Effects of Peripheral Blood Stem Cell Transplantation from Haploidentical Versus Matched Sibling Donors on High-risk and Refractory/Relapsed Acute Myeloid Leukemia / 肿瘤防治研究

Objective To investigate the clinical efficacy of peripheral blood stem cell transplantation from haploidentical and matched sibling donors for treatment of high-risk and refractory/relapsed acute myeloid leukemia (AML). Methods Data on the efficacy of haploidentical peripheral blood stem cell transplantation (Haplo-HSCT) with myeloablative conditioning regimen were retrospectively analyzed and compared with that of matched sibling donors' peripheral blood stem cell transplantation (MSD-HSCT) for treatment of high-risk refractory/relapsed AML in our center from January 1st, 2010 to June 30th, 2020. Results A total of 98 patients were enrolled, including 62 patients in the Haplo-HSCT group and 36 patients in MSD-HSCT group. The median age, conditioning regimen, and infusion doses of MNC and CD34+ cells were significantly different between the two groups, but no significant differences in other baseline parameters were found. Transplantation-related infectious complications and the incidence of acute and chronic graft-versus-host disease (GVHD) were also not significantly different between the two groups. The 3-year cumulative relapse in the Haplo-HSCT group was significantly lower than that in the MSD-HSCT group (16.2% vs. 41.1%, P=0.036). The 3-year DFS of the Haplo-HSCT and MSD-HSCT groups were 66.98% and 41.8%, respectively (P=0.140), and their OS were 73.37% and 51.41%, respectively (P=0.105). Conclusion The clinical efficacy of Haplo-HSCT for the treatment of high-risk and refractory/relapsed AML is similar to that of MSD-HSCT, and Haplo-HSCT may have better GVL effect.

Dose-enhanced immunochemotherapy followed by first-line autologous peripheral blood stem cell transplantation for young patients with high-risk aggressive B-cell lymphoma: an efficacy and prognostic factor analysis / 中华血液学杂志

Objective: This study aimed to determine the efficacy of dose-enhanced immunochemotherapy followed by autologous peripheral blood stem cell transplantation (ASCT) in young patients with newly diagnosed high-risk aggressive B-cell lymphoma. Methods: A retrospective study was conducted to examine the clinical and survival data of young patients with high-risk aggressive B-cell lymphoma who received dose-enhanced immunochemotherapy and ASCT as first-line treatment between January 2011 and December 2018 in Blood Diseases Hospital. Results: A total of 63 patients were included in the study. The median age range was 40 (14-63) years old. In terms of the induction therapy regimen, 52 cases received R-DA-EP (D) OCH, and the remaining 11 received R-HyperCVAD/R-MA. Sixteen (25.4% ) patients achieved partial response in the mid-term efficacy assessment, and ten of them were evaluated as complete response after transplantation. The median follow-up was 50 (8-112) months, and the 3-year progression-free survival (PFS) rate and overall survival (OS) rate were (83.9±4.7) % and (90.4±3.7) % , respectively. Univariate analysis demonstrated that age-adjusted international prognostic index ≥2 scores was a negative prognostic factor for OS (P=0.039) , and bone marrow involvement (BMI) was an adverse prognostic factor for OS (P<0.001) and PFS (P=0.001) . However, multivariate analysis confirmed that BMI was the only independent negative predictor of OS (P=0.016) and PFS (P=0.001) . Conclusions: The use of dose-enhanced immunochemotherapy in combination with ASCT as first-line therapy in the treatment of young, high-risk aggressive B-cell lymphoma results in good long-term outcomes, and BMI remains an adverse prognostic factor.

Application study of EAC mobilization scheme in autologous hematopoietic stem cell transplantation of lymphoma / 国际肿瘤学杂志

