Peer-reviewed veterinary case report
Gene therapy to treat anemia in cats with chronic kidney disease
By Vaden, Shelly L et al.·Published in Journal of veterinary internal medicine·2023·Department of Clinical Sciences, United States·View original on PubMed →
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Original publication title: Adeno-associated virus-vectored erythropoietin gene therapy for anemia in cats with chronic kidney disease.
- Species:
- cat
Plain-English summary
A group of 23 cats with chronic kidney disease (CKD) and anemia were treated with a new gene therapy called SB-001, which helps increase red blood cell production. After receiving the treatment, 86% of the cats showed improvement in their anemia within 70 days, with significant increases in their packed cell volume (PCV), a measure of red blood cells. While most cats tolerated the treatment well, some developed high blood pressure and related issues. Overall, SB-001 appears to be a promising option for managing anemia in cats with CKD.
People also search for: cat anemia treatment · chronic kidney disease in cats · SB-001 gene therapy for cats
Abstract
BACKGROUND: A treatment of chronic kidney disease (CKD)-associated anemia in cats is needed. SB-001 is an adeno-associated virus-vectored (AAV)-based gene therapeutic agent that is administered intramuscularly, causing the expression of feline erythropoietin. HYPOTHESIS/OBJECTIVE: We hypothesized that SB-001 injection would lead to a sustained increase in PCV in cats with CKD-associated anemia. ANIMALS: Twenty-three cats with International Renal Interest Society (IRIS) Stage 2 to 4 CKD-associated anemia were enrolled at 4 veterinary clinics. METHODS: In a prospective clinical trial, cats were treated with 1 of 3 regimens of SB-001 (Lo 1.2 × 10genome copies [GCs] on Day 0; Lo ± Hi [supplemental 2nd dose of 3.65 × 10GC on Day 42]; Hi 3.65 × 10GC IM on Day 0) and followed for 70 days. RESULTS: A response to SB-001 at any time between Day 28 and Day 70 was seen in 86% (95% confidence interval 65, 97%) of all cats. There was a significant (P < .003) increase in PCV from Day 0 to Day 28 (mean increase 6 ± 6 percentage points [pp]; n = 21), Day 42 (8 ± 9 pp; n = 21), Day 56 (10 ± 11 pp; n = 17), and Day 70 (13 ± 14 pp, n = 14). Twelve cats were hypertensive at baseline, 4 of which developed encephalopathy during the study. An additional 6 cats became hypertensive during the study. CONCLUSIONS AND CLINICAL IMPORTANCE: Results of this study suggest that SB-001 therapy represents a suitable single injection treatment that can address nonregenerative anemia in cats with CKD. It was generally well tolerated; however, hypertension and encephalopathy developed in some cats as previously described in association with erythropoiesis-stimulating agent therapy.
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Search related cases →Original publication on PubMed: https://pubmed.ncbi.nlm.nih.gov/37847024/