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 [...].

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 AgePediatric and Adult- Mean: 33 yr- Range: 10–61 yr CFTR Genotype≥1 copy G551D Baseline ppFEV1Mean: 30%	44	Recruitment PeriodPrior to commercial availability Follow-Up DurationNS	AE in n = 38 (86%):	n	%	Discontinuation:	n	%
					- Pulmonary exacerbation- Hemoptysis- Increased sputum- Increased cough- URTI- Dyspnea- Abnormal respiration - Respiratory tract congestion- Headache- Rash	20776633354	4516161414777119	- Severe abdominal pain- Dizziness/tinnitus	11	22
					SAE in n = 14 (32%):
					- Pulmonary exacerbation- Hemoptysis- Pneumothorax- Acute respiratory failure- URTI- Abdominal pain- Gastroenteritis- Abnormal LFTs- Syncope- Secondary adrenocortical insufficiency	NSNSNSNSNSNSNSNSNSNS	----------
[55]	Prospective Cohort United StatesCanadaItaly	Baseline AgePediatric and Adult- Mean: 17 yr- Range: 5–61 yr CFTR Genotype≥1 gating mutation Baseline ppFEV1Mean: 86%	23	Recruitment Period Mar 2014 to Aug 2015 Follow-Up Duration3 mo	49 AE in n = 21 (91%):	n	%	None reported
					- Respiratory, unspecified- Gastrointestinal, unspecified- Infection, unspecified- Headache- Weakness- Dizziness- Fatigue	NSNSNSNSNSNSNS	-------
					5 SAE in n = 3 (13%):	n	%
					- Respiratory infection- Acute changes in metabolic and liver status	41	174
[56]	Retrospective Cohort United Kingdom(1 center)	Baseline AgePediatric - Mean: 9 yr- Range: 6–14 yr CFTR Genotype1 copy G551D Baseline ppFEV1Mean: 68% e	4	Recruitment PeriodJan 2013 to Jun 2015 Follow-Up DurationMean: 24 mo	- Transaminitis (<3 x ULN)	n1	%25	None reported
[57]	Retrospective Cohort Scotland(11 centers)	Baseline AgePediatric - Median: 9 yr CFTR Genotype≥1 copy G551D Baseline ppFEV1Mean: 85%	26	Recruitment PeriodNS (Jan 2013 to Mar 2013 for 85%) Follow-Up DurationMean: 17 mo		n	%	None reported
					- Headache - Swollen ear - Cataracts	112	4417 f
[58]	Retrospective Cohort France(25 centers)	Baseline AgePediatric and Adult- Median: 18 yr- Range: 6–52 yr CFTR Genotype≥1 copy G551D Baseline ppFEV1Mean: 72%	57	Recruitment PeriodPre-1 Jun 2013 up to 30 Sep 2014  Follow-Up DurationUp 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	3NSNSNSNSNSNSNSNSNSNSNSNSNSNSNSNS	5----------------	- Hepatitis- Rhinopharyngitis- Abdominal pain- Vomiting- Headache- Rash- Severe depression	NSNSNSNSNSNSNS	-------
								Discontinuation:
								- Transaminitis- Liver cirrhosis diagnosis	11	22
[30]	Retrospective Cohort d Germany(multicenter)	Baseline AgeAdult - Mean: 34 yr CFTR Genotype≥1 copy G551D Baseline ppFEV1Mean: 25%	14	Recruitment PeriodSep 2012 to Apr 2013 Follow-Up DurationMean: 235 days	- Increased bronchial and nasal secretions - Headache- Worsening RLS- Abdominal pain- Hyperbilirubinemia (mild)- Transaminitis (<3 to 4x ULN)	n311111	%2177777	Discontinuation:- Increased bronchial and nasal secretions * * Trial of reduced dose before discontinuation	n1	%7
Lumacaftor/Ivacaftor
[41]	Prospective Cohort France (1 center)	Baseline AgePediatric- Mean: 16 yr CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean: 87%	32	Recruitment PeriodMar 2016 to Dec 2016 Follow-Up Duration4 h post-first dose	- Acute drop in ppFEV1- Wheeze	n323	%1009	None reported
[42]	Prospective Cohort  France(47 centers)	Baseline AgePediatric and Adult- Mean: 22 yr CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean: 65%	845	Recruitment Period1 Jan 2016 to 31 Dec 2016 Follow-Up Duration12 mo	AE in n = 494 (59%):	n	%	Interruption:	n	%
					- Respiratory- Digestive- Menstrual abnormality- Fatigue- Headache- CK > 5xULN - Transaminitis (> 3xULN)	316181533719205	372164220.6	- 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	168 n 3824921  1855443211	21 % 5310.20.1  20.60.60.50.50.40.20.10.1
[59]	Prospective Cohort United States(1 center)	Baseline AgePediatric and Adult- Mean: 23 yr- Range: 12–48 yr CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean: 70%	26	Recruitment PeriodNS Follow-Up Duration6 mo	See Discontinuation			Discontinuation:- Transaminitis- Unspecified AE	n14	%415
