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.

Tislelizumab bridging to umbilical cord blood transplantation therapy for relapsed/refractory acute myeloid leukemia: report of 1 case and review of literature / 白血病·淋巴瘤

Objective:To explore the efficacy of tislelizumab combined with umbilical cord blood transplantation (UCBT) in relapsed/refractory acute myeloid leukemia (R/R AML) patients.Methods:The diagnosis and treatment of 1 patient with R/R AML who received tislelizumab bridging to UCBT after the failure of re-induction treatment in the First Affiliated Hospital of Soochow University in November 2021 was retrospectively analyzed.Results:The 59-year-old male patient with R/R AML achieved a complete remission after initial induction chemotherapy regimen of decitabine and venetoclax, and then additional consolidation therapy regimens of decitabine and middle-dose cytarabine, middle-dose cytarabine and idarubicin were performed. The patient relapsed 16 months later and failed to achieve a second remission after re-induction therapy regimens of cladribine, azacitidine, venetoclax combined with chemotherapy, and homoharringtonine, cytarabine combined with granulocyte colony-stimulating factor. Tislelizumab significantly reduced tumor burden and the patient achieved the complete remission after bridging to UCBT. After transplantation, the patient was given maintenance treatment with azacitidine and he had sustained remission without severe transplant-related complications during 9-month follow-up.Conclusions:The use of tislelizumab bridging UCBT can be a potential therapeutic strategy for R/R AML patients.

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.

Células madre mesenquimatosas y sus diferentes usos / Mesenchymal stem cells and their different uses

Introducción: las células madre mesenquimatosas (CMM) se diferencian de diversos tipos celulares para la regeneración de tejidos, esta característica sumada con la versatilidad del antígeno leucocitario humano (HLA) representan una eficaz alternativa para el tratamiento de enfermedades con tejidos deteriorados. Se pueden obtener a partir de médula ósea, cordón umbilical (CU) y sangre fetal. Objetivo: analizar los tipos de diferenciación de las CMM, sus métodos de extracción y su relación con bancos de sangre de cordón umbilical (BSCU), a fin de demostrar la eficacia de las CMM, en patologías que impliquen alteración de algún tejido u órgano. Metodología: se revisaron varias publicaciones en español e inglés en Pubmed, Clinicalkey y Science Direct; desde 2013 hasta 2020. Se usaron los términos sangre fetal, células madre mesenquimatosas, trasplante de Células Madre de Sangre del Cordón Umbilical y bancos de sangre. Con dicha información se redactó un panorama amplio sobre las células mesenquimales y como estas participan en diversas áreas de la salud, con un énfasis importante en sus usos terapéuticos y lo referente a de donde provienen. Conclusión: a través de la pluripotencialidad de las CMM, se han podido emplear en múltiples patologías pues reestablece tejidos o líneas celulares exitosamente. Así mismo, los recursos para su obtención son claves en la tolerancia de los pacientes, por lo cual una gran opción para su obtención es el CU, que actualmente cuenta con bancos exclusivos para esto. (AU)

Introduction: mesenchymal stem cells (MSC) differentiate into multiple cell types for tissue regeneration, this characteristic added with the versatility of human leukocyte antigen (HLA) represent an effective alternative for the treatment of diseases with damaged tissues. They can be obtained from bone marrow, umbilical cord (UC), and fetal blood. Objetive: analyze the types of differentiation of MSC, their extraction methods and their relationship with umbilical cord blood banks (UCBB), in order to demonstrate the efficacy of MSC, in pathologies that involve alteration of a tissue or organ. Methodology: several publications in Spanish and English in Pubmed, Clinicalkey and Science Direct were reviewed; from 2013 to 2020. The terms fetal blood, mesenchymal stem cells, Umbilical Cord Blood Stem Cell transplantation and blood banks were used. With this information, a broad overview of mesenchymal cells and how they participate in various areas of health was drawn up, with an important emphasis on their therapeutic uses and where they come from. Conclusion: through the pluripotentiality of MSC, they have been used in multiple pathologies as it successfully re-establishes tissues or cell lines. Also, the resources for obtaining it are key in the tolerance of patients, which is why a great option for obtaining it is the UC, which currently has exclusive banks for this.