Objective:To explore the safety and efficacy of EAC [etoposide+ cytarabine+ cyclophosphamide (CTX)] mobilization scheme for mobilizing stem cells in patients with lymphoma undergoing autologous hematopoietic stem cell transplantation (ASCT).Methods:A total of 36 patients with lymphoma who had collected peripheral blood stem cells through EAC or CTX+ granulocyte colony stimulating factor (G-CSF) mobilization scheme in Cancer Hospital & Shenzhen Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College from June 2018 to March 2020 were retrospectively analyzed. Among them, 16 patients used EAC mobilization (EAC group), and 20 patients used CTX (CTX group). When white blood cells≤1.0×10 9/L, G-CSF (10 μg/kg per day) was given subcutaneously in two doses. The changes of hematology indexes, the number of collected cells, adverse reactions during mobilization collection and hematopoietic reconstitution after ASCT were observed. Results:The peripheral blood stem cells were collected on 5 d (3-8 d) after EAC+ G-CSF mobilization and 7 d (4-12 d) after CTX+ G-CSF mobilization. The success rates of collection in the EAC group and CTX group were 100% (16/16) and 75.0% (15/20) respectively, the high-quality collection rates were 87.5% (14/16) and 25.0% (5/20) respectively, and there were statistically significant differences ( P=0.041; P<0.001). The median of CD34 + cells of the two groups was 13.67×10 6/kg and 3.45×10 6/kg respectively, the median of mononuclear cells was 7.16×10 8/kg and 5.09×10 8/kg respectively, the median of CD34 + cells/mononuclear cells was 1.44% and 0.67% respectively, and there were statistically significant differences ( Z=-4.219, P<0.001; Z=-2.118, P=0.034; Z=-3.104, P=0.002). In the EAC group and CTX group, the incidences of grade 3 and above granulocytopenia were 100% (16/16) and 90.0% (18/20) respectively, the incidences of grade 3 and above hemoglobin reduction were 43.8% (7/16) and 25.0% (5/20) respectively, the incidences of grade 3 and above thrombocytopenia were 87.5% (14/16) and 65.0% (13/20) respectively, and there were no statistically significant differences ( P=0.492; P=0.298; P=0.245). There were no significant differences in the incidences of infection, adverse reactions of digestive system or other adverse reactions between the two groups (all P>0.05). All patients accepted improved Bucy scheme before ASCT. The median implantation time of neutrophils and platelets was 9.0 d and 10.5 d in the EAC group, which was 12.0 d and 13.5 d in the CTX group, and there were statistically significant differences ( Z=-4.698, P<0.001; Z=-3.757, P<0.001). Conclusion:EAC mobilization scheme can significantly increase the number of hematopoietic stem cell. This scheme has a high success rate of high-quality collection and the adverse reactions are within the controllable range. It provides a high-quality mobilization scheme for hematopoietic stem cell mobilization and collection, which is worthy of clinical promotion and application.

Validation of a formula predictive of peripheral blood stem cell yield and successful collection in healthy allogeneic donors

ABSTRACT Background: An efficient mobilization and collection of peripheral blood stem cells (PBSCs) are crucial to optimize engraftment in the recipient. We aim to validate a formula that predicted CD34+ cell yield and to describe variables that correlated with high yield mobilization and collection in healthy donors. Methods: We retrospectively analyzed clinical and laboratory data from healthy donors who underwent PBSC collection from 2006 to 2015. The predicted number of collected cells was calculated using the following formula: Total number of CD34+ (cells × 106/kg) yield = [(peripheral CD34+ cells/µL) × (0.43)/recipient body weight (kg)] × total liters processed. Results: We evaluated 338 collections from 307 allogeneic PBSC donors. The predicted versus the observed number of CD34+ cells/kg collected yielded an r-value of 0.775 (0.726-0.816; p < 0.0001). Overall, 55.7% donors had an acceptable mobilization level. Donors with a body weight <67 kg were less likely to yield a satisfactory CD34+ cell count (OR = 0.44; 95% CI 0.24-0.81), while a white blood cell (WBC) count >40 × 109/L (OR = 3.69; 2.11-6.46) and platelet count ≥200 × 109/L (OR = 2.09; 1.26-3.47) on the day of collection predicted a good level of mobilization. Predictors of a CD34+ cell yield/kg of ≥4 × 106 with only one apheresis session were: circulating CD34+ cells/µL >40 (OR = 16; 6.94-36.93), hemoglobin ≥14 g/dL (OR = 3.40; 1.53-7.57), WBC >40 × 109/L (OR = 4.61; 2.10-10.10) on the first collection day, and a positive delta weight between donor and recipient (OR = 3.10; 1.36-7.06). Conclusion: The formula for predicting CD34+ cell yield is accurate and suggests the optimal length of time for successful leukapheresis. Validation of the predictors of successful mobilization will help to further refine PBSC leukapheresis procedures.

Caso Clínico: Citopénia Autoinmune como complicación inmunológica de Trasplante Alogénico de Donante no emparentado, en paciente pediátrico con Anemia Drepanocítica / Clinical Case: Autoimmune Cytopenia as an immunological complication of Allogeneic Unrelated Donor Transplantation, in a pediatric patient with Sickle Cell Anemia