[31]	Prospective Cohort d Switzerland(1 center)	Baseline AgeAdult- Mean NR CFTR Genotype∆F508/∆F508 Baseline ppFEV1Median: 30%	20	Recruitment PeriodJan 2016 to Jan 2017 Follow-Up Duration1 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	011 1101 183 2	-55 5505 54015 10	- Respiratory intolerance Discontinuation:- Chest tightness (at 24 h)	3  1	15  5
[32]	Prospective Cohort Australia(1 center)	Baseline AgeAdult- Mean: 27 yr CFTR Genotype∆F508/∆F508 Baseline ppFEV1Median: 36%	12	Recruitment PeriodJan 2016 to Oct 2016 Follow-Up Duration1 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 5108 267 485 0216	100 428367 175058 336742 -17850	- Chest tightness/dyspnea * * n = 2 discontinued after 1mo follow-up (5 wk and 9 wk)	3	25
[33]	Prospective Cohort France(11 centers)	Baseline AgeAdult- Mean: 31 yr CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean: 32%	53	Recruitment PeriodJan 2016 to Jun 2016  Follow-Up Duration3 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	131139 2111	2521617 4222	- Respiratory intolerance- Vomiting- Fatigue	1311	2522
[25]	Prospective Cohort d,g Australia(1 center)	Baseline AgeAdult- Mean: 27 yr CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean: 36%	10	Recruitment PeriodNS Follow-Up Duration52 wk	AE in n = 6 (60%):	n	%	None reported
					- Chest tightness/dyspnea- Headache	62	6020
[43]	Retrospective CohortIreland (1 center)	Baseline AgePediatric- Mean: 14 yr CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean: 77%	15	Recruitment PeriodSep 2016 to Aug 2017 Follow-Up DurationNS		n	%	None reported
					- Acute drop in ppFEV1- Chest tightness- Increased sputum	1422	931313
[44]	Retrospective Cohort  United States(1 center)	Baseline AgePediatric and Adult- Mean: 25 yr- Range: 12–59 yr CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean: 67%	116	Recruitment PeriodNS Follow-Up DurationUp to 11 mo	AE in n = 46 (40%):	n	%	Reduced dose:	n	%
					- Chest tightness- Dyspnea- Increased cough- Diarrhea- Nausea- Decreased appetite- Rash	2312105322	201094322	- AE not specified Discontinuation:- Reasons not specified h	10  20	9  17
[60]	Retrospective Cohort Greece(1 center)	Baseline AgePediatric and Adult- Mean: 16 yr i- Range: 12–23 yr CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean 92% i	62	Recruitment PeriodMar 2016 to Aug 2017 Follow-Up Duration12 mo	- Chest tightness	n2	%3	Discontinuation:- Transaminitis- Cataract	n11	%22
[34]	Retrospective Cohort d Spain(multicenter)	Baseline Age Pediatric and Adult- Mean: 27 yr- Range: 10–45 yr CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean: 32%	20	Recruitment Period2016 Follow-Up Duration6 mo	AE in n = 15 (75%):	n	%	Discontinuation:	n	%
					- Chest tightness- Dyspnea- Headache- Weight loss- ‘Sickness’ (not defined)- Asthenia- Abdominal pain- Transaminitis	98553322	4540252515151010	- Decreased ppFEV1- AE not specified	16	530
[45]	Retrospective Cohort Canada(1 center)	Baseline AgeAdult- Median: 32 yr CFTR Genotype∆F508/∆F508 Baseline ppFEV1Median: 40%	22	Recruitment PeriodApr 2016 to Jun 2018 Follow-Up DurationMedian: 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	14433215421111	6418141495231895555	- Respiratory symptoms- Asymptomatic hypertension- Symptomatic hypertension - Headache - Hypertensive emergency- Anxiety	32 111	149 555
[61]	Retrospective Cohort United States(1 center)	Baseline AgeAdult- Mean NR CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean NR	82	Recruitment PeriodJul 2015 to Jun 2016 Follow-Up Duration12 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	n171121111111	%211321111111
[26]	Retrospective Cohort d,g Australia(7 centers)	Baseline AgeAdult- Mean: 31 yr CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean: 37%	72	Recruitment PeriodNov 2015 to Mar 2017 Follow-Up Duration12 mo		n	%	Discontinuation:	n	%
					- Chest tightness/dyspnea- Increased sputum- Decrease in ppFEV1- Headache- Fatigue- Nausea- Rash	40422512	56633713	- Chest tightness/dyspnea	22	31
[27]	Case Series (Survey) j International (31 centers)	Baseline AgeAdult - Mean: 30 yr CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean: 59%	26	Recruitment PeriodQuestionnaire sent in 2018–2019 Follow-Up DurationNS		n	%	Discontinuation:	n	%
