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Focal segmental glomerulosclerosis (FSGS)
Focal segmental glomerulosclerosis is scattered mesangial sclerosis that begins in some glomeruli and eventually involves all glomeruli
Prevalence
12-15/100,000
Age of Onset
ICD-10
N04.1
Inheritance
Autosomal dominant
Autosomal recessive
Mitochondrial/Multigenic
X-linked dominant
X-linked recessive
5 Facts you should know
FACT
It is characterized by scar tissue that forms in some of the glomeruli in the kidney
FACT
Key signs and symptoms include proteinuria, water retention, and edema
FACT
Diagnosis is established by renal biopsy
FACT
FSGS occurs more frequently in adults than in children and is most prevalent in adults aged 45 and older
FACT
Black Americans are at least four times more likely to get FSGS in comparison with white Americans
Interest over time
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Common signs & symptoms
The signs and symptoms of FSGS can vary depending on the severity of the condition, but common signs and symptoms include:
Proteinuria
Which may be quantified as protein to creatinine ratio in a random urine sample, or a 24-hour urine collection.
Hematuria
Often seen microscopically in urinalysis, but could be seen macroscopically as well.
Nephrotic syndrome
This includes: Hypoalbuminemia, hypercholesterolemia and lipiduria, as well as edema due to decreased plasma oncotic pressure.
Hypertension
Which may be secondary to kidney disease or the underlying cause of FSGS.
FSGS may present with non-specific symptoms such as general malaise, weakness, and fatigue. It's important to keep in mind that many of these symptoms are non-specific, and could occur with other types of kidney disease or other illnesses.
A definitive diagnosis of FSGS requires renal biopsy. This is essential as FSGS can present with different histological subtypes, each with different prognosis and response to treatment.
In some cases, FSGS can be idiopathic, while in others, it can be secondary to other underlying condition such as viral infections, genetic disorders, or chronic kidney diseases. A thorough medical history, physical examination and laboratory tests such as urinalysis, serum creatinine, complete blood count, electrolytes, lipid profile and urinalysis are important to identify underlying conditions and risk factors.
Sources:
https://www.uptodate.com/contents/focal-segmental-glomerulosclerosis-in-adults
https://www.ncbi.nlm.nih.gov/books/NBK539276/
https://www.kidney-international.org/article/S0085-2538(20)30141-1/fulltext
Current treatments
There are several treatment options available for FSGS, including medications, kidney transplantation, and in some cases, dialysis.
Steroids (e.g. prednisone, prednisolone)
Used to reduce inflammation and help slow down the progression of FSGS.
Immunosuppressants (e.g. Cyclosporine, tacrolimus)
Used to suppress the immune system and prevent further damage to the kidneys.
ACE inhibitors and Angiotensin receptor blockers (ARBs)
These drugs are used to lower blood pressure and prevent proteinuria (excess protein in the urine). Brand name examples of ACE inhibitors: benazepril (Lotensin), captopril (Capoten), and lisinopril (Prinivil, Zestril). Brand name examples of ARBs : losartan (Cozaar), valsartan (Diovan), telmisartan (Micardis).
Kidney transplantation
In cases where FSGS has caused significant damage to the kidneys, a kidney transplant may be necessary. During a kidney transplant, a healthy kidney from a donor is placed in the body of the person with FSGS.
Dialysis
In severe cases, when the kidneys have lost most of their ability to function, dialysis may be required to remove waste products and excess fluid from the body.
It is important to note that these treatments may vary depending on the type of FSGS and the specific needs of the patient. Consultation with the patient's Nephrologist or specialist is recommended to plan the optimal therapy.
