Sirolimus produced S-shaped effect on adult polycystic kidneys after 2-year treatment.

This double-blind trial followed 16 patients with autosomal dominant polycystic kidney disease (ADPKD) who received telmisartan or sirolimus plus telmisartan for 24 months. The 6-month pilot study showed a promising effect of sirolimus. The primary metric of this 2-year study was the change in total kidney volume at 12 and 24 months, as measured on magnetic resonance imaging. Secondary outcome was changes in renal function from the baseline at months 12 and 24. Among patients receiving sirolimus, the mean total kidney volume increased from 2845 mL to 3381 mL at 1 year and to 3901 mL at 2 years versus placebo values increasing from 2667 mL to 3680 mL and 3776 mL, respectively. The posttreatment mean total kidney volume increased less on sirolimus (P = .07) versus control therapy (P = .05) after 1 year, but there was no difference at 24 months. Kidney volume was stable on sirolimus to 12 months, increasing steadily to 24 months. In contrast, kidney volume increased steadily among patients on telmisartan alone both at 12 and 24 months. In conclusion, sirolimus appeared to retard kidney growth among patients with ADPKD during the first 6 months of therapy but not to halt growth thereafter, thus eliciting S-shaped effect. The dose of sirolimus (1 mg per day) was associated with a low rate of side effects similar those observed in kidney transplantation.

Sirolimus therapy for patients with adult polycystic kidney disease: a pilot study.

A pilot study was performed on adult polycystic kidney disease (PCKD) patients to examine the effects of the anti-proliferative mammalian target of rapamycin inhibitor sirolimus on the growth of renal cysts. Eight consecutive PCKD patients were given sirolimus (1 mg/d PO) for 6 consecutive months, in addition to an angiotensin receptor blocker (ARB), namely telmisartan. Another 8 PCKD patients served as a control group given only telmisartan. All PCKD patients had a serum creatinine value <2 mg/dL with a negative urine culture before enrollment. All patients were diagnosed by renal magnetic resonance imaging (MRI) to measure renal volumes. After a 6-month follow-up, patients were rescanned to remeasure the MRI volumes. Renal function was stable in 5/8 subjects in the sirolimus group, improved in 2 cases, and worsened in 1 with an increase of serum creatinine to >2 mg/dL resulting in his withdrawal after 5 months of follow-up. In contrast, the serum creatinine value was stable in 3 control group subjects, worsen in 3, and improved in 2. Four patients in the sirolimus group experienced infectious complications, namely, urinary tract infections (UTI) in 2 which were treated with antibiotics, and monilial pharyngitis in 2, who were treated and cured with a topical antifungal. In the control group, only 2 developed and were treated for UTIs. Hematologic tests were normal in all patients. There was an insignificant rise in kidney volume as measured by MRI in the sirolimus group (2845 vs 3221 mL after 6 months; P = NS) compared with a significant increase in the control group (2667 vs 3590 mL after 6 months; P < .05). We concluded that sirolimus, in addition to an ARB, might be beneficial for PCKD patients who present early in their illness.

The combined posterior temporoparietal and galeal fascial flap: a new flap in the elevation of the constructed auricle (second stage of microtia correction).

Over the past few years, the technique of elevating the buried ear framework in the second stage of microtia correction has shifted from skin grafting to the use of flaps and cartilage blocks in the retroauricular sulcus. While the temporoparietal fascial flap should be reserved for secondary procedures and the treatment of complications, the mastoid fascial flap is inadequate by itself and needs an additional cartilage graft. Here, we describe a new flap, the combined posterior temporoparietal and galeal fascial flap, for the elevation of the buried ear cartilage. The flap is robust, with a dependable blood supply based on the posterior branches of the superficial temporal artery. In four cases the flap was rolled up and inset into the retroauricular sulcus, while in three cases an additional conchal cartilage graft was inserted into the roll. All the patients had satisfactory ear projection at follow-up 10-14 months postoperatively. We discuss the surgical technique and the advantages of this flap. We believe that this new flap, which has not been described before, has the potential to replace other flaps in the second stage of microtia correction.