					- Pulmonary exacerbation- Post-partum acute myelocytic leukemia	11	44	- 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; ppFEV, 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 AgePediatric- Mean: 5 yr CFTR Genotype≥1 gating mutation Baseline ppFEV1Mean NR	4	AE in n = 2 (50%):	n	%	None reported
				- URTI- Nasal congestion- Headache	NSNSNS	---
[63]	Retrospective Cohort	Baseline AgePediatric - Mean: 6 yr CFTR Genotype≥1 gating mutation Baseline ppFEV1Median: 87%	10	- Transient rash- Increased obesity	n21	%2010	None reported
[64]	Prospective Cohort	Baseline AgePediatric and Adult- Mean NR CFTR Genotype≥1 copy S549R Baseline ppFEV1Mean: 54%	15	- Liver enzyme derangement	n	%	None reported
					2	13
[65]	Cross-sectional Survey	Baseline AgeAdult- Mean: 26 yr CFTR Genotype≥1G551D Baseline ppFEV1Mean: 62%	11 d	AE in n = 8 (73%) d:- Transient rash- Dizziness- Unspecified AE	nNSNSNS	%---	None reported
Lumacaftor/Ivacaftor
[66]	Prospective Cohort	Baseline AgePediatric - Mean: 13 yr CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean: 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 AgePediatric - Mean: 14 yr CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean: 87%	13		n	%	None reported
				- Drop in ppFEV1 requiring salbutamol	7	54
[68]	Prospective Cohort	Baseline AgePediatric and Adult- Mean: 23 yr e CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean 61% e	369		n	%	Discontinuation:	n	%
				- Bronchospasm- Dyspnea- Abnormal respiration - Unspecified respiratory AE- Unspecified AE	151274120	432133	- Unspecified AE	16	4
[69]	Prospective Cohort	Baseline AgePediatric and Adult- Mean: 25 yr CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean NR	311	379 AE in n = 213 (68%):- Dyspnea- Cough- GI discomfort (e.g., diarrhea, nausea, abdominal pain)- Headache- Fatigue- Unspecified	n fNSNSNS NSNSNS	%31631 65NR	Interruption (stop/restart):- Unspecified AE and other reasons g Discontinuation:- Unspecified AE and other reasons g	n12  42	%4  14
[35]	Prospective Cohort c	Baseline AgeAdult - Median: 31 yr CFTR Genotype∆F508/∆F508 Baseline ppFEV1Median: 28%	14		n	%	Discontinuation:	n	%
				- Chest tightness, breathless- Rash	71	507	- Respiratory AE and/or rash	4	29
[70]	Prospective Cohort	Baseline AgeAdult - Mean NR CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean 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 AgeMean NR h CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean NR	32	AE in 88%:	n f	%	Interruption (stop/restart):	n	%
				- Respiratory AE- Drop in ppFEV1	NSNS	87-	- Unspecified AEA Discontinuation:- Unspecified AE	1  8	3  25
[71]	Retrospective Cohort	Baseline AgePediatric and Adult - Mean NR  CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean NR	34	AE in n = 29 (85%):- Pulmonary exacerbation- Chest tightness- Dyspnea- Diarrhea- Abdominal pain	n169333	%4726999	Discontinuation:- Unspecified AE	n10	%29
				Serious AE in n = 8 (24%):
				- Respiratory failure i- Unspecified AE	17	321
[72]	Retrospective Cohort	Baseline AgePediatric and Adult - Mean: 26 yr CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean: 68%	103	See Discontinuation			Interruption/discontinuation j:	n	%
							- Chest tightness and/or pain- Elevated LFTs	17NS	17-
[73]	Retrospective Cohort	Baseline AgeAdult - Mean: 31 yr  CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean: 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	22846413211	311169614311	- Dyspnea- Chest tightness- Increased cough- Fatigue	7631	10941
[74]	Retrospective Cohort	Baseline AgeMean NR h CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean NR	54	See Discontinuation			Discontinuation:	n	%
							- Chest tightness, dyspnea, and/or drop in ppFEV1	8	15
[75]	Retrospective Cohort	Baseline AgeAdult - Mean: 31 yr  CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean NR	28	- Increased work of breathing or chest tightness- Drop in ppFEV1	n12 5	%43 18	Discontinuation- Respiratory intolerance vs. pulmonary exacerbation- Persistent respiratory intolerance/chest tightness- Rash and swelling of face- Increased anxiety	n1 3 11	%4 11 44
[76]	Retrospective Cohort	Baseline AgeAdult - Mean NR CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean NR	46		n	%	Discontinuation:	n	%
