest tightness - Dyspnea - Diarrhea - Abdominal pain	n 16 9 3 3 3	% 47 26 9 9 9	Discontinuation: - Unspecified AE	n 10	% 29
				Serious AE in n = 8 (24%):
				- Respiratory failure i - Unspecified AE	1 7	3 21
[72]	Retrospective Cohort	Baseline Age Pediatric and Adult - Mean: 26 yr   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean: 68%	103	See Discontinuation			Interruption/discontinuation j:	n	%
							- Chest tightness and/or pain - Elevated LFTs	17 NS	17 -
[73]	Retrospective Cohort	Baseline Age Adult - Mean: 31 yr   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean: 50%	71	AE in n = 41 (58%):	n	%	Discontinuation:	n	%
				- Chest tightness - Dyspnea - Increased cough - GI (pain, constipation, or diarrhea) - Rash - Pruritus - Irregular menses or metrorrhagia - Breast tension - Headache - Myalgia	22 8 4 6 4 1 3 2 1 1	31 11 6 9 6 1 4 3 1 1	- Dyspnea - Chest tightness - Increased cough - Fatigue	7 6 3 1	10 9 4 1
[74]	Retrospective Cohort	Baseline Age Mean NR h   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean NR	54	See Discontinuation			Discontinuation:	n	%
							- Chest tightness, dyspnea, and/or drop in ppFEV1	8	15
[75]	Retrospective Cohort	Baseline Age Adult - Mean: 31 yr   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean NR	28	- Increased work of breathing or chest tightness - Drop in ppFEV1	n 12   5	% 43  18	Discontinuation - Respiratory intolerance vs. pulmonary exacerbation - Persistent respiratory intolerance/chest tightness - Rash and swelling of face - Increased anxiety	n 1   3   1 1	% 4   11   4 4
[76]	Retrospective Cohort	Baseline Age Adult - Mean NR   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean NR	46		n	%	Discontinuation:	n	%
				- Drop in ppFEV1 - Transaminitis	21 2	46 4	- Dyspnea, cough, CFPEx, and/or chest tightness - Transaminitis - Headache - Muscle ache - Fatigue - Rash	4   1 NS NS NS NS	9   2 - - - -
[77]	Retrospective Cohort	Baseline Age Adult - Mean NR   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean NR	28	See Discontinuation			Discontinuation:	n	%
							- SOB and/or drop in ppFEV1	15	54
[78]	Retrospective Cohort	Baseline Age Mean: 32 yr h   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean: 62%	20	Overall AE:	n	%	Interruption (stop/restart):	n	%
				- Chest tightness - Elevated LFTs - Upset stomach - Increased stool output - Rash - Elevated thyroid function test - RA exacerbation	NS NS NS NS NS NS NS	- - - - - - -	- Unspecified AE  - full-dose restart  - half-dose restart   Discontinuation:  - Unspecified AE	2 4     2	10 20     10
[79]	Retrospective Cohort	Baseline Age Mean NR h   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean NR	60	- Heartburn/reflux - Abdominal pain - Loose/oily stools	n 20 19 17	% 33 32 28	None reported
[80]	Retrospective Cohort	Baseline Age Mean: 29 yr h,k   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean: 80% k	34	See Discontinuation			Discontinuation:	n	%
							Overall total:  - Respiratory AE (70%) f  - Unspecified reasons g	11 NS NS	32 - -
[81]	Cohort l	Baseline Age Pediatric - Mean NR   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean NR	39		n	%	None reported
				- AST >3x ULN	2	5
[82]	Cohort l	Baseline Age Pediatric and Adult - Range: 13–48 yr   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean NR	47	See Discontinuation			Discontinuation:	n	%
							- Thoracic oppression and unspecified AE	4	9
[83]	Cohort l	Baseline Age Adult - Mean: 28 yr   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean: 61%	46	See Discontinuation			Discontinuation:	n	%
							- Dyspnea, increased sputum, and unspecified AE	6	13
[37]	Cohort c,l	Baseline Age Adult - Mean: 31 yr   CFTR Genotype ∆F508/∆F508 Baseline ppFEV1 Mean: 28%	30	- Drop in ppFEV1 - Dyspnea, chest tightness, or chest pain - Increased sputum   *Based on 31 trials of LUM/IVA in 30 patients	n 30 * 25 * NS	% 97 81 -	Discontinuation: - Respiratory AE, unspecified - Hypertension	n 3 1	% 10 3
[84]	Cohort l	Baseline Age Adult - Mean: 31yr   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean: 40%	8	See Interruption			Interruption in n = 1 (13%):	n	%
							- Drop in ppFEV1 - Eczema	1 1	13 13
[38]	Cohort c,l	Baseline Age Mean NR h   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean NR	19	See Discontinuation			Discontinuation:	n	%
							- Chest tightness and dyspnea	4	21
[85]	Cross-sectional questionnaire	Baseline Age Mean NR h   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean NR	11	AE in n = 5 (46%): - Increased cough - Chest pain - Trouble breathing - Chest tightness - Stomach pain	n	%	Discontinuation:	n	%
					4 2 2 1 1	36 18 18 9 9	- Increased cough	1	9
Tezacaftor/Ivacaftor
[86]	Prospective Cohort	Baseline Age Pediatric - Mean: 16 yr   CFTR Genotype ∆F508 homozygous or heterozygous   Baseline ppFEV1 Mean: 82%	72	See Discontinuation			Discontinuation: Overall total: - New-onset hemoptysis - Persistent nausea/vomiting - Elevated LFTs - Mental health changes - Alterations in blood glucose - Acholic stools	n 8 NS NS NS NS NS NS	% 11 - - - - - -
[87]	Prospective Cohort	Baseline Age Mean NR h   CFTR Genotype NR   Baseline ppFEV1 Mean NR	50		n	%	Discontinuation:	n	%
				- AE not specified	5	10	- Liver function abnormalities	1	2
[88]	Prospective Cohort	Baseline Age Adult - Mean: 34 yr   CFTR Genotype ∆F508/∆F508   Baseline ppFEV1 Mean: 51%	5 m	AE in n = 5 (11%) m:	n	% m	Discontinuation:	n	% m
				- Sleep pattern disturbance - Out of body experience - Visual hallucination - Depersonalization - “Brain fog” - Severe migraine	2 1 1 1 1 1	5 2 2 2 2 2	- Out of body experience, visual hallucination - Depersonalization, “brain fog” - Severe migraine	1   1 1	2   2 2
[89]	Retrospective Cohort	Baseline Age Adult - Mean NR CFTR Genotype ∆F508/∆F508 Baseline ppFEV1 Mean NR	18	See Discontinuation			Discontinuation:	n	%
							- Hair loss and fatigue	1	6
[39]	Cohort c,l	Baseline Age Adult - Mean NR   CFTR Genotype NR   Baseline ppFEV1 Mean: 34%	22	AE in n = 3 (14%): - Rash - Blurred vision - Viral symptoms	n	%	Discontinued:	n	%
					2 1 1	9 5 5	- Blurred vision	1	5
Elexacaftor/Tezacaftor/Ivacaftor
[40]	Retrospective Cohort	Baseline Age Adult - Mean: 36 yr   CFTR Genotype ≥1 copy ∆F508   Baseline ppFEV1 Mean: 31%	11		n	%	None reported
				- Transaminitis	4	36