Introducción: La enfermedad de células falciformes es una condición heredada en la quese produce una hemoglobina anómala que desfavorece a la oxigenación tisular, crisis vaso-oclusivas y reacciones hemolíticas. Los pacientes con esta enfermedad presentan una activación anómala de la vía del complemento llevándolos al aumento en frecuencia de infecciones y enfermedades autoinmunes. Presentamos un caso de asociación de una enfermedad autoinmune en un paciente con enfermedad de células falciforme. Caso clínico: Niño de 10 años con Anemia drepanocítica (2009) con esplenectomía y crisis veno-oclusivas recurrentes, fue sometido a trasplante Alogénicoen abril del 2019fuera de la institución con donante isogrupo O+ no emparentado (10/10). Tratado con: Fludarabina ­Busulfan, Timoglobulina+ y Metotexate. Desarrolló Bicitopenia autoinmune y síndrome febril al día +165 post TPH. Glóbulos blancos: 360 uL, neutrófilos: 14 %, hemoglobina: 7.90 g/dL, plaquetas: 25000 uL, ferritina: 4695 ng/ml, IgG total: 9.88 gr/l, LDH: 190 UI/l. Proteína C reactiva: 2.79 mg/dL, Procalcitonina 0.13 ng/mL. Evolución: posterior a descartar infección viral, se completó un tratamiento antibiótico de amplio espectro y se realizó la suspensión del tratamiento inmunosupresor por sospecha de toxicidad, sin respuesta. Se realizó un estudio medular por citometría de flujo determinando una disminución de la línea linfoide B, y se concluye Citopenia Autoinmune como complicación inmunológica del trasplante. Desenlace: recibióterapia transfusional (plaquetoféresis + glóbulos rojos concentrados). Se utilizó metilprednisolona IV por 3 días y prednisona 30 mg por 14 días con reducción posterior gradual para inicio de Rituximab y ciclosporina. Se completó el tratamiento con Imnunoglobulina 6g IV por 5 días. Al alta glóbulos blancos: 5080 uL, neutrófilos: 67%, hemoglobina: 9.20 g/dL, plaquetas: 20000 uL, después de 18 días de ingreso hospitalario. Conclusión: Los resultados con el tratamiento en este caso sugieren que puede serrazonable considerar las citopeniasautoinmunes como una manifestación hematológica diagnóstica de la EICH crónica. Alternativamente, es posible que el tratamiento de citopenia inmune con esteroides, Rituximab y otros inmunosupresores

Introduction: Sickle cell disease is an inherited condition in which an abnormal hemoglobin is produced that impairs tissue oxygenation, vaso-occlusive crises and hemolytic reactions. Patients with this disease present an abnormal activation of the complement pathway, leading to an increase in the frequency of infections and autoimmune diseases. We present a case of association of an autoimmune disease in a patient with sickle cell disease. Clinical case:10-year-old boy with sickle cell anemia (2009) with splenectomy and recurrent veno-occlusive crisis, underwent Allogeneic transplantation in April 2019 outside the institution with an unrelated isogroup O + donor (10/10). Treated with: Fludarabine -Busulfan, Thymoglobulin + and Metotexate. He developed autoimmune bicytopenia and febrile syndrome at +165 day post HSCT. White blood cells: 360 uL, neutrophils: 14%, hemoglobin: 7.90 g / dL, platelets: 25,000 uL, ferritin: 4695 ng / ml, total IgG: 9.88 gr / l, LDH: 190 IU/l. C-reactive protein: 2.79 mg/dL, procalcitonin 0.13 ng / mL. Evolution:after ruling out viral infection, the patient completed a broad-spectrum antibiotic treatment and underwent suspension of immunosuppressive treatment due to suspected toxicity, with no response. A medullary study by flow cytometry was performed, determining a decrease in the B lymphoid line, and autoimmune cytopenia was concluded as an immunologicalcomplication of the transplant. Outcome:The patient received transfusion therapy (plateletpheresis + concentrated red blood cells). He also received IV methylprednisolone for 3 days and 30 mg prednisone for 14 days with gradual subsequent reduction to start Rituximab and cyclosporine. The treatment with Immunoglobulin 6g IV for 5 days was completed. At discharge, white blood cells: 5080 uL, neutrophils: 67%, hemoglobin: 9.20 g / dL, platelets: 20,000 uL, after 18 days of hospital admission. Conclusion:The results with treatment in this case suggest that it may be reasonable to consider autoimmune cytopenias asa diagnostic hematological manifestation of chronic GVHD. Alternatively, it is possible to treat immune cytopenia with steroids, rituximab, and other immunosuppressants

Trasplante haploidéntico de progenitores hematopoyéticos en pacientes con diagnóstico de leucemia mieloide aguda / Haploidentical transplantation of hematopoietic progenitors in patients diagnosed with acute myeloid leukemia

Resumen Introducción: En pacientes con leucemia mieloide aguda (LMA) el trasplante de progenitores hematopoyético (TPH) es el único tratamientoz curativo. El objetivo de este estudio es presentar la experiencia y resultados del trasplante haploidéntico en pacientes adultos con LMA en la Fundación Valle del Lili, Cali - Colombia. Materiales y métodos: Estudio de cohorte retrospectivo de pacientes que recibieron trasplante haploidéntico entre 2013 y 2017, con acondicionamiento mieloablativo y ciclofosfamida postrasplante, en Fundación Valle del Lili, Cali (Colombia). Resultados: Se realizaron 47 trasplantes en pacientes con leucemia mieloide aguda en la fecha de estudio, se incluyeron en el análisis 21 pacientes con donante haploidéntico, a 3 años tanto la supervivencia global y libre de eventos fue del 38%. La incidencia acumulada de mortalidad relacionada al trasplante fue del 26% a 100 días y del 38,3%, a 38 meses de seguimiento. La incidencia acumulada de recaída a 38 meses fue del 19%. Con respecto a la enfermedad injerto versus huésped (EICH) se encontró que la incidencia acumulada de EICH aguda grado II-IV, grado III-IV y EICH crónico fue del 19%, 5% y 19% respectivamente. Conclusión: Los resultados de este estudio sugieren que el trasplante haploidéntico es una alternativa factible como tratamiento para pacientes con diagnóstico de LMA en nuestro medio.

