Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| University of Texas | OTHER |
| Wake Forest University Health Sciences | OTHER |
| University of Miami | OTHER |
| University of Washington |
Not provided
Not provided
Not provided
Not provided
This is an observational study collecting patient/caregiver reports on suspected medication/drug-induced acute porphyria attacks, as well as safe use of drugs previously labeled "unsafe" or with unknown risk. Participants will be recruited through the RDCRN Contact Registry for the Porphyrias Consortium. The study will be advertised on the Consortium website and through the American Porphyria Foundation's social media network.
Contact Registrants will be sent an email invitation describing the study. A unique link will be generated for each registrant and included in the email invitation. After 2 weeks, a second email invitation will be sent to the registrants who have not yet participated. After 1 month, a third email invitation will be sent to the registrants who have not yet participated. We may send additional emails periodically (e.g. every 6-12 months) to subjects enrolled to remind them to complete new surveys if they have taken any other medications of interest or have experienced any new reactions/attacks.
If the registrant wants to participate in the study, he/she will follow the survey link in the email invitation, which directs him/her to an IRB-approved online consent form. If the participant consents to participate, he/she will be directed to online forms to report general information (ex. age, sex, test results, family history), suspected medication-related acute attacks, and "uneventful use of possibly risky medication". If a patient has multiple medication-related acute attacks, the patient/caregiver will be able to submit multiple report forms that will be linked. In addition to the report forms, patients will be asked to review a list of "Medications of Interest" and report current or past use.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Web-based Survey | Other | Participants will be directed to online forms to report general information (ex. age, sex, test results, family history), suspected medication-related acute attacks, and "uneventful use of possibly risky medication". If a patient has multiple medication-related acute attacks, the patient/caregiver will be able to submit multiple report forms that will be linked. In addition to the report forms, patients will be asked to review a list of "Medications of Interest" and report current or past use. |
| Measure | Description | Time Frame |
|---|---|---|
| Acute Attack Form | Designed to capture PBG levels, characteristics of the acute attack, timing of medication administration, and pharmacological information on the medication. | 5 years from study start |
| Uneventful Use of Possible Risky Medication Form | Designed to capture details of medication in question, ALA/PGB results during use of medication in questions, concomitant medication use, diet history, and alcohol use. | 5 years from study start |
| Use of Medications of Interest Form | Designed to assess medications used by category and effects of each. | 5 years from study start |
Not provided
Not provided
Inclusion Criteria:
There are no restrictions on age, sex or ethnicity. However, the following criteria will be applied to those reports that are considered evaluable (analyzed and scored):
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Participants will be recruited through the RDCRN Contact Registry for the Porphyrias Consortium.
Not provided
| Name | Affiliation | Role |
|---|---|---|
| D. Montgomery Bissell, MD | University of California, San Francisco | Study Chair |
| Bruce Wang, MD | University of California, San Francisco | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of South Florida Data Management Coordinating Center | Tampa | Florida | 33612 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 15767622 | Background | Anderson KE, Bloomer JR, Bonkovsky HL, Kushner JP, Pierach CA, Pimstone NR, Desnick RJ. Recommendations for the diagnosis and treatment of the acute porphyrias. Ann Intern Med. 2005 Mar 15;142(6):439-50. doi: 10.7326/0003-4819-142-6-200503150-00010. | |
| 4868613 | Background | de Matteis F. Disturbances of liver porphyrin metabolism caused by drugs. Pharmacol Rev. 1967 Dec;19(4):523-57. No abstract available. |
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D011164 | Porphyrias |
| D046350 | Porphyria, Variegate |
| D046349 | Coproporphyria, Hereditary |
| D017118 | Porphyria, Acute Intermittent |
| ID | Term |
|---|---|
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D017094 | Porphyrias, Hepatic |
| D008107 | Liver Diseases |
Not provided
Not provided
| OTHER |
| Icahn School of Medicine at Mount Sinai | OTHER |
| University of Alabama at Birmingham | OTHER |
| University of California, San Francisco | OTHER |
| University of Utah | OTHER |
| National Institutes of Health (NIH) | NIH |
Not provided
Not provided
Not provided
| 17763398 | Background | Seth AK, Badminton MN, Mirza D, Russell S, Elias E. Liver transplantation for porphyria: who, when, and how? Liver Transpl. 2007 Sep;13(9):1219-27. doi: 10.1002/lt.21261. |
| 1244784 | Background | Slavin SA, Christoforides C. Thiopental administration in acute intermittent porphyria without adverse effect. Anesthesiology. 1976 Jan;44(1):77-9. doi: 10.1097/00000542-197601000-00021. No abstract available. |
| 17578481 | Background | Thunell S, Pomp E, Brun A. Guide to drug porphyrogenicity prediction and drug prescription in the acute porphyrias. Br J Clin Pharmacol. 2007 Nov;64(5):668-79. doi: 10.1111/j.0306-5251.2007.02955.x. Epub 2007 Jun 19. |
| D004066 | Digestive System Diseases |
| D012873 | Skin Diseases, Genetic |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |