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Assessment of Health‐Related Quality of Life in Patients With Cystinuria on Tiopronin Therapy

Journal article
Published on December 13, 2019

Topics:

Nephrology Cystinuria
Contributors:
Modersitzki F, Goldfarb DS, Goldstein RL et al.
Name of Journal:
Urolithiasis


View Publication
DOI:
10.1007/s00240-019-01174-6
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Summary

A survey study assessed the quality of life (QoL) in patients with cystinuria receiving tiopronin compared to those not receiving tiopronin1


Background

Cystinuria is a rare genetic disorder leading to recurrent kidney stones.2 These stones affect daily and professional activities and reduce quality of life (QoL).3,4

Management of cystinuria includes lifestyle adaptations, such as increased fluid intake, as well as alkalinization of urine and prescription of cystine-binding thiol drugs (CBTD) such as tiopronin.1,5 Tiopronin in cystinuria reduces stone formation by lowering the cystine level in urine to increase solubility.5

There is limited evidence on the effect of tiopronin in cystinuria on health-related quality of life (HRQoL).1


Aim

The researchers investigated the impact of tiopronin in cystinuria on HRQoL.1


Approach

A survey was mailed to patients with cystinuria who were enrolled in two registries1:

  • Patients receiving tiopronin from the Thiola® Total Care Hub registry
  • Patients not receiving tiopronin from the Cystinuria Contact Registry at New York University School of Medicine

The survey included the following1:

  • Kidney stone experience
  • The short form-36 version 2 (SF-36v2) generic HRQoL questionnaire
  • The Wisconsin Stone QoL disease-specific questionnaire (WISQOL)

Information on treatment and underlying stone disease was collected.1 Analysis accounted for the time between respondents’ last stone events and their HRQoL assessments.1


Findings

In total, there were 312 respondents to the survey with 267 (85.6%) receiving tiopronin and 45 (14.6%) not receiving tiopronin1

The tiopronin-receiving group had more severe cystinuria.1 They had treatment with extracorporeal shock wave lithotripsy and higher1:

  • Mean numbers of kidney stone events
  • Related hospitalizations
  • Surgeries

Endocrine, musculoskeletal, neurologic, and skin comorbidities showed statistically significant differences between the groups.1

Use of pain medications was similar between the groups.1

Patients with cystinuria receiving tiopronin had better HRQoL measured by both the SF-36v2 and WISQOL1

Both groups had SF-36v2 scores below the US normative mean (score <50).1

Scores for the SF-36v2 questionnaire showed better HRQoL in patients receiving tiopronin compared with those who did not.1 Significant differences were seen for all domains except physical functioning.1 For the tiopronin and non-tiopronin groups, respectively, the scores were1:

  • Role physical: 47.2 versus 43.9 (P<0.001)
  • Bodily pain: 46.3 versus 42.3 (P<0.001)
  • General health: 45.1 versus 42.3 (P<0.001)
  • Vitality: 48.2 versus 45.3 (P<0.001)
  • Social functioning: 47.6 versus 43.9 (P<0.001)
  • Role emotional: 47.7 versus 45.3 (P<0.001)
  • Physical functioning: 49.3 versus 49.2 (P=0.359)
  • Physical component score: 49.5 versus 47.5 (P<0.001)
  • Mental component score: 46.9 versus 44.4 (P<0.001)

Despite fewer respondents and unequal variances, the WISQOL questionnaire also showed better HRQoL in patients receiving tiopronin compared with those who did not, with the following scores, respectively1:

  • Social impact: 77.6 versus 69.7 (P<0.001)
  • Emotional impact: 68.3 versus 60.0 (P<0.001)
  • Disease impact: 65.8 versus 59.3 (P<0.001)
  • Vitality impact: 67.5 versus 58.3 (P<0.001)

For both questionnaires, worse scores were associated with shorter time since the last stone event and the presence of more comorbidities.1


Key takeaway

Patients with cystinuria who were receiving tiopronin had better QoL than patients not receiving tiopronin.1 This observation may help inform clinical management when considering pharmacological treatment or surgery.1




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Footnotes

THIOLA (tiopronin) is a reducing and complexing thiol indicated, in combination with high fluid intake, alkali, and diet modification, for the prevention of cystine stone formation in adults and pediatric patients 20 kg and greater with severe homozygous cystinuria, who are not responsive to these measures alone.

CBTD, cystine-binding thiol drug; HRQoL, health-related quality of life; QoL, quality of life; SF-36v2, short form-36 version 2; WISQOL, Wisconsin Stone quality of life questionnaire.

  1. Modersitzki F et al. Urolithiasis. 2020;48(4):313-320.
  2. Streeper NM et al. J Endourol. 2017;31:S48-S53.
  3. Penniston KL and Nakada SY. Curr Opin Urol. 2016;26:50-55.
  4. Parr JM et al. BJU Int. 2015;116(suppl 3):31-35.
  5. Thiola® (tiopronin). Prescribing Information. San Antonio, TX: Mission Pharmacal Company. 01/2021.

MA-DS-24-0047 | December 2024