Abstract Introduction: In patients with acute myeloid leukemia (AML), hematopoietic progenitor transplantation (PHT) is the only curative treatment. The objective of this study is to present the experience and results of haploidentical transplantation in adult patients with AML at the Valle del Lili Foundation, Cali - Colombia. Materials and methods: Retrospective cohort study of patients who received haploidentical transplantation between 2013 and 2017, with myeloablative conditioning and post-transplant cyclophosphamide, in Fundación Valle del Lili, Cali (Colombia). Results: 47 transplants were performed in patients with acute myeloid leukemia on the study date, 21 patients with haploidentical donors were included in the analysis, at 3 years both overall and event-free survival was 38%. The cumulative incidence of transplant-related mortality was 26% at 100 days and 38.3% at 38 months of follow-up. The cumulative incidence of relapse at 38 months was 19%. Regarding graft versus host disease (GVHD), it was found that the cumulative incidence of acute GVHD grade II-IV, grade III-IV and chronic GVHD was 19%, 5% and 19% respectively. Conclusion: The results of this study suggest that haploidentical transplantation is a feasible alternative as a treatment for patients diagnosed with AML in our environment.

Autologous peripheral blood stem cell transplantation for POEMS syndrome / 中华内科杂志

Six patients with POEMS syndrome who received autologous peripheral blood stem cell transplantation (auto-PBSCT) were retrospectively analyzed.Conditioning regimen was high dose melphalan.Peripheral blood stem cells were collected after mobilization with cyclophosphamide (CTX) and growth factors.One patient presenting hydrothorax and ascites was treated with 3 cycles of lenalidomide and dexamethasone before mobilization.Auto-PBSCT was fairly tolerable.Hematopoietic reconstitution was successful in all patients without transplantation-related mortality.A decrease or normalization of serum vascular epithelial growth factor (VEGF) was observed in all patients at 3 months after transplantation.The neurological remission was seen in 5/6 patients.

Characterization and Differentiation of Circulating Blood Mesenchymal Stem Cells and the Role of Phosphatidylinositol 3-Kinase in Modulating the Adhesion

Bone marrow mesenchymal stem cells (BM MSCs) can differentiate into multi-lineage tissues. However, obtaining BM MSCs by aspiration is difficult and can be painful; therefore peripheral blood (PB) MSCs might provide an easier alternative for clinical applications. Here, we show that circulating PB MSCs proliferate as efficiently as BM MSCs in the presence of extracellular matrix (ECM) and that differentiation potential into osteoblast in vitro and in vivo. Both BM MSCs and PB MSCs developed into new bone when subcutaneously transplanted into immune-compromised mice using hydroxyapatite/tricalcium phosphate as a carrier. Furthermore, LY294002 and Wortmannin blocked mesenchymal stem cell attachment in a dose-dependent manner, suggesting a role of phosphatidylinositol 3-kinase in MSC attachment. Our data showed that the growth of PB MSCs could be regulated by interaction with the ECM and that these cells could differentiate into osteoblasts, suggesting their potential for clinical applications.

Successful engraftment after infusion of multiple low doses of CD34+ cells from a poorly matched sibling donor in a patient with severe aplastic anemia / 영남의대학술지

The dose of CD34+ cells is known to influence the outcome of allogeneic peripheral blood stem cell (PBSC) and/or T-cell-depleted transplantation. A previous study proposed that 2×10⁶ CD34+ cells/kg is the ideal minimum dose for allogeneic transplantation, although lower doses did not preclude successful therapy. In the case we present here, CD34+ cells were collected from a matched sibling donor on the day of allogeneic hematopoietic stem cell transplantation; however, the number of cells was not sufficient for transplantation. Consequently, PBSCs were collected three additional times and were infused along with cord blood cells from the donor that were cryopreserved at birth. The cumulative dose of total nuclear cells and CD34+ cells was 15.9×10⁸ cells/kg and 0.95×10⁶ cells/kg, respectively. White blood cells from this patient were engrafted on day 12. In summary, we report successful engraftment after infusion of multiple low doses of CD34+ cells in a patient with severe aplastic anemia.

