Abstract

To assess the therapeutic potential and the safety of intravitreous use of a bone marrow mononuclear fraction (BMMF) containing CD34+ cells in patients with Stargardt type macular dystrophy. The study was conducted on 10 patients with Stargardt dystrophy with worse eye visual acuity ≤ 20/125. A bone marrow aspirate was obtained from all patients, and after processing in the cell therapy center (CTC), 0.1 ml of the intravitreous BMMF suspension was injected into the eye with worse visual acuity. A sham injection was performed in the contralateral eye. The patients were evaluated at baseline and one, three, and six months after the injection. All of them were submitted to measurement of best corrected visual acuity (BCVA), microperimetry, multifocal electroretinography (mfERG) and full field electroretinography (ffERG), autofluorescence (AF), and optical coherence tomography (OCT). Fluorescein angiography was also performed before and six months after the injection. All patients completed the six-month period of evaluation. Mean visual acuity of the treated eye was 1.1 logMAR (20/250) before intravitreous (IV) injection, 0.96 logMAR (20/200+2) one month after injection, and 0.92 logMAR (20/160-1) 3 months after injection. In the untreated eye, mean VA was 1.0 logMAR (20/200) at baseline and 0.96 logMAR (20/200+2) and 0.94 logMAR (20/160-2) one and three months after injection, respectively. In the treated group, VA at baseline ranged from best acuity of 20/125-1 to worst acuity of 20/640+2, going through 20/100+2 and 20/400 during the first month. In the untreated group, BCVA ranged from 20/100+2 to 20/400 at baseline and from 20/100 to 20/400 after one month. The results for the treated group differed significantly at all follow-up times, whereas no significant difference was observed in the untreated group. Regarding the mean sensitivity of microperimetry, although there was improvement throughout all months, a significant difference occurred only during the first month. In the untreated eye, there was no significant difference in any analysis. Angiofluoresceinography did not reveal neovessel formation or tumor growth. The remaining exams were used in order to aid the diagnosis. The results indicate that the use of intravitreous BMMF in patients with Stargardt dystrophy is safe and is associated with a discrete improvement of BCVA and microperimetry in the treated eye compared to the untreated one.

Highlights

  • Hereditary retinal dystrophies (HRD) are characterized by progressive loss of photoreceptor and/or retinal pigment epithelium function, with a consequent early impairment of patient vision [1]

  • The clinical signs and symptoms are characterized by bilateral loss of central vision and dyschromatopsia, with mapping revealing macular atrophy, beaten bronze macular lesions, and whiteyellowish flecks corresponding to accumulation of lipofuscin at the retinal pigment epithelium (RPE) level [7,8,9,10]

  • A prospective, nonrandomized, open study of changes in visual acuity (VA), microperimetry, optical coherence tomography (OCT), and angiofluoresceinography induced by an intravitreous (IV) injection of bone marrow mononuclear fraction (BMMF) containing CD34 + cells was conducted on ten patients with Stargardt macular dystrophy

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Summary

Introduction

Hereditary retinal dystrophies (HRD) are characterized by progressive loss of photoreceptor and/or retinal pigment epithelium function, with a consequent early impairment of patient vision [1]. Among the HRD, Stargardt disease is the most common juvenile macular dystrophy and a frequent hereditary cause of central visual dysfunction in young patients, typically during the first and second decades of life [2, 3]. The prevalence of Stargardt disease is 1 : 8,000 to 1 : 10,000 [4]. The clinical signs and symptoms are characterized by bilateral loss of central vision and dyschromatopsia, with mapping revealing macular atrophy, beaten bronze macular lesions, and whiteyellowish flecks corresponding to accumulation of lipofuscin at the retinal pigment epithelium (RPE) level [7,8,9,10]. Visual acuity (VA) may vary from 20/30 up to 20/200

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