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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

39,720 - 49,650

US Estimated

61,620 - 77,025

Europe Estimated

Age of Onset

ICD-10

N04.1

Inheritance

This condition does not appear to have a clear pattern of inheritance.

Rare View

FSGS is a rare disease that attacks the glomeruli and causes serious scarring, leading to permanent kidney damage and even kidney failure. FSGS is one of the causes of a serious condition known as nephrotic syndrome. FSGS causes asymptomatic proteinuria or nephrotic syndrome, and usualy results in progressive kidney injury. The most common presenting symptom is edema.

5 Facts you should know

FACT

1

It is characterized by scar tissue that forms in some of the glomeruli in the kidney

FACT

2

Key signs and symptoms include proteinuria, water retention, and edema

FACT

3

Diagnosis is established by renal biopsy

FACT

4

FSGS occurs more frequently in adults than in children and is most prevalent in adults aged 45 and older

FACT

5

Black Americans are at least four times more likely to get FSGS in comparison with white Americans

Focal segmental glomerulosclerosis (FSGS) is also known as...

Focal segmental glomerulosclerosis (FSGS) is also known as:

  • FSGS
  • Focal glomerular sclerosis
  • Focal nodular glomerulosclerosis

What’s your Rare IQ?

Which of these symptoms in most likely to be seen in a patient with FSGS?

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

TitleDescriptionPhasesStatusInterventionsMore Information
Obinutuzumab in Primary FSGSThe purpose of this study is to evaluate the safety and efficacy of Obinutuzumab in inducing complete or partial remission of proteinuria.Phase 2RecruitingDrug: ObinutuzumabMore Info
Tumor Necrosis Factor Inhibition in Focal Segmental Glomerulosclerosis and Treatment Resistant Minimal Change DiseaseThe 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 2RecruitingDrug: adalimumabMore Info
Use of Acthar in Patients With FSGS That Will be Undergoing Renal TransplantationThis study will evaluate the use of Acthar in patients to undergo renal transplantation and will measure the rate of FSGS recurrence.Phase 3RecruitingDrug: ActharMore Info
PRI-VENT FSGS: Preemptive Rituximab to Prevent Recurrent Focal Segmental Glomerulosclerosis Post-TransplantThis 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 3RecruitingDrug: Rituximab|Procedure: PlasmapheresisMore 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 2RecruitingDrug: PF-06730512More Info
A Study of TRPC5 Channel Inhibitor in Patients With Diabetic Nephropathy, Focal Segmental Glomerulosclerosis, and Treatment-Resistant Minimal Change DiseaseThis 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 2RecruitingDrug: GFB-887|Drug: PlaceboMore Info
Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular DiseasesTo 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 2RecruitingDrug: SparsentanMore Info
Atrasentan in Patients With Proteinuric Glomerular DiseasesThe 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 2RecruitingDrug: AtrasentanMore Info

Top Treatments in Research

AgentClass/Mechanism of ActionDevelopment StatusCompanyClinical StudiesMore Information
ObinutuzumabHumanized anti-CD20 monoclonal antibodyPhase 2Mayo ClinicMore InfoMore Info
adalimumabAdalimumab is a monoclonal antibody to human tumor necrosis factor (TNF) alphaPhase 2University of MichiganMore InfoMore Info
ActharACTH 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 3University of Colorado, DenverMore InfoMore Info
Rituximab/PlasmapheresisThe antibody binds to the cell surface protein CD20Phase 3University of MinnesotaMore InfoMore Info
PF-06730512An Fc fusion protein that targets the ROBO2/SLIT2 pathwayPhase 2PfizerMore InfoMore Info
GFB-887GFB-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 pathwayPhase 2Goldfinch Bio, Inc.More InfoMore Info
SparsentanSparsentan, 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 3Travere Therapeutics, Inc.More InfoMore Info
AtrasentanAtrasentan 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 2Chinook Therapeutics U.S., Inc.More InfoMore Info