Relief of HHV-6 Encephalomyelitis-induced Electric Shock-like Pain with Levetiracetam after Blood Stem Cell Transplantation

We encountered a case of HHV-6 encephalomyelitis following blood stem cell transplantation. A woman in her 30’s suffered from paroxysmal electric shock-like pain, itching, tremor of lower limbs and excessive sweating on the 15th day after cord blood transplantation. Urinary retention also occurred. Pregabalin and opioid administration was started, and foscarnet was administered after a finding of HHV-6DNA positive in cerebrospinal fluid. Combined use of levetiracetam 1000 mg per day decreased both frequency and intensity of the electric shock-like pain. In a week before the negative results of HHV-6 amplification, she became excessively drowsy. With withdrawal of levetiracetam, her consciousness returned to be clear. The main symptoms on discharge were pain and itching with numbness around the anus, and were well controlled with oxycodone 15 mg, pregabalin 225 mg, and lorazepam 0.5 mg per day. In our case of HHV-6 encephalomyelitis after cord blood transplantation, levetiracetam was effective in suppressing electric shock-like pain.

Human Umbilical Cord Mesenchymal Stem Cells Over Platelet Rich Fibrin Scaffold for Mandibular Cartilage Defects Regenerative Medicine

ABSTRACT Objective: To evaluate the regeneration of mandibular cartilage defect after implantation of human umbilical cord mesenchymal stem cells (hUCMSC) over platelet rich fibrin (PRF) as scaffold. Material and Methods: 20 male Wistar rats were randomly divided into four experimental groups consisting of: a control group featuring untreated mandibular defects (C), experimental groups whose mandibular defects were implanted with hUCMSC (E1), mandibular defects implanted with PRF (E2), mandibular defects implanted with hUCMSC and PRF scaffold (E3). The subjects were sacrificed after six weeks of observation for immunohistochemical examination in order to evaluate the expression of Ki67, Sox9, FGF 18, type 2 collagen, and aggrecan, in addition to histology examination to evaluate chondrocyte number and cartilage thickness. Data was analyzed with univariate analysis (ANOVA). Results: The implantation of hUCMSC and PRF scaffold proved capable of regenerating mandibular cartilage defect through the expression of FGF 18, Sox9, Ki67, chondrosis counts, type 2 collagen, aggrecan, and cartilage thickness. The regeneration were significantly higher in group E3. Conclusion: Human umbilical cord mesenchymal stem cells in platelet rich fibrin scaffold proved capable of regenerating mandibular cartilage defect.

Donación dirigida de sangre de Cordón Umbilical de Hermano Compatible en el Sistema de Salud Público de Chile / Directed sibling donor cord blood donation in the Chilean Public Health System

Resumen: Introducción: La sangre de cordón umbilical (SCU) como fuente para trasplante de células proge- nitoras hematopoyéticas (TPH) está bien establecida. Internacionalmente, menos del 10% de los TPH de SCU corresponde a donantes hermanos compatibles. Dentro de la red del Programa Infantil Nacional de Drogas Antineoplásicas (PINDA), existe desde enero 2004 un programa de donación dirigida de SCU para TPH. Pacientes y Método: Se diseñó un estudio observacional, retrospectivo, descriptivo, se revisaron el número y características de las unidades de SCU recolectadas en el PINDA y el número, características y evolución de los pacientes trasplantados con esas unidades entre enero de 2004 y octubre de 2018. Resultados: Sesenta unidades de SCU han sido recolectadas, de ellas 55 con registro completo. La mediana de volumen de las unidades almacenadas fue 74,8 ml (30,0-170,8), la mediana de células nucleadas totales 7,6 x 10e8 (2,0-21,1), mediana de células CD34+ 1,6 x 10e6 (0,2-11,6). Cuatro pacientes con leucemias de alto riesgo fueron trasplantados; mediana de segui miento es de 8 años. Todos desarrollaron complicaciones severas post TPH, uno de ellos falleció de recaída y los tres actualmente vivos presentan un Karnofsky/Lansky 100%. Conclusión: El programa ha permitido el trasplante de 4 pacientes que de otro modo no habrían tenido acceso a un donante. Este programa de donación dirigida puede ser considerado una primera etapa para el desarrollo de un banco público de sangre de cordón umbilical en Chile.

