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Related Concept Videos

Renal Failure: Dose Adjustments01:11

Renal Failure: Dose Adjustments

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In patients with renal impairment, drugs undergo significant changes in their pharmacokinetics, which require dosage adjustments to ensure safe and effective therapy.
Reduced renal clearance and elimination rate are common outcomes of renal impairment. These alterations lead to a prolonged elimination half-life and an altered apparent volume of distribution for drugs. As a result, dosage adjustments are typically necessary to maintain optimal drug levels in the body.
However, dosage adjustments...
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Drug Dosing in Renal Diseases: Dose Adjustments Based on Drug Clearance and Elimination Rate Constant01:25

Drug Dosing in Renal Diseases: Dose Adjustments Based on Drug Clearance and Elimination Rate Constant

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In patients with renal disease, dosage adjustments are necessary to maintain therapeutic plasma drug concentrations and prevent toxicity or subtherapeutic exposure. Renal impairment alters drug pharmacokinetics, especially in conditions like uremia, where changes such as prolonged elimination half-life and altered apparent volume of distribution can significantly affect drug disposition. These changes require careful modification of the dosing regimen to achieve the desired clinical...
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Renal Drug Excretion: Tubular Reabsorption01:25

Renal Drug Excretion: Tubular Reabsorption

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Tubular reabsorption, a process occurring post-glomerular filtration of drugs in the renal tubule, is a critical determinant of drug half-life. During the process of renal excretion, as the glomerular filtrate progresses to the distal convoluted tubule (DCT), drugs that are highly permeable, lipophilic, and nonionized undergo passive reabsorption from the tubular fluid into the surrounding peritubular capillaries. This reabsorption process restricts their elimination through the kidneys. This...
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Renal Drug Excretion: Tubular Secretion01:28

Renal Drug Excretion: Tubular Secretion

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Active tubular secretion is a robust, energy-demanding process that utilizes carrier systems to transport drugs into renal tubules. The active renal secretion systems include the organic anion transporter (OAT) for weak acids and the organic cation transporter (OCT) for weak bases. Structurally similar drugs can compete for the same transporter, potentially leading to drug accumulation and toxicity. However, this principle can be exploited therapeutically. One example is probenecid (Probalan),...
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Continuous Renal Replacement Therapy01:30

Continuous Renal Replacement Therapy

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Continuous Renal Replacement Therapy, also known as CRRT, is a procedural treatment for acute kidney injury (AKI) that gradually removes uremic toxins and fluids while maintaining acid-base balance and stabilizing electrolytes. It is particularly useful for hemodynamically unstable patients. Unlike intermittent hemodialysis, which is faster, CRRT provides a gentler approach over 24 hours, closely mimicking the function of natural kidneys. However, CRRT is not ideal for patients with...
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Renal Regulation of Acid-Base Balance01:29

Renal Regulation of Acid-Base Balance

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Metabolic reactions in the body produce nonvolatile acids, such as sulfuric acid, which generate an acid load of approximately 1 mEq of H+ per kilogram of body weight daily. Excreting H+ in the urine is essential to balance this acid load.
In the kidneys, cells within the proximal convoluted tubules (PCT) and the collecting ducts secrete hydrogen ions (H+) into the tubular fluid. Specifically, in the PCT, Na+/H+ antiporters secrete H+ while reabsorbing Na+.
However, the intercalated cells in...
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Improved Home Blood Pressure Control by CT-guided Ozone-mediated Renal Denervation for Patients with Resistant Hypertension
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NTBC and Correction of Renal Dysfunction.

Arianna Maiorana1, Carlo Dionisi-Vici2

  • 1Metabolic Unit, Department of Pediatric Specialties, Bambino Gesù Children's Research Hospital, Rome, Italy. arianna.maiorana@opbg.net.

Advances in Experimental Medicine and Biology
|July 30, 2017
PubMed
Summary

Hereditary tyrosinemia type 1 (HT1) treatment with NTBC significantly improves liver and kidney function. Early diagnosis and treatment, especially via newborn screening, are crucial for optimal patient outcomes.

Keywords:
Fanconi syndromeNTBC therapyRenal tubular dysfunctionRickets

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Area of Science:

  • Biochemistry
  • Genetics
  • Pediatrics

Background:

  • Hereditary tyrosinemia type 1 (HT1) causes severe liver disease and kidney dysfunction, including Fanconi syndrome and hypophosphatemic rickets.
  • Animal models have been instrumental in understanding HT1 pathophysiology and NTBC treatment efficacy.

Purpose of the Study:

  • To evaluate the therapeutic effects of NTBC on liver and kidney function in Hereditary tyrosinemia type 1.
  • To assess the long-term renal outcomes associated with NTBC treatment.

Main Methods:

  • Utilized animal models to study HT1 pathophysiology.
  • Administered NTBC therapy and monitored liver and kidney function parameters.
  • Analyzed patient data based on age at treatment initiation.

Main Results:

  • NTBC therapy demonstrated significant improvements in renal tubular function, normalizing hypophosphatemia and proteinuria within weeks.
  • Both acute and chronic administration of NTBC proved effective in enhancing kidney function.
  • Treatment initiated at birth via newborn screening resulted in normal outcomes.

Conclusions:

  • NTBC therapy has transformed the prognosis of HT1, offering substantial short- and long-term renal benefits.
  • The timing of NTBC initiation is critical, with early diagnosis and treatment yielding the best results.