Quality of life in patients with retinitis pigmentosa submitted to intravitreal use of bone marrow-derived stem cells (Reticell -clinical trial)

Rubens C Siqueira, Andre Messias, Katharina Messias, Rafael S Arcieri, Milton A Ruiz, Neiglene F Souza, Lia C Martins, Rodrigo Jorge, Rubens C Siqueira, Andre Messias, Katharina Messias, Rafael S Arcieri, Milton A Ruiz, Neiglene F Souza, Lia C Martins, Rodrigo Jorge

Abstract

Introduction: Retinitis pigmentosa (RP) is a severe neurodegenerative disease of the retina that can lead to blindness. Even without treatment, a clinical study with the use of stem cells is currently underway and the results are being evaluated. In the present report we assess the vision-related quality of life in patients with RP submitted to intravitreal use of bone marrow-derived stem cells.

Method: The study included 20 patients with RP submitted to intravitreal use of bone marrow-derived stem cells. We evaluate the vision-related quality of life (VRQOL) of patients using the National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25). Patients were scheduled to answer the questionnaire before treatment and 3 and 12 months after treatment.

Results: All patients completed the survey as scheduled. There was a statistically significant improvement (P<0.05) in the quality of life of patients 3 months after treatment, whereas by the 12th month there was no statistically significant difference from baseline.

Conclusions: Cell therapy with intravitreal use of bone marrow-derived stem cells can improve the quality of life of patients with RP, although the improvement is lost with time. A larger number of cases will be necessary to evaluate the repercussions of this therapy on the quality of life of these patients.

Trial registration: Clinicaltrials.gov: NCT01560715 . Registered March 19, 2012.

Figures

Figure 1
Figure 1
Quality of life behaviour of all patients submitted to cell therapy.
Figure 2
Figure 2
Average quality of life behaviour of all patients submitted to cell therapy.

References

    1. Delyfer MN, Léveillard T, Mohand-Saïd S, Hicks D, Picaud S, Sahel JA. Inherited retinal degenerations: therapeutic prospects. Biol Cell. 2004;96:261–9. doi: 10.1111/j.1768-322X.2004.tb01414.x.
    1. Hartong DT, Berson EL, Dryja TP. Retinitis pigmentosa. Lancet. 2006;368:1795–809. doi: 10.1016/S0140-6736(06)69740-7.
    1. Berson EL, Rosner B, Sandberg MA, Hayes KC, Nicholson BW, Weigel-DiFranco C, et al. A randomized trial of vitamin A and vitamin E supplementation for retinitis pigmentosa. Arch Ophthalmol. 1993;111:761–72. doi: 10.1001/archopht.1993.01090060049022.
    1. Berson EL, Rosner B, Sandberg MA, Weigel-DiFranco C, Moser A, Brockhurst RJ, et al. Clinical trial of docosahexaenoic acid in patients with retinitis pigmentosa receiving vitamin A treatment. Arch Ophthalmol. 2004;122:1297–305. doi: 10.1001/archopht.122.9.1297.
    1. Li T, Sandberg MA, Pawlyk BS, Rosner B, Hayes KC, Dryja TP, et al. Effect of vitamin A supplementation on rhodopsin mutants threonine-17/methionine and proline-347/serine in transgenic mice and in cell cultures. Proc Natl Acad Sci U S A. 1998;95:11933–8. doi: 10.1073/pnas.95.20.11933.
    1. Clemson CM, Tzekov R, Krebs M, Checchi JM, Bigelow C, Kaushal S. Therapeutic potential of valproic acid for retinitis pigmentosa. Br J Ophthalmol. 2011;95:89–93. doi: 10.1136/bjo.2009.175356.
    1. Siqueira RC. Stem cell therapy for retinal diseases: update. Stem Cell Res Ther. 2011;2:50. doi: 10.1186/scrt91.
    1. Siqueira RC, Messias A, Voltarelli JC, Scott IU, Jorge R. Intravitreal injection of autologous bone marrow-derived mononuclear cells for hereditary retinal dystrophy: a phase I trial. Retina. 2011;31:1207–14. doi: 10.1097/IAE.0b013e3181f9c242.
    1. Siqueira RC. Autologous bone marrow-derived stem cells transplantation for retinitis pigmentosa (RETICELL). Clinicaltrial identifier: NCT01560715 [Internet]. Bethesda (MD): National Library of Medicine, 2012, Available from: (cited 15 April 2012).
    1. Mangione CM, Lee PP, Pitts J, Gutierrez P, Berry S, Hays RD. Psychometric properties of the National Eye Institute Visual Function Questionnaire (NEI-VFQ). NEI-VFQ Field Test Investigators. Arch Ophthalmol. 1998;116:1496–504. doi: 10.1001/archopht.116.11.1496.
    1. Mangione CM, Lee PP, Gutierrez PR, Spritzer K, Berry S, Hays RD. Development of the 25-item National Eye Institute Visual Function Questionnaire. Arch Ophthalmol. 2001;119:1050–8. doi: 10.1001/archopht.119.7.1050.
    1. Messias K, Jägle H, Saran R, Ruppert AD, Siqueira R, Jorge R, et al. Psychophysically determined full-field stimulus thresholds (FST) in retinitis pigmentosa: relationships with electroretinography and visual field outcomes. Doc Ophthalmol. 2013;127:123–9. doi: 10.1007/s10633-013-9393-y.
    1. Siqueira RC, Messias A, Voltarelli JC, Messias K, Arcieri RS, Jorge R. Fixation stability and central retinal sensitivity after intravitreal autologous bone-marrow stem cells for hereditary retinal dystrophy. In: ARVO, 2012, Fort Lauderdale. ARVO Meeting Abstracts, vol. 53. 2012. p. 6432.
    1. Arcieri RS, Messias K, Castro VM, Siqueira RC, Jorge R, Messias A. Intravitreal autologous bone-marrow stem cells in retinitis pigmentosa patients: one-year results. ARVO 2013. Invest Ophthalmol Vis Sci. 2013;54: E-Abstract 643.
    1. Seo JH, Yu HG, Lee BJ. Assessment of functional vision score and vision-specific quality of life in individuals with retinitis pigmentosa. Korean J Ophthalmol. 2009;23:164–8. doi: 10.3341/kjo.2009.23.3.164.
    1. Sugawara T, Sato E, Baba T, Hagiwara A, Tawada A, Yamamoto S. Relationship between vision-related quality of life and microperimetry-determined macular sensitivity in patients with retinitis pigmentosa. Jpn J Ophthalmol. 2011;55:643–6. doi: 10.1007/s10384-011-0080-9.
    1. Park SS, Caballero S, Bauer G, Shibata B, Roth A, Fitzgerald PG, et al. Long-term effects of intravitreal injection of GMP-grade bone-marrow-derived CD34+ cells in NOD-SCID mice with acute ischemia-reperfusion injury. Invest Ophthalmol Vis Sci. 2012;53:986–94. doi: 10.1167/iovs.11-8833.

Source: PubMed

Patrocinadores e Colaboradores

Condições médicas

Intervenções de drogas

3
Se inscrever