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Objective To evaluate the effect of pretransplant iron overload on the clinical efficacy of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with severe aplastic anemia (SAA). Methods Clinical data of 80 SAA recipients who underwent allo-HSCT for the first time were retrospectively analyzed. According to the incidence of iron overload, all recipients were divided into the iron overload group (n=20) and non-iron overload group (n=60). The engraftment rate, incidence of postoperative complications and clinical prognosis of the recipients afterallo-HSCT were statistically compared between two groups. The influencing factors of 2-year overall survival (OS) and 180 d transplantation related mortality (TRM) were analyzed by Cox proportional hazards regression model. Results The engraftment rate of neutrophils in the non-iron overload group was 98% (59/60), significantly higher than 75% (15/20) in the iron overload group (P < 0.05). The engraftment rate of platelet in the non-iron overload group was 90% (54/60), significantly higher than 65% (13/20) in the iron overload group (P < 0.05). The incidence rate of bloodstream infection in the non-iron overload group was 23% (14/60), remarkably lower than 40% (8/20) in the iron overload group (P < 0.05). The 180 d TRM of the recipients in the non-iron overload group was 17%, significantly lower than 45% in the iron overload group (P < 0.05). The 1- and 2-year OS of the recipients in the non-iron overload group were 82% and 80%, significantly higher than 50% and 44% in the iron overload group (both P < 0.05). Iron overload or not was an independent risk factor of the OS and TRM of the recipients (both P < 0.05). Conclusions Iron overload can affect the OS and TRM of SAA patients after allo-HSCT.
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Abstract Mucopolysaccharidosis II (MPS II—Hunter syndrome) is an X-linked lysosomal storage disorder caused by a deficiency in iduronate-2 sulfatase. Enzyme replacement therapy does not cross the blood-brain barrier (BBB), limiting the results in neurological forms of the disease. Another treatment option for MPS, hematopoietic stem cell transplantation (HSCT) has become the treatment of choice for the severe form of MPS I since it can preserve neurocognition when performed early in the course of the disease. Even though the intravenous therapy does not cross the BBB, it has become the recommended treatment for MPS II, and HSCT was not often indicated. In an attempt to understand why this treatment modality is rejected by most specialists as a treatment option for patients with Hunter syndrome, we sought to raise all HSCT cases already reported in the scientific literature. Databases used were Medline/PubMed, Lilacs/BVS Cochrane Library, DARE, SciELO, and SCOPUS. Different combinations of the terms "mucopolysaccharidosis II," "Hunter syndrome," "hematopoietic stem cell transplantation," "bone marrow transplantation," and "umbilical cord blood stem cell transplantation" were used. A total of 780 articles were found. After excluding redundant references and articles not related to the theme, 26 articles were included. A descriptive summary of each article is presented, and the main features are summed up. The clinical experience with HSCT in MPS II is small, and most of the available literature is outdated. The available data reveal poor patient selection criteria, varied conditioning regimens, distinct follow-up parameters, and post-HSCT outcomes of interest, making impossible to compare and generalize the results obtained. Recently, after the development of new conditioning protocols and techniques and the creation of bone marrow donor registries and umbilical cord banks, HSCT has become more secure and accessible. It seems now appropriate to reconsider HSCT as a treatment option for the neuronopathic form of MPS II.
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Objective To observe the safety and clinic effect of umbilical blood stem cell transplantation for the patients with chronic liver failure (CLF).Methods 44 patients with CLF were included in the research and divided into two groups,22 in control group received internal medicine treatment,the other 22 in treatment group received umbilical blood stem cell transplantation in addition to internal medicine treatment.The biochemical index,MELD scores,clinical symptoms,survival situation and adverse reaction of the patients were observed within 2,4,12 and 24 weeks.Results Albumin and prothrombin activity of treatment group were higher than those of control group,the MELD scores of the treatment group was lower than that of control group,the survival rate was higher than the control group,and the difference is significant between the two groups (P ＜ 0.05).There was no significant difference between the two groups in terms of alanine aminotransferase and total bilirubin (P ＞ 0.05).After 4 weeks treatment,fatigue,inappetite,abdominal distention and ascitic fluid of the treatment group were better than that of control group,the difference was statistically significant (P ＜ 0.05).Besides,the patients of the both groups did not have any adverse reaction or hepatocellular carcinoma.Conclusion Umbilical blood stem cell transplantation is safe and effective for the patients with CLF and can improve the survival rate of the patients.
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Objective To investigate the clinical efficacy of umbilical blood stem cell transplantation (UCBSCT) on the treatment of hepatitis B liver cirrhosis. Methods Forty-eight patients with hepatitis B liver cirrhosis were enrolled and divided into the treatment group and the control group. There were 25 patients in the treatment group , who received UCBSCT treatment based on conventional liver protection treatment and 23 patients in the control group , who received conventional liver protection treatment. The changes of liver function , coagulation function, clinical symptoms, signs and side effects were studied before the treatment and at 2, 4, 12 and 24 weeks post-treatment. Results The levels of albumin, cholinesterase, and prothrombin activity in the treatment group were higher than those before treatment and were higher than those in the control group. The parameters in the control group were not significantly changed before and after the treatment (P > 0.05). The levels of alanine aminotransferase,aspartate aminotransferase,total bilirubin in both two groups were not significantly changed before and after the treatment (P > 0.05). After 4-week treatment,the differences on improvement of appetite , lacking in strength , abdominal distension , ascites were statistically significant in the treatment group compared with the control group (P < 0.05). No adverse reactions were observed in all groups. Conclusion UCBSCT on the treatment of hepatitis B liver cirrhosis is safe and reliable.