^a Rates not reported for all AE, as indicated by ‘NS’; ^b To avoid redundancy, if AE only reported in context of dose modification, interruption, and/or discontinuation of therapy, it was not listed in overall AE; ^c Study population part of a compassionate, ‘early access’, ‘expanded access’, ‘managed access’, or ‘named patient’ program; ^d Total study cohort of n = 11, but only n = 9 patients completed symptom questionnaire; AE frequency calculated based on total n = 11, ^e Mean baseline age and ppFEV₁ based on n = 135 in final analysis of outcomes assessing effectiveness; n = 234 excluded from this analysis; ^f As reported, unable to accurately determine the absolute number of patients who experienced AE; ^g Frequency of specific reasons for interruption or discontinuation not clear and include reasons unrelated to AE; ^h Included age groups (i.e., pediatric and/or adult) not specified; ⁱ Respiratory failure occurred in 1 individual on two occasions, both within 24 h of initiating and reinitiating LUM/IVA; ^j Of the n = 25 who stopped, 9 restarted and 6 of experienced the same AE; unclear which AE the 3 who restarted experienced and whether the 6 who experienced the same AE then discontinued permanently; ^k Mean baseline age and ppFEV₁ based on n = 23 in final analysis of outcomes assessing effectiveness; n = 11 excluded from this analysis; ^l Unable to discern if prospective versus retrospective based on reported information; ^m Total study cohort of n = 44, but focused on neurocognitive AE in n = 5; AE frequencies calculated based on total n = 44. AST, aspartate aminotransferase; CFTR, cystic fibrosis transmembrane conductance regulator; CFPEx, cystic fibrosis pulmonary exacerbation; GI, gastrointestinal; LFT, liver function test; LUM/IVA, lumacaftor/ivacaftor; NR, not reported; NS, not specified; ppFEV₁, percent predicted forced expiratory volume in 1 sec; RA, rheumatoid arthritis; SOB, shortness of breath; ULN, upper limit of normal; URTI, upper respiratory tract infection; yr, year(s).

appears below. The authors state that the scientific conclusions are unaffected. The original article has been updated.

In the original article [1], there was a mistake in Table A2 as published. Reference citation [48,49,53,54] were wrong. “Talkwalker” was misspelled. The corrected

Table A2. Summary of methodological ratings of included case series ^a,b.

Criteria	McKinzie et al., 2017 [47]	Nash et al., 2020 [27]	Rotolo et al., 2020 [52]	Safirstein et al., 2020 [53]	Talwalkar et al., 2017 [48]
1. Study objective clearly stated	Y	Y	N	Y	Y
2. Study population clearly defined, using case definition	N	N	N	N	N
3. Cases consecutive	NR	NR	NR	NR	NR
4. Subjects comparable	CD	CD	CD	N	N
5. Intervention clearly described	Y	Y	Y	Y	Y
6. Outcome measures clearly defined, valid, reliable, implemented consistently	N	N	N	Y	N
7. Adequate length of follow-up	Y	Y	Y	Y	CD
8. Statistical methods well-described	N/A	N/A	N/A	N/A	N
9. Results well-described	Y	N	Y	Y	Y
Final rating	Poor	Poor	Fair	Good	Poor

^a Studies were rated against the 9 criteria of the Quality Assessment for Case Series Studies from the National Institutes of Health, National Heart, Lung, and Blood Institute [24] from the standpoint of AE assessment; ^b Only case series with a full manuscript were assessed for quality. AE, adverse event; CD, cannot determine; N, no; N/A, not applicable; NR, not reported; Y, yes.

appears below. The authors state that the scientific conclusions are unaffected. The original article has been updated.