Incidence of fibrosing colonopathy with pancreatic enzyme replacement therapy in patients with cystic fibrosis
Copyright © 2023. Published by Elsevier B.V..
BACKGROUND: High daily doses of pancreatic enzyme replacement therapy (PERT) were historically associated with risk of fibrosing colonopathy (FC) in people with cystic fibrosis (pwCF), leading to development of PERT dosing guidelines and reformulated products. This study quantified incidence of FC in pwCF treated with PERT following those measures.
METHODS: This large prospective cohort study included eligible pwCF enrolled in the Cystic Fibrosis Foundation Patient Registry with ≥1 clinic visit in 2012-2014 and follow-up through 2020. Data on PERT exposure, demographics, and medical history were collected. Clinical data, imaging, and histopathology of suspected cases were examined by an independent adjudication panel of physicians familiar with this complication.
RESULTS: Base Study Population included 26,025 pwCF who contributed 155,814 person-years [mean (SD) 6.0 (2.0) years] of follow-up. Over 7.8 years, 29 pwCF had suspected FC; three cases were confirmed by adjudication, 22 cases were confirmed as not FC, and four cases were indeterminate. There were 22,161 pwCF exposed to any PERT, with mean PERT use time of 5.583 person-years and mean daily dose of 8328 U lipase per kg per day. All three confirmed cases and four indeterminate cases of FC occurred during current use of PERT. Incidence rates per 1000 person-years exposed were 0.0242 (95 % CI [0.0050, 0.0709]) for confirmed FC and 0.0566 (95 % CI [0.0227, 0.1166]) for indeterminate or confirmed FC.
CONCLUSIONS: The incidence of FC in pwCF is very low in the era of current treatment guidelines and more stringent quality standards for PERT products.
| Medienart: |
E-Artikel |
|---|
| Erscheinungsjahr: |
2023 |
|---|---|
| Erschienen: |
2023 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:22 |
|---|---|
| Enthalten in: |
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 22(2023), 6 vom: 23. Nov., Seite 1017-1023 |
| Sprache: |
Englisch |
|---|
| Beteiligte Personen: |
Chiuve, Stephanie E [VerfasserIn] |
|---|
| Links: |
|---|
| Themen: |
Cohort study |
|---|
| Anmerkungen: |
Date Completed 04.12.2023 Date Revised 04.12.2023 published: Print-Electronic Citation Status MEDLINE |
|---|
| doi: |
10.1016/j.jcf.2023.08.013 |
|---|
| funding: |
|
|---|---|
| Förderinstitution / Projekttitel: |
|
| PPN (Katalog-ID): |
NLM363722327 |
|---|
| LEADER | 01000naa a22002652 4500 | ||
|---|---|---|---|
| 001 | NLM363722327 | ||
| 003 | DE-627 | ||
| 005 | 20231226093944.0 | ||
| 007 | cr uuu---uuuuu | ||
| 008 | 231226s2023 xx |||||o 00| ||eng c | ||
| 024 | 7 | |a 10.1016/j.jcf.2023.08.013 |2 doi | |
| 028 | 5 | 2 | |a pubmed24n1212.xml |
| 035 | |a (DE-627)NLM363722327 | ||
| 035 | |a (NLM)37880023 | ||
| 035 | |a (PII)S1569-1993(23)00880-9 | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rakwb | ||
| 041 | |a eng | ||
| 100 | 1 | |a Chiuve, Stephanie E |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Incidence of fibrosing colonopathy with pancreatic enzyme replacement therapy in patients with cystic fibrosis |
| 264 | 1 | |c 2023 | |
| 336 | |a Text |b txt |2 rdacontent | ||
| 337 | |a ƒaComputermedien |b c |2 rdamedia | ||
| 338 | |a ƒa Online-Ressource |b cr |2 rdacarrier | ||
| 500 | |a Date Completed 04.12.2023 | ||
| 500 | |a Date Revised 04.12.2023 | ||
| 500 | |a published: Print-Electronic | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a Copyright © 2023. Published by Elsevier B.V. | ||
| 520 | |a BACKGROUND: High daily doses of pancreatic enzyme replacement therapy (PERT) were historically associated with risk of fibrosing colonopathy (FC) in people with cystic fibrosis (pwCF), leading to development of PERT dosing guidelines and reformulated products. This study quantified incidence of FC in pwCF treated with PERT following those measures | ||
| 520 | |a METHODS: This large prospective cohort study included eligible pwCF enrolled in the Cystic Fibrosis Foundation Patient Registry with ≥1 clinic visit in 2012-2014 and follow-up through 2020. Data on PERT exposure, demographics, and medical history were collected. Clinical data, imaging, and histopathology of suspected cases were examined by an independent adjudication panel of physicians familiar with this complication | ||
| 520 | |a RESULTS: Base Study Population included 26,025 pwCF who contributed 155,814 person-years [mean (SD) 6.0 (2.0) years] of follow-up. Over 7.8 years, 29 pwCF had suspected FC; three cases were confirmed by adjudication, 22 cases were confirmed as not FC, and four cases were indeterminate. There were 22,161 pwCF exposed to any PERT, with mean PERT use time of 5.583 person-years and mean daily dose of 8328 U lipase per kg per day. All three confirmed cases and four indeterminate cases of FC occurred during current use of PERT. Incidence rates per 1000 person-years exposed were 0.0242 (95 % CI [0.0050, 0.0709]) for confirmed FC and 0.0566 (95 % CI [0.0227, 0.1166]) for indeterminate or confirmed FC | ||
| 520 | |a CONCLUSIONS: The incidence of FC in pwCF is very low in the era of current treatment guidelines and more stringent quality standards for PERT products | ||
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a Cohort study | |
| 650 | 4 | |a Cystic fibrosis | |
| 650 | 4 | |a Fibrosing colonopathy | |
| 650 | 4 | |a Pancreatic enzyme replacement therapy | |
| 650 | 4 | |a Pharmacoepidemiology | |
| 700 | 1 | |a Fife, Daniel |e verfasserin |4 aut | |
| 700 | 1 | |a Leitz, Gerhard |e verfasserin |4 aut | |
| 700 | 1 | |a Peterson, Craig |e verfasserin |4 aut | |
| 700 | 1 | |a Campbell, Neil M |e verfasserin |4 aut | |
| 700 | 1 | |a Rennig, Amanda |e verfasserin |4 aut | |
| 700 | 1 | |a Rodrigues, Lino |c Jr |e verfasserin |4 aut | |
| 700 | 1 | |a Decktor, Dennis |e verfasserin |4 aut | |
| 700 | 1 | |a Dowd, Christopher |e verfasserin |4 aut | |
| 700 | 1 | |a Marshall, Bruce C |e verfasserin |4 aut | |
| 700 | 1 | |a Borowitz, Drucy |e verfasserin |4 aut | |
| 773 | 0 | 8 | |i Enthalten in |t Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society |d 2002 |g 22(2023), 6 vom: 23. Nov., Seite 1017-1023 |w (DE-627)NLM15136754X |x 1873-5010 |7 nnns |
| 773 | 1 | 8 | |g volume:22 |g year:2023 |g number:6 |g day:23 |g month:11 |g pages:1017-1023 |
| 856 | 4 | 0 | |u http://dx.doi.org/10.1016/j.jcf.2023.08.013 |3 Volltext |
| 912 | |a GBV_USEFLAG_A | ||
| 912 | |a GBV_NLM | ||
| 951 | |a AR | ||
| 952 | |d 22 |j 2023 |e 6 |b 23 |c 11 |h 1017-1023 | ||