Abstract: Introduction: Cord blood (CB) as a source of Hematopoietic Stem Cells for Transplantation (HSCT) is well established. Worldwide, nonetheless, less than 10% of the CB HSCTs are performed with a match sibling donor. Since 2004, the Chilean National Childhood Cancer Program (PINDA) net work, has established a CB directed donation program for HSCT. Patients and Method: An obser vational, descriptive and retrospective study was designed to assess the number and characteristics of the CB units collected in the program as well as the number, clinical characteristics and follow-up of the patients who received an HSCT from those CB units between January 2004 and October 2018. Results: Sixty CB units have been collected; 55 of them with full records and stored. The median volume collected was 74.8 ml (30.0-170.8), the median number of total nucleated cells was 7.6 x 10e8 (2.0-21.1), and the median of CD34+ cells was 1.6 x 10e6 (0.2-11.6). Four high-risk leukemia patients received HSCT, all of them developed severe complications after transplantation and one patient died due to relapse. Those patients currently alive have a 100% Karnofsky/Lansky score. The median follow-up time was 8 years. Conclusion: The PINDA program has allowed 4 patients to be transplan ted who otherwise would not have had access to a donor. This directed donation program could be seen as a model for the development of a public cord blood bank in Chile.

Effect of pretransplant iron overload on clinical efficacy of allogeneic hematopoietic stem cell transplantation on severe aplastic anemia / 器官移植

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.

Research progresses of the way to improve umbilical cord blood stem cells homing and engraftment / 中国医师杂志

Umbilical cord blood is an alternative hematopoietic stem cell source has been widely recognized.Initially,umbilical cord blood transplantation was limited,given the low engraftment.So the method of improving cord blood homing and engraftment has been widely studied in various fields.This paper briefly reviews the progress of main researches in recent years.

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.

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.

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.

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.

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.

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.

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.

Comparison of umbilical cord blood transplantation and hematopoietic stem cell transplantation from HLA-matched sibling donors in the treatment of myelodysplastic syndrome-EB or acute myeloid leukemia with myelodysplasia-related changes / 中华血液学杂志

