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- Publisher Website: 10.1093/ndt/gfw294
- Scopus: eid_2-s2.0-85034853782
- PMID: 27484667
- WOS: WOS:000414407200011
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Article: Prognostic enrichment design in clinical trials for autosomal dominant polycystic kidney disease: The HALT-PKD clinical trial
Title | Prognostic enrichment design in clinical trials for autosomal dominant polycystic kidney disease: The HALT-PKD clinical trial |
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Authors | |
Keywords | autosomal dominant polycystic kidney disease eGFR HALT-PKD study image classification total kidney volume |
Issue Date | 2017 |
Citation | Nephrology Dialysis Transplantation, 2017, v. 32, n. 11, p. 1857-1865 How to Cite? |
Abstract | Background Patients with mild autosomal dominant polycystic kidney disease (ADPKD) are less likely to be informative in randomized clinical trials (RCTs). We previously developed an imaging classification of ADPKD (typical diffuse cyst distribution Class 1A-E and atypical cyst distribution Class 2) for prognostic enrichment design in RCTs. We investigated whether using this classification would have increased the power to detect a beneficial treatment effect of rigorous blood pressure (BP) control on HALT-PKD participants with early disease (Study A). Methods Post hoc analysis of the early disease HALT-PKD study, an RCT that studied the effect of rigorous versus standard BP control on rates of total kidney volume (TKV) increase and estimated glomerular filtration rate (eGFR) decline in ADPKD patients with eGFR >60 mL/min/1.73 m 2. Results Five hundred and fifty-one patients were classified by two observers (98.2% agreement) into Class 1A (6.2%), 1B (20.3%), 1C (34.1%), 1D (22.1%), 1E (11.8%) and 2 (5.4%). The TKV increase and eGFR decline became steeper from Class 1A through 1E. Rigorous BP control had been shown to be associated with slower TKV increase, without a significant overall effect on the rate of eGFR decline (faster in the first 4 months and marginally slower thereafter). Merging Classes 1A and 2 (lowest severity), 1B and 1C (intermediate severity) and 1D and 1E (highest severity) detected stronger beneficial effects on TKV increase and eGFR decline in Class 1D and E with a smaller number of patients. Conclusions Strategies for prognostic enrichment, such as image classification, should be used in the design of RCTs for ADPKD to increase their power and reduce their cost. |
Persistent Identifier | http://hdl.handle.net/10722/316152 |
ISSN | 2023 Impact Factor: 4.8 2023 SCImago Journal Rankings: 1.414 |
ISI Accession Number ID |
DC Field | Value | Language |
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dc.contributor.author | Irazabal, María V. | - |
dc.contributor.author | Abebe, Kaleab Z. | - |
dc.contributor.author | Bae, Kyongtae Ty | - |
dc.contributor.author | Perrone, Ronald D. | - |
dc.contributor.author | Chapman, Arlene B. | - |
dc.contributor.author | Schrier, Robert W. | - |
dc.contributor.author | Yu, Alan S. | - |
dc.contributor.author | Braun, William E. | - |
dc.contributor.author | Steinman, Theodore I. | - |
dc.contributor.author | Harris, Peter C. | - |
dc.contributor.author | Flessner, Michael F. | - |
dc.contributor.author | Torres, Vicente E. | - |
dc.date.accessioned | 2022-08-24T15:49:25Z | - |
dc.date.available | 2022-08-24T15:49:25Z | - |
dc.date.issued | 2017 | - |
dc.identifier.citation | Nephrology Dialysis Transplantation, 2017, v. 32, n. 11, p. 1857-1865 | - |
dc.identifier.issn | 0931-0509 | - |
dc.identifier.uri | http://hdl.handle.net/10722/316152 | - |
dc.description.abstract | Background Patients with mild autosomal dominant polycystic kidney disease (ADPKD) are less likely to be informative in randomized clinical trials (RCTs). We previously developed an imaging classification of ADPKD (typical diffuse cyst distribution Class 1A-E and atypical cyst distribution Class 2) for prognostic enrichment design in RCTs. We investigated whether using this classification would have increased the power to detect a beneficial treatment effect of rigorous blood pressure (BP) control on HALT-PKD participants with early disease (Study A). Methods Post hoc analysis of the early disease HALT-PKD study, an RCT that studied the effect of rigorous versus standard BP control on rates of total kidney volume (TKV) increase and estimated glomerular filtration rate (eGFR) decline in ADPKD patients with eGFR >60 mL/min/1.73 m 2. Results Five hundred and fifty-one patients were classified by two observers (98.2% agreement) into Class 1A (6.2%), 1B (20.3%), 1C (34.1%), 1D (22.1%), 1E (11.8%) and 2 (5.4%). The TKV increase and eGFR decline became steeper from Class 1A through 1E. Rigorous BP control had been shown to be associated with slower TKV increase, without a significant overall effect on the rate of eGFR decline (faster in the first 4 months and marginally slower thereafter). Merging Classes 1A and 2 (lowest severity), 1B and 1C (intermediate severity) and 1D and 1E (highest severity) detected stronger beneficial effects on TKV increase and eGFR decline in Class 1D and E with a smaller number of patients. Conclusions Strategies for prognostic enrichment, such as image classification, should be used in the design of RCTs for ADPKD to increase their power and reduce their cost. | - |
dc.language | eng | - |
dc.relation.ispartof | Nephrology Dialysis Transplantation | - |
dc.subject | autosomal dominant polycystic kidney disease | - |
dc.subject | eGFR | - |
dc.subject | HALT-PKD study | - |
dc.subject | image classification | - |
dc.subject | total kidney volume | - |
dc.title | Prognostic enrichment design in clinical trials for autosomal dominant polycystic kidney disease: The HALT-PKD clinical trial | - |
dc.type | Article | - |
dc.description.nature | link_to_subscribed_fulltext | - |
dc.identifier.doi | 10.1093/ndt/gfw294 | - |
dc.identifier.pmid | 27484667 | - |
dc.identifier.scopus | eid_2-s2.0-85034853782 | - |
dc.identifier.volume | 32 | - |
dc.identifier.issue | 11 | - |
dc.identifier.spage | 1857 | - |
dc.identifier.epage | 1865 | - |
dc.identifier.eissn | 1460-2385 | - |
dc.identifier.isi | WOS:000414407200011 | - |
dc.identifier.f1000 | 726606200 | - |