Analysis of factors influencing the mobilization and collection of autologous peripheral blood stem cells / 白血病·淋巴瘤

Objective@#To explore the factors influencing the mobilization and collection of autologous peripheral blood stem cells.@*Methods@#The clinical data of 62 patients who received autologous peripheral blood hematopoietic stem cell mobilization in Shanxi Provincial Cancer Hospital from April 2012 to March 2017 were collected. The effects of age, gender, disease type, chemotherapy cycle, disease status, different schemes and the number of CD34+ cells in peripheral blood of patients 1 d before collection on the number of CD34+ cells and the success rate of CD34+ cells collection were analyzed. Measurement data were compared by one-way ANOVA and t test; count data were compared by χ 2 test; multivariate analysis was performed by multiple linear regression analysis.@*Results@#There were statistically significant differences in the number of CD34+ cells between patients with chemotherapy >6 cycles and ≤6 cycles [(2.6±1.3)×106/kg vs. (5.8±2.2)×106/kg; t = 5.221, P < 0.01], and the difference in the success rate of CD34+ cell collection between the two groups was statistically significant [68.8% (11/16) vs. 97.8% (45/46); χ2 = 8.396, P = 0.004]. The difference in the CD34+ cells yield was not statistical significance between male and female patients [(5.4±2.2)×106/kg vs. (4.5±2.8)×106/kg; t = 1.302, P = 0.198)], but the collection success rate in males was higher than that in females [97.6% (40/41) vs. 76.2% (16/21)], and the difference was statistically significant (χ 2 = 5.017, P = 0.025). The success rate of CD34+ cell collection in patients with ≥10/μl CD34+ cell in the peripheral blood was significantly higher than that in patients with < 10/μl CD34+ cells 1 d before the collection[97.9% (47/48) vs. 64.3% (9/14)], and the difference was statistically significant (χ 2 = 10.668, P = 0.001). The differences in CD34+ cells yield and collection success rate between patients with different age, disease type, disease status and mobilization regimen were not statistically significant (all P > 0.05). Multi-factor analysis showed that > 6 cycles chemotherapy before mobilization was the adverse factor affecting stem cell collection (b = -3.435, P < 0.01).@*Conclusions@#The effective mobilization and collection of autologous peripheral blood stem cells are related to the number of chemotherapy cycles before mobilization. The stem cell mobilization and collection should be conducted as soon as possible when the chemotherapy is ≤ 6 cycles and the patient reaches partial remission or above. In addition, peripheral blood CD34+ cell count should be monitored during mobilization. When the peripheral blood CD34+ cell count is > 10/μl, the collection could be started on the next day to obtain a better collection effect, so as to improve the success rate of collection.

The role of mesenchymal stem cells in allogeneic hematopoietic stem cell transplantation for patients with refractory severe aplastic anemia / 中华血液学杂志

Objective@#To evaluate the efficacy and safety of mesenchymal stem cells in allogeneic hematopoietic stem cell transplantation for patients with refractory severe aplastic anemia (R-SAA) .@*Method@#The clinical data of 25 R-SAA patients receiving co-transplantation of mesenchymal stem cells combined with peripheral blood stem cells from sibling donors (10 cases) and unrelated donors (15 cases) from March 2010 to July 2018 in Zhengzhou University Affiliated Tumor Hospital were retrospectively analyzed. Antithymocyte globulin (ATG) treatment was ineffective/relapsed in 11 cases, and cyclosporine (CsA) treatment ineffective/relapsed in 14 cases.@*Results@#There were 13 male and 12 female among these patients. One patient had a primary graft failure, one patient had a poorly engraftment of platelets, and the remaining 23 patients achieved hematopoietic engraftment. The median time of granulocyte engraftment was 12.5 (10-23) days and 15 (11-25) days for megakaryocyte. Incidences of grade Ⅰ/Ⅱ acute graft-versus-host disease (aGVHD) and chronic graft-versus-host disease (cGVHD) were 37.5% (9/24) and 21.7% (5/23) , respectively. There was no severe GVHD and no severe complications that related to transplantation. 21 of 25 (84%) patients were alive with a median follow-up of 22.9 (1.6-107.8) months. The 5-year overall survival rate after transplantation was (83.6±7.5) %.@*Conclusion@#The combination of mesenchymal stem cells is reliable and safe in the treatment of R-SAA in peripheral blood stem cell transplantation of unrelated donors and sibling donors, which could significantly reduce the incidence of GVHD and severe transplantation-related complications.

Long-term efficacy of autologous hematopoietic stem cell transplantation in systemic sclerosis patients / 中华风湿病学杂志

Objective@#To observe the long-term efficacy and safety of autologous hematopoietic stem cell transplantation (AHSCT) for systemic sclerosis (SSc) patients.@*Methods@#Between May 2007 and June 2009,4 patients with SSc were enrolled in the study. Peripheral blood stem cells were mobilized with cyclopho-sphamide (CTX) followed by granulocyte colony stimulating factor (G-CSF). Conditioning was performed with i.v. cyclophosphamide 50 mg·kg-1·d-1 for 4 days. The results of the modified Rodnan skin score (mRSS), thoracic high-resolution computer tomography and pulmonary function were collected after transplantation.@*Results@#There was an improvement in mRSS, lung function and HRCT in the six months after AHSCT. Within six month to one year after transplantation, one patient had sustained and two patients recurred. After active treatments two patients were improved again. During the follow-up of 8.7 (4.1-9.8) years, three patients were stable and one patient died. Infection and hepatic function injury were the major complications. There was not transplant-related mortality.@*Conclusion@#AHSCT with CTX as a pre-conditioning regimen is safe and effective for SSc. The efficacy for patients with short course, rapid progress and edema is significant. However, long-term efficacy is poor, and long-term maintenance treatment is needed.