				- Drop in ppFEV1 - Transaminitis	212	464	- Dyspnea, cough, CFPEx, and/or chest tightness- Transaminitis- Headache- Muscle ache- Fatigue- Rash	4 1NSNSNSNS	9 2----
[77]	Retrospective Cohort	Baseline AgeAdult - Mean NR CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean NR	28	See Discontinuation			Discontinuation:	n	%
							- SOB and/or drop in ppFEV1	15	54
[78]	Retrospective Cohort	Baseline AgeMean: 32 yr h CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean: 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	NSNSNSNSNSNSNS	-------	- Unspecified AE - full-dose restart - half-dose restart Discontinuation: - Unspecified AE	24  2	1020  10
[79]	Retrospective Cohort	Baseline AgeMean NR h CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean NR	60	- Heartburn/reflux- Abdominal pain- Loose/oily stools	n201917	%333228	None reported
[80]	Retrospective Cohort	Baseline AgeMean: 29 yr h,k CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean: 80% k	34	See Discontinuation			Discontinuation:	n	%
							Overall total: - Respiratory AE (70%) f - Unspecified reasons g	11NSNS	32--
[81]	Cohort l	Baseline AgePediatric- Mean NR CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean NR	39		n	%	None reported
				- AST >3x ULN	2	5
[82]	Cohort l	Baseline AgePediatric and Adult - Range: 13–48 yr CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean NR	47	See Discontinuation			Discontinuation:	n	%
							- Thoracic oppression and unspecified AE	4	9
[83]	Cohort l	Baseline AgeAdult - Mean: 28 yr CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean: 61%	46	See Discontinuation			Discontinuation:	n	%
							- Dyspnea, increased sputum, and unspecified AE	6	13
[37]	Cohort c,l	Baseline AgeAdult - Mean: 31 yr CFTR Genotype∆F508/∆F508Baseline ppFEV1Mean: 28%	30	- Drop in ppFEV1- Dyspnea, chest tightness, or chest pain- Increased sputum *Based on 31 trials of LUM/IVA in 30 patients	n30 *25 *NS	%9781-	Discontinuation:- Respiratory AE, unspecified- Hypertension	n31	%103
[84]	Cohort l	Baseline AgeAdult - Mean: 31yr  CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean: 40%	8	See Interruption			Interruption in n = 1 (13%):	n	%
							- Drop in ppFEV1 - Eczema	11	1313
[38]	Cohort c,l	Baseline AgeMean NR h CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean NR	19	See Discontinuation			Discontinuation:	n	%
							- Chest tightness and dyspnea	4	21
[85]	Cross-sectional questionnaire	Baseline AgeMean NR h CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean NR	11	AE in n = 5 (46%):- Increased cough- Chest pain- Trouble breathing- Chest tightness- Stomach pain	n	%	Discontinuation:	n	%
					42211	36181899	- Increased cough	1	9
Tezacaftor/Ivacaftor
[86]	Prospective Cohort	Baseline AgePediatric- Mean: 16 yr CFTR Genotype∆F508 homozygous or heterozygous Baseline ppFEV1Mean: 82%	72	See Discontinuation			Discontinuation:Overall total:- New-onset hemoptysis- Persistent nausea/vomiting- Elevated LFTs- Mental health changes- Alterations in blood glucose- Acholic stools	n8NSNSNSNSNSNS	%11------
[87]	Prospective Cohort	Baseline AgeMean NR h CFTR GenotypeNR Baseline ppFEV1Mean NR	50		n	%	Discontinuation:	n	%
				- AE not specified	5	10	- Liver function abnormalities	1	2
[88]	Prospective Cohort	Baseline AgeAdult- Mean: 34 yr CFTR Genotype∆F508/∆F508 Baseline ppFEV1Mean: 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	211111	522222	- Out of body experience, visual hallucination- Depersonalization, “brain fog”- Severe migraine	1 11	2 22
[89]	Retrospective Cohort	Baseline AgeAdult- Mean NRCFTR Genotype∆F508/∆F508Baseline ppFEV1Mean NR	18	See Discontinuation			Discontinuation:	n	%
							- Hair loss and fatigue	1	6
[39]	Cohort c,l	Baseline AgeAdult - Mean NR CFTR GenotypeNR  Baseline ppFEV1Mean: 34%	22	AE in n = 3 (14%):- Rash- Blurred vision- Viral symptoms	n	%	Discontinued:	n	%
					211	955	- Blurred vision	1	5
Elexacaftor/Tezacaftor/Ivacaftor
[40]	Retrospective Cohort	Baseline AgeAdult- Mean: 36 yr CFTR Genotype≥1 copy ∆F508 Baseline ppFEV1Mean: 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; ppFEV 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.