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1.
Med. infant ; 31(2): 147-157, Junio 2024. Tab
文章 在 西班牙语 | LILACS, UNISALUD, BINACIS | ID: biblio-1566859

摘要

Los sobrevivientes de un trasplante alogénico de células progenitoras hematopoyéticas (TACPH) pediátrico presentan alto riesgo de padecer problemas de salud. Debido a esta vulnerabilidad, la continuidad del cuidado impacta en su pronóstico y la transición a la medicina del adulto (TMA) es un proceso clave. Objetivo: Evaluar el proceso actual de TMA de los receptores de TACPH en nuestro hospital. Métodos: Diseño: observacional retrospectivo y prospectivo. Población: todos los pacientes (p) que realizaron su TMA desde enero/2022 a marzo/2023. Instrumentos: entrevista personal; material escrito; resumen de historia clínica; escalas TRAQ 5.0 (transición), PedsQL 4.0 (CVRS) y Lansky (funcionalidad); elección de estrategias de seguimiento según complejidad y requerimientos; contacto con profesionales de adultos; entrevista telefónica luego de 6 meses posTMA; red conformada. Resultados: 36p completaron la TAM (33 presencial, 3 virtual). Edad m19 años (m6 años de seguimiento), 70% del interior del país, 58% TACPH por enfermedad maligna, 64% TACPH familiar. A la TMA: antecedente EICHc 50%, segunda enfermedad maligna 2%, compromiso órganos 75% (m2/p, r0-8, mayormente endocrinológicas, oculares y neurológicas), 94% Lansky ≥80 (r50-100), PedsQL m82 (27% ≤75), TRAQ m3.4 (r1.7- 4.8). Derivación: todos los p cubrían sus necesidades (30% en centros de alta complejidad o expertos en THA) pero 3p debieron readecuar las estrategias, 5p presentaban complicaciones en actividad o necesidad de pronta resolución. Contacto posterior: 30/33p continuaban seguimiento, 3p pudieron retomarlo, 9p nuevas complicaciones/tratamientos. Red: 20 profesionales/instituciones. Conclusiones: Se refuerza la necesidad y utilidad de un proceso de TMA tanto formal como personalizado según necesidades individuales de los pacientes con TACPH (AU)


Pediatric allogeneic hematopoietic stem cell transplant (HSCT) survivors are at high risk for health problems. Because of this vulnerability, continuity of care impacts their prognosis and transition to adult medicine (TAM) is a key process. Objective: To evaluate the current process of TAM of HSCT recipients in our hospital. Methods: A retrospective and prospective observational study was conducted. The population included all patients (p) who underwent TAM from January 2022 to March 2023. Instruments used included personal interviews, written materials, medical history summaries, the TRAQ 5.0 (transition), PedsQL 4.0 (HRQoL), and Lansky (functionality) scales. Follow-up strategies were chosen according to complexity and requirements, with contact established with adult professionals and a telephone interview conducted six months post-TAM in an established network network. Results: 36p completed TAM (33 face-to-face, 3 online). Mean age was 19 years (with a mean of 6 years of follow-up); 70% were from the provinces of the country, 58% underwent HSCT due to malignant disease, 64% had familial HSCT. At TAM: 50% had a history of GVHD, 2% had a second malignant disease, and 75% had organ involvement (mean of 2 per patient, ranging from 0 to 8, mostly endocrinological, ocular, and neurological), 94% had Lansky ≥80 (range, 50-100), mean PedsQL was 82 (27% ≤75), mean TRAQ was 3.4 (range, 1.7-4.8). Referral needs were met for all patients (30% in tertiary-level centers or with experts in allogeneic HSCT), although 3 patients had to readjust strategies, and 5 had complications requiring prompt resolution. In subsequent contact, 30 out of 33 patients continued follow-up, 3 resumed it, and 9 experienced new complications or treatments. The network included 20 healthcare providers/institutions. Conclusions: This study reinforces the need for and usefulness of a formal and personalized TAM process according to the individual needs of patients with HSCT (AU)


Subject(s)
Humans , Adolescent , Quality of Life , Survival , Transplantation, Homologous , Risk Factors , Hematopoietic Stem Cell Transplantation , Transition to Adult Care/organization & administration , Chronic Disease , Prospective Studies , Retrospective Studies , Interview , Treatment Adherence and Compliance
2.
Rev. invest. clín ; Rev. invest. clín;76(2): 91-96, Mar.-Apr. 2024. tab, graf
文章 在 英语 | LILACS-Express | LILACS | ID: biblio-1569950

