Title: Pharmacotherapy for Primary Biliary Cholangitis: An Assessment of Medication
Candidacy and Rates of Treatment
Version: 1 Date: 12 Oct 2023
Reviewer's report:
As it is generally acknowledged, UDCA remains the first-line therapy for the disease, but
alternative therapies, such as obeticholic acid, have recently emerged, albeit restricted to patients
with either intolerance or poor response to UDCA.  The authors performed an observational
study, analyzing records from a large urban health system with regards to medications.
Accordingly, they managed to quantify candidacy and usage rates of pharmacotherapy in
patients with an established diagnosis of PBC. Of interest, they noted an almost universal
compliance to guidelines and UDCA prescription.
However, I would like to comment on the following:
1) Background: It is well-known that use of UDCA has improved the natural history of the
disease, but ref 3-6 reflect mainly historical data. It should be noted that the disease nowadays
is detected in earlier stages and epidemiology has slightly changed.
2) Table 1 should be improved in terms of appearance/design, i.e the total sample size would
better be presented in the first raw (n=495) instead of a separate one. In addition, regarding
ethnicity, it is not clear why patients of Hispanic origin are considered  as a distinct population,
since in that case the total number of patients would be 505. It would be also interesting if
demographic parameters other than ethnicity could be included, such as age, disease duration
and presence of cirrhosis (clinical or in imaging).
3) Results: calculation of percentages is not always straightforward (it is unclear if the calculation
corresponds to the total sample size or not). It would also be helpful to explain what the
difference between barrier and lack of offering OCA is. Also, there is a significant amount
of patients who were not offered OCA for indeterminate reasons or received indeterminate
medication and therefore results could be altered. Moreover, low percentages of fibrate treatment
raise a question.
4) Discussion: A reference is required for the sentence ”Despite prior investigations identifying
that as many as 30% of patients with PBC may have never received treatment with UDCA..”
Other than the above, the study is well written and provides significant information about use of
UDCA based on real world experience. Therefore, my final decision is to accept the publication
with some improvements.
Title: Pharmacotherapy for Primary Biliary Cholangitis: An Assessment of Medication
Candidacy and Rates of Treatment
Version: 1 Date: 12 Oct 2023
Reviewer's report:
As it is generally acknowledged, UDCA remains the first-line therapy for the disease, but
alternative therapies, such as obeticholic acid, have recently emerged, albeit restricted to patients
with either intolerance or poor response to UDCA.  The authors performed an observational
study, analyzing records from a large urban health system with regards to medications.
Accordingly, they managed to quantify candidacy and usage rates of pharmacotherapy in
patients with an established diagnosis of PBC. Of interest, they noted an almost universal
compliance to guidelines and UDCA prescription.
However, I would like to comment on the following:
1) Background: It is well-known that use of UDCA has improved the natural history of the
disease, but ref 3-6 reflect mainly historical data. It should be noted that the disease nowadays
is detected in earlier stages and epidemiology has slightly changed.
2) Table 1 should be improved in terms of appearance/design, i.e the total sample size would
better be presented in the first raw (n=495) instead of a separate one. In addition, regarding
ethnicity, it is not clear why patients of Hispanic origin are considered  as a distinct population,
since in that case the total number of patients would be 505. It would be also interesting if
demographic parameters other than ethnicity could be included, such as age, disease duration
and presence of cirrhosis (clinical or in imaging).
3) Results: calculation of percentages is not always straightforward (it is unclear if the calculation
corresponds to the total sample size or not). It would also be helpful to explain what the
difference between barrier and lack of offering OCA is. Also, there is a significant amount
of patients who were not offered OCA for indeterminate reasons or received indeterminate
medication and therefore results could be altered. Moreover, low percentages of fibrate treatment
raise a question.
4) Discussion: A reference is required for the sentence ”Despite prior investigations identifying
that as many as 30% of patients with PBC may have never received treatment with UDCA..”
Other than the above, the study is well written and provides significant information about use of
UDCA based on real world experience. Therefore, my final decision is to accept the publication
with some improvements.
Please review this paper.
user
Title: Pharmacotherapy for Primary Biliary Cholangitis: An Assessment of Medication
Candidacy and Rates of Treatment
Version: 1 Date: 12 Oct 2023
Reviewer's report:
As it is generally acknowledged, UDCA remains the first-line therapy for the disease, but
alternative therapies, such as obeticholic acid, have recently emerged, albeit restricted to patients
with either intolerance or poor response to UDCA.  The authors performed an observational
study, analyzing records from a large urban health system with regards to medications.
Accordingly, they managed to quantify candidacy and usage rates of pharmacotherapy in
patients with an established diagnosis of PBC. Of interest, they noted an almost universal
compliance to guidelines and UDCA prescription.
However, I would like to comment on the following:
1) Background: It is well-known that use of UDCA has improved the natural history of the
disease, but ref 3-6 reflect mainly historical data. It should be noted that the disease nowadays
is detected in earlier stages and epidemiology has slightly changed.
2) Table 1 should be improved in terms of appearance/design, i.e the total sample size would
better be presented in the first raw (n=495) instead of a separate one. In addition, regarding
ethnicity, it is not clear why patients of Hispanic origin are considered  as a distinct population,
since in that case the total number of patients would be 505. It would be also interesting if
demographic parameters other than ethnicity could be included, such as age, disease duration
and presence of cirrhosis (clinical or in imaging).
3) Results: calculation of percentages is not always straightforward (it is unclear if the calculation
corresponds to the total sample size or not). It would also be helpful to explain what the
difference between barrier and lack of offering OCA is. Also, there is a significant amount
of patients who were not offered OCA for indeterminate reasons or received indeterminate
medication and therefore results could be altered. Moreover, low percentages of fibrate treatment
raise a question.
4) Discussion: A reference is required for the sentence ”Despite prior investigations identifying
that as many as 30% of patients with PBC may have never received treatment with UDCA..”
Other than the above, the study is well written and provides significant information about use of
UDCA based on real world experience. Therefore, my final decision is to accept the publication
with some improvements.
assistant