OBJECTIVETo analyze a Duchenne muscular dystrophy(DMD) patient's muscular regeneration, dystrophin expression and locomotive variation before and after he underwent umbilical cord blood stem cell transplantation in order to assess the therapeutic effect.

METHODSA 12-year-old DMD boy who could not walk for 3 years was confirmed by gene analysis and dystrophin protein immune test on his muscle. He had no other chronic disease. By HLA matching, a piece of umbilical cord blood stem cell with 6 HLA sites matching to the boy was found in Guangdong Umbilical Cord Blood Bank. The number of the nucleated cells of the umbilical cord blood stem cell was 24.08x 10(8). After pretreatment for the DMD boy with busulfan, cyclophosphamide and rabbit anti-human thymocyte globulin, the allergenic cord blood stem cells were transplanted into him by intravenous injection. Cyclosporin A, methylprednisolone, MMF, prostaglandin E1 and ganciclovir were given after the transplantation. At the same time, Gran, the granulocytic cell stimulating factor, and gamma globulin were administered. The biochemistry profile including serum creatine kinase (CK), the reconstruction of blood making, the deletion exon of DMD gene, the regenerating muscles, the dystrophin protein expression, and the locomotive function of the DMD boy were tested regularly.

RESULTS(1) The white blood cells (WBC) of peripheral blood decreased gradually to zero after pretreatment. In a period of 15 days after transplantation, the neutrophil increased to 0.5x 10(9)/L; at 25 days, WBC increased to normal level. Blood platelet was more than 20x 10(9)/L at 22 days. The hemoglobin rose to 85-100 g/L. At 140 days, sternal puncture revealed the rapid growth of neutrophil, blood platelet and hemoglobin. (2)At 140 days, the blood type of the DMD boy transformed from type O to type AB (the donor's blood type being AB). There was no grafe versus host reaction. (3) At 18, 30, 43, 55, 74 and 233 days after transplantation, the PCR-short tandem repeat test of the boy's peripheral blood DNA showed that his genotype was completely the same as the donor's. The results of PCR-short tandem repeat tests of the bone marrow cells DNA by sternal puncture at 140, 183 and 235 days were the same as those of the blood DNA. (4) At 60 days, DMD gene analysis by PCR showed that the defected DMD gene (exon 19 deletion) had been corrected by the umbilical cord stem cells transplantation. (5) At 75 days, the biopsy of calf muscle showed there were myoblast cells and muscular tubes growing. The dystrophin expressions were weak, but a few of them were strong. DNA analysis showed that the donor's gene DNA accounted for 1%-13%. At 126 days, obviously increased dystrophin positive muscular fibers of the boy were found. The donor's fibers rose to 2.5%-25%. (6) The serum CK of the boy declined from 5735 U/L to 274 U/L. (7) At 100 days, physical examination revealed improvement in his arms and legs.

CONCLUSIONThe therapy of Duchenne muscular dystrophy with allogeneic umbilical cord blood hematopoietic stem cell transplantation may reset up the blood-making function, decrease the serum CK level, restore the dystrophin in muscles, and improve the locomotive function of the DMD boy. These data suggest that the allogeneic umbilical cord blood hematopoietic stem cell transplantation may benefit the DMD boys.

Alprostadil ; therapeutic use ; Busulfan ; therapeutic use ; Child ; Combined Modality Therapy ; Cord Blood Stem Cell Transplantation ; methods ; Cyclosporine ; therapeutic use ; Dystrophin ; genetics ; Ganciclovir ; therapeutic use ; Humans ; Male ; Methylprednisolone ; therapeutic use ; Muscular Dystrophy, Duchenne ; genetics ; therapy ; Polymerase Chain Reaction ; Treatment Outcome

Objective:To evaluate the effects of aquatic exercise on lower-limb motor function and activities of daily living for patients with stroke. Methods:The randomized controlled trials (RCTs) about effects of aquatic therapeutic exercise on stroke patients were recalled from the databases of Cochrane Library, PEDro, PubMed, EMBASE, Web of Knowledge, Web of Science, OVID, EBSCO, CMCI, CNKI, Wanfang and VIP. The methodological quality of the included RCTs was evaluated. The data were extracted, and analysed with RevMan 5.3. Results:A total of 23 RCTs that represented 861 participants were evaluated. Compared with the control group, aquatic exercise significantly improved the performance of Berg Balance Scale (WMD = 2.69, 95%CI 1.21 to 4.16,P < 0.001), Timed Up and Go Test (WMD = -1.56, 95%CI -3.07 to -0.05,P < 0.05), Functional Reach Test (WMD = 2.69, 95%CI 1.21 to 4.16,P < 0.001), sway velocity of center of pressure (SVCOP) (left/right) (WMD = -1.38, 95%CI -2.72 to -0.05,P < 0.05), SVCOP (anteroposterior) (WMD = -1.64, 95%CI -3.10 to 　-0.18,P < 0.05), walking speed (SMD = 0.33, 95%CI 0.07 to 0.58,P < 0.05), Two Minute Walk Test (WMD = 12.75, 95%CI 4.17 to 21.34,P < 0.01), Functional Ambulation Category (WMD = 0.94, 95%CI 0.67 to 1.20,P < 0.001), muscle strength of knee extensor (WMD = 4.30, 95%CI 1.53 to 7.07,P < 0.01), muscle strength of knee flexor (WMD = 4.80, 95%CI 0.29 to 9.32,P < 0.05), and Functional Independence Measurement (WMD = 6.12, 95%CI 3.98 to 8.27,P < 0.001), but not significantly in the score of modified Barthel Index (WMD = 2.92, 95%CI -6.74 to 12.58,P = 0.55). Conclusion:Aquatic exercise can improve balance, walking and muscle strength of lower extremities of stroke patients, but do not for activities of daily living.

Objective:To explore the application of mobile medical technology in aquatic rehabilitation assessment for patients with spinal cord injury. Methods:From November, 2017 to April, 2018, 72 patients with spinal cord injury accepted aquatic exercise were randomly divided into control group (n = 36) and experimental group (n = 36). All the patients were assessed with Water Orientation Assessment of Alyn (WOTA), using paper scale for the control group, mobile scale for the experimental group, twice. The time for assessment and for recall was recorded. The accuracy of results and variety between assessment was compared. Results:The time for assessment and for recall was less in the experimental group than in the control group (t > 10.492, P < 0.001), with more accuracy of standard total score, the variety of total score and standard total score (χ² > 4.545, P < 0.05). Conclusion:Compared with the paper-based assessment, the assessment based on mobile technology may improve work efficiency.

Cardiac Rehabilitation ; Exercise Therapy ; Heart Failure/surgery* ; Heart Transplantation ; Humans