Long-term efficacy of autologous hematopoietic stem cell transplantation in systemic sclerosis patients / 中华风湿病学杂志

Objective To observe the long-term efficacy and safety of autologous hematopoietic stem cell transplantation (AHSCT) for systemic sclerosis (SSc) patients.Methods Between May 2007 and June 2009,4 patients with SSc were enrolled in the study.Peripheral blood stem cells were mobilized with cyclophosphamide (CTX) followed by granulocyte colony stimulating factor (G-CSF).Conditioning was performed with i.v.cyclophosphamide 50 mg ·kg-1 ·d-1 for 4 days.The results of the modified Rodnan skin score (mRSS),thoracic high-resolution computer tomography and pulmonary function were collected after transplantation.Results There was an improvement in mRSS,lung function and HRCT in the six months after AHSCT.Within six month to one year after transplantation,one patient had sustained and two patients recurred.After active treatments two patients were improved again.During the follow-up of 8.7 (4.1-9.8) years,three patients were stable and one patient died.Infection and hepatic function injury were the major complications.There was not transplant-related mortality.Conclusion AHSCT with CTX as a pre-conditioning regimen is safe and effective for SSc.The efficacy for patients with short course,rapid progress and edema is significant.However,long-term efficacy is poor,and long-term maintenance treatment is needed.

Analysis of factors influencing the mobilization and collection of autologous peripheral blood stem cells / 白血病·淋巴瘤

Objective To explore the factors influencing the mobilization and collection of autologous peripheral blood stem cells. Methods The clinical data of 62 patients who received autologous peripheral blood hematopoietic stem cell mobilization in Shanxi Provincial Cancer Hospital from April 2012 to March 2017 were collected. The effects of age, gender, disease type, chemotherapy cycle, disease status, different schemes and the number of CD34+cells in peripheral blood of patients 1 d before collection on the number of CD34+cells and the success rate of CD34+cells collection were analyzed. Measurement data were compared by one-way ANOVA and t test; count data were compared by χ2 test; multivariate analysis was performed by multiple linear regression analysis. Results There were statistically significant differences in the number of CD34+cells between patients with chemotherapy＞6 cycles and≤6 cycles [(2.6±1.3)×106/kg vs. (5.8±2.2)×106/kg;t＝5.221, P<0.01], and the difference in the success rate of CD34+cell collection between the two groups was statistically significant [68.8% (11/16) vs. 97.8% (45/46); χ 2＝ 8.396, P ＝ 0.004]. The difference in the CD34+cells yield was not statistical significance between male and female patients [(5.4±2.2)×106/kg vs. (4.5± 2.8)×106/kg; t ＝ 1.302, P＝ 0.198)], but the collection success rate in males was higher than that in females [97.6% (40/41) vs. 76.2% (16/21)], and the difference was statistically significant (χ2＝5.017, P ＝0.025). The success rate of CD34 + cell collection in patients with ≥10/μl CD34 + cell in the peripheral blood was significantly higher than that in patients with < 10/μl CD34+cells 1 d before the collection[97.9% (47/48) vs. 64.3% (9/14)], and the difference was statistically significant (χ 2 ＝ 10.668, P＝ 0.001). The differences in CD34+cells yield and collection success rate between patients with different age, disease type, disease status and mobilization regimen were not statistically significant (all P＞ 0.05). Multi-factor analysis showed that ＞6 cycles chemotherapy before mobilization was the adverse factor affecting stem cell collection (b ＝ -3.435, P< 0.01). Conclusions The effective mobilization and collection of autologous peripheral blood stem cells are related to the number of chemotherapy cycles before mobilization. The stem cell mobilization and collection should be conducted as soon as possible when the chemotherapy is ≤ 6 cycles and the patient reaches partial remission or above. In addition, peripheral blood CD34+cell count should be monitored during mobilization. When the peripheral blood CD34+cell count is ＞10/μl, the collection could be started on the next day to obtain a better collection effect, so as to improve the success rate of collection.

