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Thiola EC represents several modifications of Thiola that promise better, more efficacious therapy of cystinuria. First, pill size has changed from 100 mg to 300 mg, meaning that typical pill burden will be reduced from, on average, 10 pills per day to 3-5 pills per day. This change will be welcomed by patients whose fluid intake and administration of potassium citrate are daily impositions. Second, the preparation is now enteric-coated, formulated to offer delayed release of active tiopronin. Lastly, Thiola EC can be taken with food which is an improvement to the inconvenient dosing regimen of Thiola, which can only be taken one hour before, or two hours after meals. These changes may affect compliance and side effect profiles compared to those of Thiola. In combination with potassium citrate or other alkali preparations, adverse GI effects are relatively common in actively-treated patients with cystinuria. It is possible that GI side effects may be reduced by Thiola EC.
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| Measure | Description | Time Frame |
|---|---|---|
| Wisconsin Stone Quality Of Life (WiSQoL) Score | The WiSQoL questionnaire is designed to understand the quality of life of patients with a history of kidney stones. There are 7 main questions that each have sub-questions. The questions are answered on a scale from 1 (very true / always or almost always) to 5 (not at all true / not at all, never). The total range of questions is 28-140; the lower the score, the worse the quality of life. | Baseline |
| Wisconsin Stone Quality Of Life (WiSQoL) Score | The WiSQoL questionnaire is designed to understand the quality of life of patients with a history of kidney stones. There are 7 main questions that each have sub-questions. The questions are answered on a scale from 1 (very true / always or almost always) to 5 (not at all true / not at all, never). The total range of questions is 28-140; the lower the score, the worse the quality of life. | Month 6 |
| Wisconsin Stone Quality Of Life (WiSQoL) Score | The WiSQoL questionnaire is designed to understand the quality of life of patients with a history of kidney stones. There are 7 main questions that each have sub-questions. The questions are answered on a scale from 1 (very true / always or almost always) to 5 (not at all true / not at all, never). The total range of questions is 28-140; the lower the score, the worse the quality of life. | Month 12 |
| Wisconsin Stone Quality Of Life (WiSQoL) Score | The WiSQoL questionnaire is designed to understand the quality of life of patients with a history of kidney stones. There are 7 main questions that each have sub-questions. The questions are answered on a scale from 1 (very true / always or almost always) to 5 (not at all true / not at all, never). The total range of questions is 28-140; the lower the score, the worse the quality of life. | Month 18 |
| Wisconsin Stone Quality Of Life (WiSQoL) Score |
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Inclusion Criteria:
Exclusion Criteria:
1. Unable to sign informed consent.
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Patients with cystinuria on Thiola EC.
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| Name | Affiliation | Role |
|---|---|---|
| David Goldfarb, MD | NYU Langone Health | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | 35294 | United States | ||
| NYU Langone Health |
Individual participant data that underlie the results reported in this article, after deidentification (text, tables, figures, and appendices) will be shared upon reasonable request.
Beginning 9 months and ending 36 months following article publication or as required by a condition of awards and agreements supporting the research.
The investigator who proposed to use the data, researchers who provide a methodologically sound proposal, and investigators whose proposed use of the data has been approved by an independent review committee ("learned intermediary") identified for this purpose will have access to the data upon reasonable request. Requests should be directed to david.goldfarb@nyulangone.org. To gain access, data requestors will need to sign a data access agreement.
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| ID | Term |
|---|---|
| D003555 | Cystinuria |
| ID | Term |
|---|---|
| D000608 | Renal Aminoacidurias |
| D015499 | Renal Tubular Transport, Inborn Errors |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
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The WiSQoL questionnaire is designed to understand the quality of life of patients with a history of kidney stones. There are 7 main questions that each have sub-questions. The questions are answered on a scale from 1 (very true / always or almost always) to 5 (not at all true / not at all, never). The total range of questions is 28-140; the lower the score, the worse the quality of life. |
| Month 24 |
| Short Form-36 Health Survey (SF-36v2) Score | Scales are standardized with a scoring algorithm or by the SF-36v2 scoring software to obtain a score ranging from 0 to 100. Higher scores indicate better health status. | Baseline |
| Short Form-36 Health Survey (SF-36v2) Score | Scales are standardized with a scoring algorithm or by the SF-36v2 scoring software to obtain a score ranging from 0 to 100. Higher scores indicate better health status. | Month 6 |
| Short Form-36 Health Survey (SF-36v2) Score | Scales are standardized with a scoring algorithm or by the SF-36v2 scoring software to obtain a score ranging from 0 to 100. Higher scores indicate better health status. | Month 12 |
| Short Form-36 Health Survey (SF-36v2) Score | Scales are standardized with a scoring algorithm or by the SF-36v2 scoring software to obtain a score ranging from 0 to 100. Higher scores indicate better health status. | Month 18 |
| Short Form-36 Health Survey (SF-36v2) Score | Scales are standardized with a scoring algorithm or by the SF-36v2 scoring software to obtain a score ranging from 0 to 100. Higher scores indicate better health status. | Month 24 |
| New York |
| New York |
| 10016 |
| United States |
| Duke University | Durham | North Carolina | 27708 | United States |
| University of Texas Southwestern | Dallas | Texas | 75390 | United States |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |