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

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).

In the original article [1], there was a mistake in Table 2 as published. Subtitle “Lumacaftor/Ivacaftor” was wrong. The corrected Table 2 appears below. The authors state that the scientific conclusions are unaffected. The original article has been updated.

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).

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

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.