Rehabilitation in a Patient with POEMS Syndrome Following High-dose Chemotherapy with Autologous Peripheral Blood Stem Cell Transplantation / The Japanese Journal of Rehabilitation Medicine

POEMS (polyneuropathy, organomegaly, endocrinopathy, M protein, and skin changes) syndrome is an extremely rare neurological disease exhibiting various symptoms. Few reports have investigated rehabilitation in this disease. The present study reported the details of rehabilitation in a 40-year-old man with POEMS syndrome. Abnormal sensation was initially observed in the distal legs, followed by deterioration of muscle strength. He was admitted to our hospital 2 months after onset and received high-dose chemotherapy with autologous peripheral blood stem cell transplantation for acute exacerbation of polyneuropathy. Electrophysiological examination revealed axonal neuropathy. Gradual improvement in muscle strength was observed after high-dose chemotherapy with autologous peripheral blood stem cell transplantation. He was able to walk with a knee-ankle-foot orthosis and crutches at the time of discharge, but he used a wheelchair for routine activities. He could ascend and descend stairs in his house with bottom shuffling. As it is difficult to predict the extent of ultimate improvement and timing of remission in this disease, it is important to devise a rehabilitation program from a long-term perspective and to aim at recovery of independence for daily living activities and social reintegration using supportive devices and compensatory methods.

Rehabilitation in a Patient with POEMS Syndrome Following High-dose Chemotherapy with Autologous Peripheral Blood Stem Cell Transplantation / The Japanese Journal of Rehabilitation Medicine

POEMS (polyneuropathy, organomegaly, endocrinopathy, M protein, and skin changes) syndrome is an extremely rare neurological disease exhibiting various symptoms. Few reports have investigated rehabilitation in this disease. The present study reported the details of rehabilitation in a 40-year-old man with POEMS syndrome. Abnormal sensation was initially observed in the distal legs, followed by deterioration of muscle strength. He was admitted to our hospital 2 months after onset and received high-dose chemotherapy with autologous peripheral blood stem cell transplantation for acute exacerbation of polyneuropathy. Electrophysiological examination revealed axonal neuropathy. Gradual improvement in muscle strength was observed after high-dose chemotherapy with autologous peripheral blood stem cell transplantation. He was able to walk with a knee-ankle-foot orthosis and crutches at the time of discharge, but he used a wheelchair for routine activities. He could ascend and descend stairs in his house with bottom shuffling. As it is difficult to predict the extent of ultimate improvement and timing of remission in this disease, it is important to devise a rehabilitation program from a long-term perspective and to aim at recovery of independence for daily living activities and social reintegration using supportive devices and compensatory methods.

Nutritional Intervention for a Patient with Acute Lymphoblastic Leukemia on Allogeneic Peripheral Blood Stem Cell Transplantation

Hematopoietic stem cell transplantation (HSCT) causes many complications such as anorexia, nausea, vomiting, diarrhea, and mucositis. Most patients undergoing HSCT have risk for malnutrition in the process of transplantation so artificial nutrition support is required. The purpose of this case report is to share our experience of applying nutrition intervention during the transplantation period. According to HSCT process, the change of the patient's gastrointestinal symptoms, oral intake and nutritional status was recorded. By encouraging oral intake and providing parenteral nutrition, the patient had only 0.3%, losing weight during the transplantation period. In conclusion, it emphasized that the nutritional status changes during the HSCT period should be closely monitored and nutritional management through appropriate nutritional support and interventions in hospital and after discharge.

Efficacy and Safety of Unmanipulated Haploidentical Related Donor Allogeneic Peripheral Blood Stem Cell Transplantation in Patients with Relapsed/Refractory Acute Myeloid Leukemia / 中华医学杂志(英文版)

<p><b>Background</b>Studies of haploidentical-related donor (HRD) stem cell transplantation using a combination of peripheral blood stem cells (PBSCs) and bone marrow as the graft have reported encouraging results for patients with hematological diseases. However, few studies specifically reported transplantation of only PBSCs from HRDs among patients with relapsed or refractory acute myeloid leukemia (AML). Here, the long-term outcomes and side effects of unmanipulated HRD PBSC transplantation (HRD-PBSCT) for relapsed/refractory AML were analyzed.</p><p><b>Methods</b>We performed a retrospective analysis of the outcomes in relapsed/refractory AML patients who underwent PBSCT from HRDs (n = 36).</p><p><b>Results</b>Thirty-one (86.1%) patients in the HRD-PBSCT group achieved platelet recovery. The cumulative incidence of acute graft-versus-host disease (aGVHD) in the HRD-PBSCT group was 40.00%, and the cumulative incidence of grades 2-4 aGVHD in this group was 13.33%. A total of 13 patients in the HRD-PBSCT group had recurrent disease at a median of 183 days after transplantation (range: 10-1700 days), reaching cumulative incidences of relapse of 50.28% at 5 years. On multivariate analysis, donor age and patient age >40 years were independent risk factors for inferior disease-free survival or overall survival (P < 0.05). The results of the present study demonstrate rapid and complete neutrophil engraftment, a low incidence of grade 2-4 aGVHD, and promising survival rates in patients after HRD-PBSCT. Thus, granulocyte colony-stimulating factor-primed PBSCs may be a reliable graft source in unmanipulated HRD-HSCT under myeloablative conditioning when no matched sibling donor is available.</p><p><b>Conclusions</b>Our results support the feasibility, effectiveness, and tolerability of PBSCs as a graft source in unmanipulated HRD transplantation under myeloablative conditioning in patients with leukemia.</p>

