Hydroxychloroquine Ameliorates Hematuria in Children with X-Linked Alport Syndrome: Retrospective Case Series Study

Abstract

Purpose

As a rare collagen type IV hereditary kidney disease, X-linked Alport syndrome (XLAS) is the most common form of Alport syndrome, the prevalence of which is estimated at 1:10,000 of the population, four times higher than the prevalence rate of autosomal recessive Alport syndrome. To describe a series of eight XLAS children with persistent hematuria and proteinuria and the clinical outcomes after hydroxychloroquine (HCQ) treatment to assess its efficacy as early intervention.

Patients and Methods

The study retrospectively analysed 8 patients with persistent hematuria and proteinuria at different onset ages who were diagnosed with XLAS and been treated with HCQ. The urinary erythrocyte count, urinary albuminn were measured. Descriptive statistics were used to estimate the patients’ responses to HCQ treatment after one month, three months, and six months.

Results

After the first month, the three months, and the six months of HCQ treatment, the urinary erythrocyte counts of four, seven, and eight children were significantly reduced; the decreasing proteinuria was found in two, four, and five children. Only one child with increasing proteinuria was found after 1-month HCQ treatment. This proteinuria was maintained after 3-month HCQ treatment but decreased to minor after 6-month HCQ treatment.

Conclusion

We present the first potential efficacy of HCQ treatment in XLAS with hematuria and persistent proteinuria. It suggested that HCQ could be an effective treatment to ameliorate hematuria and proteinuria.

Keywords:

• X-linked Alport syndrome
• hematuria
• proteinuria
• hydroxychloroquine
• therapy

Declaration

This study complies with the Declaration of Helsinki.

Consent to Publish

The participants have consented to the submission of the study to the journal.

Acknowledgments

All phases of this study were supported by the Shanghai municipal commission of science and technology (grant no. 19ZR1442300) and Shanghai Children’s Hospital (grant no. 2021YQ04, 2021YGZQ06). We are grateful for the support from the patients and their family members.

Disclosure

The authors report no conflicts of interest in this work.