Objective@#To compare the clinical efficacy of umbilical cord blood transplantation (UCBT) and hematopoietic stem cell transplantation from HLA-matched sibling donors (MSD-HSCT) in the treatment of myelodysplastic syndrome-EB (MDS-EB) or acute myeloid leukemia with myelodysplasia-related changes (AML-MRC) .@*Methods@#A cohort of 64 patients (including 38 cases of MDS-EB and 26 cases of AML-MRC) who received UCBT/MSD-HSCT from February 2011 to December 2017 were retrospectively analyzed.@*Results@#①Compared with MSD-HSCT group, UCBT group had a higher proportion of AML-MRC patients [52.8% (19/36) vs 25.0% (7/28) , P=0.025], and a lower median age [13 (1.5-52) years vs 32 (10-57) years, P=0.001]. ②The engraftment of neutrophils both in UCBT and MSD-HSCT groups on +42 d was 100%, and the median engraftment time was 17.5 (11-31) d and 11.5 (10-20) d, respectively. The engraftment of platelet at +100 d in UCBT group was 91.4%, the median engraftment time was 40 (15-96) d; The engraftment of platelet at +100 d in MSD-HSCT group was 100%, and the median engraftment time was 15 (11-43) d. ③There were no statistically significant differences in terms of the cumulative incidence of Ⅱ-Ⅳ and Ⅲ/Ⅳ aGVHD of 100 d and transplant related mortality (TRM) of 180 d, relapse rate, overall survival (OS) , disease-free survival (DFS) between UCBT and MSD-HSCT groups (P>0.05) . ④The 3-year cumulative incidence of chronic GVHD (cGVHD) and severe chronic GVHD in UCBT group were lower than of MSD-HSCT group [28.3% (95%CI 13.4%-45.3%) vs 67.9% (95%CI 46.1%-82.4%) , P=0.002; 10.3% (95%CI 2.5%-24.8%) vs 50.0% (95%CI 30.0%-67.1%) , respectively, P<0.001]. The cumulative 3-year incidence of GVHD-free and relapse-free survival (GRFS) of UCBT group was significantly higher than of MSD-HSCT group [55.0% (95%CI 36.0%-70.6%) vs 28.6% (95%CI 13.5%-45.6%) , P=0.038].@*Conclusion@#UCBT could obtain better quality of life after transplantation than MSD-HSCT in treatment of MDS-EB/AML-MRC.

Comparison of umbilical cord blood transplantation and hematopoietic stem cell transplantation from HLA-matched sibling donors in the treatment of myelodysplastic syndrome-EB or acute myeloid leukemia with myelodysplasia-related changes / 中华血液学杂志

Objective: To compare the clinical efficacy of umbilical cord blood transplantation (UCBT) and hematopoietic stem cell transplantation from HLA-matched sibling donors (MSD-HSCT) in the treatment of myelodysplastic syndrome-EB (MDS-EB) or acute myeloid leukemia with myelodysplasia-related changes (AML-MRC) . Methods: A cohort of 64 patients (including 38 cases of MDS-EB and 26 cases of AML-MRC) who received UCBT/MSD-HSCT from February 2011 to December 2017 were retrospectively analyzed. Results: ①Compared with MSD-HSCT group, UCBT group had a higher proportion of AML-MRC patients [52.8% (19/36) vs 25.0% (7/28) , P=0.025], and a lower median age [13 (1.5-52) years vs 32 (10-57) years, P=0.001]. ②The engraftment of neutrophils both in UCBT and MSD-HSCT groups on +42 d was 100%, and the median engraftment time was 17.5 (11-31) d and 11.5 (10-20) d, respectively. The engraftment of platelet at +100 d in UCBT group was 91.4%, the median engraftment time was 40 (15-96) d; The engraftment of platelet at +100 d in MSD-HSCT group was 100%, and the median engraftment time was 15 (11-43) d. ③There were no statistically significant differences in terms of the cumulative incidence of Ⅱ-Ⅳ and Ⅲ/Ⅳ aGVHD of 100 d and transplant related mortality (TRM) of 180 d, relapse rate, overall survival (OS) , disease-free survival (DFS) between UCBT and MSD-HSCT groups (P>0.05) . ④The 3-year cumulative incidence of chronic GVHD (cGVHD) and severe chronic GVHD in UCBT group were lower than of MSD-HSCT group [28.3% (95%CI 13.4%-45.3%) vs 67.9% (95%CI 46.1%-82.4%) , P=0.002; 10.3% (95%CI 2.5%-24.8%) vs 50.0% (95%CI 30.0%-67.1%) , respectively, P<0.001]. The cumulative 3-year incidence of GVHD-free and relapse-free survival (GRFS) of UCBT group was significantly higher than of MSD-HSCT group [55.0% (95%CI 36.0%-70.6%) vs 28.6% (95%CI 13.5%-45.6%) , P=0.038]. Conclusion: UCBT could obtain better quality of life after transplantation than MSD-HSCT in treatment of MDS-EB/AML-MRC.

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.