Clinical analysis of autologous peripheral blood hematopoietic stem cell mobilization regimen in 56 multiple myeloma patients / 中国肿瘤临床

Objective: To compare the efficacy between chemotherapy plus granulocyte colony-stimulating factor (G-CSF) and chemotherapy plus G-CSF and granulocyte-macrophage colony-stimulating factor (GM-CSF) for the mobilization of peripheral blood stem cells (PBSC) and hematopoietic recovery after transplantation in patients with multiple myeloma (MM). Methods: A retrospective study of autologous PBSC (APBSC) mobilization data of 56 MM patients who were treated with chemotherapy plus G-CSF or chemotherapy plus G-CSF and GM-CSF from May 2008 to July 2016 in Tianjin Medical University Cancer Institute and Hospital was conducted. The mobilization efficacy and hematopoietic recovery were analyzed. Results: In the univariate analysis, the successful collection rate of a single harvest in women and in patients with ISS stage Ⅲ and R-ISS stage Ⅱ/Ⅲ and treated with chemotherapy plus G-CSF was lower (P<0.05). However, age (≤60 years vs.>60 years), subtype, D-S staging (Ⅰ+Ⅱvs.Ⅲ), number of cycles of chemotherapy before mobilization (≤6 cycles vs.>6 cycles), disease phase before mobilization (PR vs. CR), and interval between diagnosis and mobilization (≤18 months vs.>18 months) were not correlated with CD34+ cell collection and successful mobilization rates (P>0.05). In the multivariate model, the successful mobilization rate in patients who received the chemotherapy plus G-CSF and GM-CSF mobilization regimen was higher (OR=12.009, 95% CI=1.961-73.537). The effect of mobilization regimens remained significant (P=0.007). Hematopoietic recovery without transplantation-related mortality occurred successfully in all patients. Conclusions: Chemotherapy plus G-CSF and GM-CSF mobilization regimens can significantly increase the effect of APBSC mobilization and ensure the recovery of hematopoietic function after transplantation. Chemotherapy plus G-CSF and GM-CSF mobilization regimens are safe and effective for mobilizing APBSCs.

Study on the efficacy of CD34 + cells combined with sarpogrelate hydrochlotride tablets for the re-construction of the blood supply of thromboangiitis obliterans / 中国医师杂志

Objective To investigate the effect of autologous peripheral blood stem cell transplanta-tion combined with CD34 + cells and oral sarpogrelate hydrochloride for the treatment of vascular reconstruc-tion and blood supply on the thromboangiitis obliterans. Methods Thromboangiitis obliterans ( TAO) of 262 patients with 262 lower limbs were divided into stem cells CD34 + cells transplantation combined with sarpogrelate hydrochloride group ( group A) with 100 lower limbs, CD34 + stem cell transplantation group ( group B) with 91 lower limbs, and sarpogrelate hydrochloride oral group ( group C) with 71 lower limbs. The degree of lower limb local blood flow variability were calculated at the three levels of skin, blood vessels and blood by preoperative and postoperative use of multi-function monitoring instrument, Doppler detector, transcutaneous oxygen pressure monitor, and digital subtraction angiography ( DSA) , respectively. Results⑴ The degree of shank, foot cyanosis, cool skin, and pain was relieved significantly in groups A and B than in group C for 1 month after the treatment (P<0. 05). ⑵Intermittent claudication distance, the skin temperature of the lower leg and foot to patients in the groups A and B than in group C, with a significant difference (F=7. 01, F=7. 04, P<0. 05) for 3 months after the treatment. ⑶ Among the patients with amputation, 10 cases were in group A, 16 cases in group B, and 31 cases in group C for 6 months after the treatment. ⑷ Transcutaneous oxygen pressure was increased from ( 30. 43 ± 4. 31 ) mmHg to ( 37. 21 ± 9. 01)mmHg (F=5. 69, P<0. 05), ankle brachial index from (0. 32 ±0. 23) to (0. 91 ± 0. 16) (F=6. 71, P<0. 05), the volume of blood flow index from the photoelectric (0. 22 ± 0. 04) to (0. 83 ± 0. 13 (F=5. 69, P<0. 05), oxygen saturation from (42. 41 ±9. 61)％ to (79. 61 ±20. 34)％ (F=5. 74, P<0. 05), and DSA score (0. 23 ± 0. 03) increased to (1. 35 ± 0. 23) (F=7. 14, P<0. 05), which was sig-nificantly higher than group B and group C ( F=7. 01, F=7. 04, F=7. 12, F=7. 08, F=7. 15, P<0. 05) for one year after treatment. Conclusions Treatment of peripheral blood stem cells CD34 + cell transplantation combined with oral sarpogrelate hydrochloride can significantly improve the vascular regener-ation and its blood supply in TAO lower extremity limb.