“Currently, no medical therapy has been shown to be beneficial in PSC. A 2-year randomized, controlled trial using UDCA at a dose of 12 to 15 mg/kg/d in patients with PSC was associated with improved liver tests, however there was no beneficial effect on survival, liver histology, cholangiographic appearance or symptoms. 26 Subsequently, studies testing higher doses of UDCA (between 17 and 23 mg/kg/d) showed trends towards improved survival but did not reach statistical significance. Recently, a 5-year randomized controlled trial of UCDA at 28 to 30 mg/kg/d demonstrated that high dose UDCA was associated with improved liver tests but did not improve survival and was associated with higher rates of serious adverse events. Based on these results, high dose UDCA (25 to 30 mg/kg/d) cannot be recommended in patients with PSC. Consideration of medical therapy for patients with PSC in the setting of prospective studies is reasonable, but there is not treatment that can be recommended at this time”. Medical management of PSC is therefore limited to complications that arise during the course of the disease. Most of these complications and treatments are similar to those listed earlier for the management of PBC.
Primary Immune Deficiency Treatment Consortium (PIDTC) Scientific Workshop
Thursday, April 7, 2011 -
Saturday, April 9, 2011
Location: San Francisco, CA Description: This was a 3-day meeting. Participation at the workshop was by invitation and included representatives from each of the centers participating at PIDTC as well as representatives of CIBMTR, USIDNET, NIAID, ORDR, EBMT, and ESID. This was meant to favor collaboration and promote international collaborative trials in the field of these rare disorders. Special attention was paid to invitation of young investigators at a senior stage in their training or at the beginning of their academic careers. The meeting was open to representatives of the patient advocacy groups that are active in the field of primary immune deficiencies (PIDs) with the intent of promoting communication and collaboration. The results of the meeting will be published in a peer-reviewed journal.