Autologous stem cell-based therapy for sickle cell leg ulcer: a pilot study

作者：Meneses Jose Valber L; Fortuna Vitor; de Souza Eliane Silva; Daltro Gildasio Cerqueira; Meyer Roberto; Minniti Caterina P; Borojevic Radovan

来源：British Journal of Haematology, 2016, 175(5): 949-955.

DOI：10.1111/bjh.14326

• stem cell transplantation
• sickle cell disease
• leg ulcer
• clinical trial
• bone marrow

Recurrent chronic leg ulcers are among the most severe vasculopathic complications of sickle cell disease (SCD). Their treatment remains a challenge. Stem cell therapy with bone marrow mononuclear cells (BMMC) is a promising new therapeutic option for other forms of chronic ulcers. This prospective pilot study was performed to evaluate safety and feasibility of BMMC implantation in patients with SCD and chronic leg ulcers (SCLU). Ulcer closure, recurrence and local pain were evaluated. BMMC were successfully administered to 23 SCLU patients and no serious adverse events occurred. During the 6-month follow-up period, 91<bold></bold>3% of patients had improved ulcer pain compared with baseline and 29<bold></bold>2% of the treated ulcers achieved total healing. The frequency of progenitor stem cells (CD34CD45(low) and fibroblast colony-forming units) in BMMC was found to be significantly reduced in SCLU patients and compared to SCD patients without ulcers (P<0<bold></bold>004 and P<0<bold></bold>01, respectively). No relationship was observed between treatment outcome and the number of implanted BM progenitor stem cells. In conclusion, BMMC implantation is a feasible and safe procedure, showing favourable outcomes for the treatment of SCLU, and encouraging further controlled clinical trials.

• 出版日期2016-12