Sources:
https://www.niddk.nih.gov/health-information/kidney-disease/focal-segmental-glomerulosclerosis-fsgs
https://www.kidney.org/atoz/content/focal-segmental-glomerulosclerosis-fsgs
Top Clinical Trials
| Title | Description | Phases | Status | Interventions | More Information |
|---|---|---|---|---|---|
| Obinutuzumab in Primary FSGS | The purpose of this study is to evaluate the safety and efficacy of Obinutuzumab in inducing complete or partial remission of proteinuria. | Phase 2 | Recruiting | Drug: Obinutuzumab | More Info |
| Tumor Necrosis Factor Inhibition in Focal Segmental Glomerulosclerosis and Treatment Resistant Minimal Change Disease | The researchers are testing adalimumab, a treatment which blocks tumor necrosis factor (TNF), to see if it changes levels of urine biomarker levels (TIMP1 and MCP1). | Phase 2 | Recruiting | Drug: adalimumab | More Info |
| Use of Acthar in Patients With FSGS That Will be Undergoing Renal Transplantation | This study will evaluate the use of Acthar in patients to undergo renal transplantation and will measure the rate of FSGS recurrence. | Phase 3 | Recruiting | Drug: Acthar | More Info |
| PRI-VENT FSGS: Preemptive Rituximab to Prevent Recurrent Focal Segmental Glomerulosclerosis Post-Transplant | This is a phase III, multicenter, randomized, open label, clinical trial to test that hypothesis that plasmapheresis plus rituximab prior to kidney transplantation can prevent recurrent FSGS in children and adults. | Phase 3 | Recruiting | Drug: Rituximab|Procedure: Plasmapheresis | More Info |
| A Study to Evaluate PF-06730512 in Adults With Focal Segmental Glomerulosclerosis (FSGS) | The purpose of this Phase 2 adaptive study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of PF-06730512 following multiple intravenous infusions in adult subjects with FSGS. | Phase 2 | Recruiting | Drug: PF-06730512 | More Info |
| A Study of TRPC5 Channel Inhibitor in Patients With Diabetic Nephropathy, Focal Segmental Glomerulosclerosis, and Treatment-Resistant Minimal Change Disease | This is a phase 2a study evaluating the safety and tolerability of multiple ascending doses of GFB-887 in patients with diabetic nephropathy (DN), focal segmental glomerulosclerosis (FSGS), and treatment-resistant minimal change disease (TR-MCD). | Phase 2 | Recruiting | Drug: GFB-887|Drug: Placebo | More Info |
| Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases | To evaluate the safety, efficacy and tolerability of sparsentan oral suspension and assess changes in proteinuria after once-daily dosing over the 108-week treatment period. | Phase 2 | Recruiting | Drug: Sparsentan | More Info |
| Atrasentan in Patients With Proteinuric Glomerular Diseases | The AFFINITY Study is a phase 2, open-label, basket study to evaluate the efficacy and safety of atrasentan in patients with proteinuric glomerular disease who are at risk of progressive loss of renal function. | Phase 2 | Recruiting | Drug: Atrasentan | More Info |
Top Treatments in Research
| Agent | Class/Mechanism of Action | Development Status | Company | Clinical Studies | More Information |
|---|---|---|---|---|---|
| Obinutuzumab | Humanized anti-CD20 monoclonal antibody | Phase 2 | Mayo Clinic | More Info | More Info |
| adalimumab | Adalimumab is a monoclonal antibody to human tumor necrosis factor (TNF) alpha | Phase 2 | University of Michigan | More Info | More Info |
| Acthar | ACTH is an important component of the hypothalamic-pituitary-adrenal axis and is often produced in response to biological stress (along with its precursor corticotropin-releasing hormone from the hypothalamus). Its principal effects are increased production and release of cortisol by the cortex of the adrenal gland. | Phase 3 | University of Colorado, Denver | More Info | More Info |
| Rituximab/Plasmapheresis | The antibody binds to the cell surface protein CD20 | Phase 3 | University of Minnesota | More Info | More Info |
| PF-06730512 | An Fc fusion protein that targets the ROBO2/SLIT2 pathway | Phase 2 | Pfizer | More Info | More Info |
| GFB-887 | GFB-887 is a precision-based, podocyte-targeting, small molecule inhibitor of TRPC5, designed specifically to treat individuals with kidney diseases associated with an over-activation of the TRPC5-Rac1 pathway | Phase 2 | Goldfinch Bio, Inc. | More Info | More Info |
| Sparsentan | Sparsentan, which possesses two clinically validated mechanisms of action in a single molecule, works by selectively blocking the action of two potent vasoconstrictor and mitogenic agents, angiotensin II (AII) and endothelin 1 (ET1), at their respective receptors. Sparsentan is highly selective (10,000-fold) for the AII receptor sub-type 1 and the ET receptor sub-type A. As such, Sparsentan combines the properties of an angiotensin receptor blocker (ARB) and an endothelin receptor antagonist (ERA) in the same molecule. | Phase 3 | Travere Therapeutics, Inc. | More Info | More Info |
| Atrasentan | Atrasentan is a selective and potent inhibitor of the endothelin A receptor, or ETA, which has the potential to provide benefit in multiple chronic kidney diseases by reducing proteinuria and having direct anti-inflammatory and anti-fibrotic effects to preserve kidney function. | Phase 2 | Chinook Therapeutics U.S., Inc. | More Info | More Info |
