Study Highlights Experiences of Patients Treated with ADV7103 for Distal Renal Tubular Acidosis

According to a recent article, a new study is demonstrating how the effects of distal renal tubular acidosis impact a patient’s quality of life.

Distal Renal Tubular Acidosis (dRTA)

Distal renal tubular acidosis (dRTA) is a kidney disease that occurs when the kidneys don’t remove acids from the blood and dispose of them into the urine properly. The body’s normal functions produce acid, which the kidneys take from the blood and excrete into urine. When too much acid remains (acidosis), the blood becomes too acidic, which leads to imbalances and problems with cell functions. dRTA (also sometimes called classical dRTA) is the type 1 form of renal tubular acidosis. What differentiates it from other types is the “distal” part of its name. Distal means distant, and it refers to the point where the defect occurs in the tube of the kidney that forms urine. It’s relatively distant from the point where fluid from the blood enters the tiny tube (tubule) that collects fluid and wastes to form urine. The defect causes acid to build up in the blood. dRTA can occur on its own, but it is often a symptom of another disease.

Symptoms:

  • Low potassium levels
  • Stunted growth in children
  • Confusion or decreased alertness
  • Fatigue
  • Severe weakness
  • Kidney stones
  • Irregular heartbeat
  • Muscle cramps and pain
  • Pain in the back, abdomen, and/or bones
  • Decreased output of urine
  • Buildup of calcium in the kidneys
  • Paralysis
  • Progressive kidney and bone disease in adults

Treatments:

The major goal of therapy is to restore normal growth and prevent kidney stones. dRTA treatment includes, but is not limited to:

  • Correcting acidosis with medications like sodium bicarbonate and sodium citrate (also called alkali therapy)
  • Treating the underlying disease causing dRTA
  • Potassium supplements for newborns

The Study

The qualitative study examined the experiences of patients with dRTA, along with their caregivers, five years after being enrolled in a clinical study where they underwent treatment with ADV7103. ADV7103 is a prolonged release granule formulation that contains potassium citrate and potassium bicarbonate.

Patients diagnosed with dRTA can have numerous symptoms that will decrease their quality of life (QoL). Some of these symptoms involve issues with the growth of the kidney and bones, and occasionally even hearing loss.

The study was done through semi-structured interviews, each lasting for about an hour. There were 6 adult dRTA patients who were involved, and 13 pediatric patients diagnosed with dRTA. These interviews followed a guide which involved open-ended questions to help researchers understand how patients diagnosed with dRTA and treated with ADV7103 had their QoL impacted.

The Findings

Researchers discovered that the main areas of the patient’s lives that were affected were their education and work, social and family life, and emotional and physical well-being. These results were further examined to compare the differences between the patients’ initial standard of care (SoC) and the care they received after taking ADV7103.

Patients revealed that taking ADV7103 drastically changed their lives for the better. For the parents of children diagnosed with dRTA, treatment meant that they no longer had to spend grueling hours explaining their children’s condition and treatments to school administrators. Furthermore, these parents, a lot of whom had quit work to take care of their child’s treatment, were able to go back to working. In addition, it became easier for families to participate in family events and functions since the worries surrounding managing their child’s treatments were no longer a pressing concern.

For all patients, they admitted to a feeling of relief after no longer needing to answer tiring questions about their disease and their disease treatments. The emotional burden of their disease perception was relieved greatly in most patients.

Finally, patient’s shared that their gastrointestinal issues, along with their issues with taste, were improved following their treatment with ADV7103. This meant less hospitalizations for patients.

Compared to the SoC, the mean satisfaction score of patients treated with ADV7103 was 9 out of 10, with 10 being very satisfied. Moreover, ADV7103 met or exceeded the expectations of 14 patients out of the 17 studied.

What It Means

The results of the study proves that dRTA and treatments for the disease have a substantial impact on the patients’ and  their parents’ quality of life. In spite of this, the treatment ADV7103 lessens the burden of treatment stresses and can greatly improve the everyday life of patients and their families.

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