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Objective To explore the clinical eficacy of umbilical cord blood stem cell transplantation in treatment of decompensated cirrhosis.Methods Thirty patients with decompensated cirrhosis were given umbilical cord blood stem cell transplantation (treatment group) and 30 patients with decompensated cirrhosis were given traditional treatment (control group).Liver function and blood coagulation function was tested after 4,8 weeks treatment respectively,and adverse effects were recorded at the same time.Results After 8 weeks treatment,total bilirubin,albumin and prothrombin time in treatment group was improved compared with that before treatment[(71.3 ± 37.8) μ mol/L vs.(107.3 ± 53.2) μ mol/L,(30.1 ± 4.9) g/L vs.(27.5 ± 5.1) g/L,(15.0 ± 2.9) s vs.(16.7 ± 3.9) s],and there was significant difference (P ＜ 0.05).There was no significant difference in the index before and after treatment in control group (P＞ 0.05).No obvious adverse reactions were observed in the process of umbilical cord blood stem cell transplantation.Conclusion Umbilical cord blood stem cell transplantation is safe and effective in treatment of deeompensated cirrhosis.
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As células-tronco hematopoéticas (CTH) são células que possuem a capacidade de se autorrenovar e se diferenciar em células especializadas do tecido sanguíneo e do sistema imune. Na medicina, sua importância pode ser evidenciada por seu uso rotineiro do tratamento de doenças onco-hematológicas e imunológicas. A dificuldade de se encontrarem doadores compatíveis de medula óssea tem estimulado a busca por fontes alternativas de CTH, notadamente o sangue de cordão umbilical e placentário (SCUP) e o sangue periférico. O número de unidades de SCUP armazenadas no mundo tem sido crescente desde a década de 1990. Em 2004 foi criada a rede BrasilCord, estabelecendo uma rede nacional de bancos de SCUP com o objetivo de aumentar as chances de localização de doadores e ampliar o número de bancos de SCUP no país. A despeito do baixo volume coletado e do maior tempo necessário para regenerar o tecido hematopoético, as CTH de SCUP vêm em alta concentração sanguínea, sua utilização como fonte de CTH para transplante apresenta menor risco de causar doença enxerto versus hospedeiro e possuem maior facilidade de obtenção do que as CTH provenientes de medula óssea.
Hematopoietic stem cells (HSC) are cells capable of self-renewal and differentiation into specialized blood tissue and immune system cells. In medicine, their importance is evidenced by their routine use in the treatment of onco-hematological and immunologic diseases. The difficulty of finding compatible bone marrow donors has motivated the search for alternative sources of HSC, notably placental/umbilical cord blood (PUCB). The number of PUCB units stored worldwide has been increasing since 1990. In 2004, the BrasilCord network was created, establishing a national network of PUCB banks with the aim of increasing the chances of finding donors and expanding the number of PUCB banks in the country. Despite the small volume collected and the greater amount of time required for the regeneration of the hematopoietic tissue, the blood concentration of HSC in PUCB is higher, their use as a source for HSC for transplantation presents a lower risk of causing graft versus host disease and they are more easily obtained compared to HSC originating from the bone marrow.
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Humanos , Trasplante de Células , Trasplante de Células Madre de Sangre del Cordón Umbilical , Células Madre HematopoyéticasRESUMEN
PURPOSE: Human umbilical cord blood as a potential source of hematopoietic stem cell for stem cell transplantation in children has recently been advocated. Clinical application of cord blood transplantation, however, requires adequate blood volume and number of stem cells. Currently, the number of stem cells in the cord blood is usually measured by flowcytometry, which requires strict quality control and high costs. Here, we postulate that the number of nucleated red blood cells(NRBC) which are relatively immature erythroid cells may correlate well with that of CD34-expressing(CD34+) cells which are considered lymphohematopoietic precursors. If so, CD34+ cell-rich cord blood can be selected and such cells enumerated by a simple and cost-effective blood smears. METHODS: Correlation between CD34+ cell and nucleated RBC in human cord blood were checked. Sixty cord blood specimens(30 specimens for group A with NRBC>500/mul and 30 specimens for group B with NRBC500/ul; 55+/-61(SD))(p<0.005). 3.Regression relationship among CD34+ cell, WBC, and NRBC counts was as following. ln(CD34+ cell) = -12.21 + 1.46xln(WBC)+ 0.25xln(NRBC) gamma2=30.07 p=0.0001 4.There was a significant correlation between CD34+ cell counts and NRBC counts(gamma=0.4334, p<0.005) CONCLUSIONS: Our results suggest that there is a significant correlation between CD34+ cell counts and NRBC counts, and that CD34+ cell-rich cord blood specimens can be selected by a simple NRBC counting.