摘要

ABSTRACT Background: Chronic myelogenous leukemia is a neoplastic proliferation of the granulocytic series. In Mexico, chronic myelogenous leukemia accounts for approximately 10% of all leukemias. Tyrosine-kinase inhibitors are considered front-line therapy in high-income countries, whereas allogeneic hematopoietic stem cell transplantation is a recognized therapeutic approach, mainly in low- and middle-income countries. Objective: To analyze the overall survival of persons with chronic myelogenous leukemia who have received tyrosine-kinase inhibitors or allogeneic hematopoietic stem cell transplantation in a medical center, since 1994, and briefly discuss the current indications of these treatments in the tyrosine-kinase inhibitors era. Methods: We retrospectively analyzed all patients with a diagnosis of chronic myelogenous leukemia treated in a medical center between 1994 and 2023; subsets of individuals who received an allogeneic hematopoietic stem cell transplantation or tyrosine-kinase inhibitors therapy as first-line treatment were analyzed. Results: 60 persons with chronic myelogenous leukemia were treated with allogeneic hematopoietic stem cell transplantation or tyrosine-kinase inhibitors: 35 received an allogeneic hematopoietic stem cell transplantation, whereas 25 were given tyrosine-kinase inhibitors. All patients who underwent an allogeneic hematopoietic stem cell transplantation engrafted successfully, and the procedure was completed on an outpatient basis in most cases (29/35). The median survival in allogeneic hematopoietic stem cell transplantation was 78.3 months (CI 95%: 0-205.6) and in persons given tyrosine-kinase inhibitors the median was not reached. Conclusion: Tyrosine-kinase inhibitors were significantly superior to allogeneic hematopoietic stem cell transplantation in prolonging the overall survival of persons with chronic myelogenous leukemia in our single institution experience. (Rev Invest Clin. 2024;76(2):91-6)

3.
An. Fac. Med. (Perú) ; 85(1): 28-33, ene.-mar. 2024. tab, graf
文章 在 西班牙语 | LILACS-Express | LILACS | ID: biblio-1556797

摘要

RESUMEN Introducción. El trasplante autólogo de células progenitoras hematopoyéticas es una terapia eficaz en neoplasias malignas hematológicas. El número de células que CD34+ en sangre periférica es el mejor predictor del rendimiento de recolección de células progenitoras hematopoyéticas. Objetivo. Determinar el número de células CD34+ en sangre periférica asociado al éxito de recolección de progenitores hematopoyéticos por aféresis en trasplante autólogo. Métodos. Se evaluó retrospectivamente los datos de 236 procedimientos de aféresis de células progenitoras hematopoyéticas para el trasplante autólogo en el Hospital Edgardo Rebagliati Martins (Lima, Perú) de julio del 2020 a julio del 2023. Se utilizó la curva ROC (características operativas del receptor) para determinar el número de células CD34+ en sangre periférica necesario para lograr una recolección por aféresis ≥ 2 x 106 células CD34+/kg. Resultados. El 61% fueron hombres, con mediana de edad de 58 años, el valor de corte fue de 18,38 células CD34+/μL (sensibilidad de 94,1% y especificidad de 96,9%). Conclusión. El número de células CD34+ sangre periférica para una recolección exitosa de células progenitoras hematopoyéticas para el trasplante autólogo fue de 18,38 células CD34+/μL.


ABSTRACT Introduction. Autologous hematopoietic progenitor cell transplantation is an effective therapy in hematological malignancies, the number of CD34+ cells in peripheral blood is the best predictor of hematopoietic progenitor cell harvesting performance. Objective. To determine the number of CD34+ cells in peripheral blood associated with the successful collection of hematopoietic progenitors by apheresis in autologous transplantation. Methods. The data of 236 hematopoietic progenitor cell apheresis procedures for autologous transplantation at the Edgardo Rebagliati Martins Hospital (Lima, Peru) were retrospectively evaluated from July 2020 to July 2023. The ROC (receiver operating characteristics) curve was used to determine the number of CD34+ cells in peripheral blood necessary to achieve an collection by apheresis ≥ 2 x 106 CD34+ cells/kg. Results. 61% were men, with a median age of 58 years, the cut-off value was 18.38 CD34+ cells/μL (sensitivity of 94.1% and specificity of 96.9%). Conclusion. The number of peripheral blood CD34+cells for successful collection of hematopoietic progenitor cells for autologous transplantation was 18.38 CD34+ cells/μL.

4.
Arch. argent. pediatr ; 122(1): e202310061, feb. 2024. tab, ilus
文章 在 英语, 西班牙语 | BINACIS, LILACS | ID: biblio-1525854

摘要

El síndrome de Wiskott-Aldrich es un error innato de la inmunidad de herencia ligada al cromosoma X, producido por variantes en el gen que codifica la proteína del síndrome de Wiskott-Aldrich (WASp). Reportamos el caso clínico de un paciente de 18 meses con diagnóstico de Wiskott-Aldrich que no presentaba donante antígeno leucocitario humano (HLA) idéntico y recibió un trasplante de células progenitoras hematopoyéticas (TCPH) con donante familiar haploidéntico. La profilaxis para enfermedad de injerto contra huésped incluyó ciclofosfamida (PT-Cy). El quimerismo del día +30 fue 100 % del donante y la evaluación postrasplante de la expresión de la proteína WAS fue normal. Actualmente, a 32 meses del trasplante, presenta reconstitución hematológica e inmunológica y quimerismo completo sin evidencia de enfermedad injerto contra huésped. El TCPH haploidéntico con PT-Cy se mostró factible y seguro en este caso de síndrome de WiskottAldrich en el que no se disponía de un donante HLA idéntico.


Wiskott-Aldrich syndrome (WAS) is an X-linked genetic disorder caused by mutations in the gene that encodes the Wiskott-Aldrich syndrome protein (WASp). Here, we report the clinical case of an 18-month-old boy diagnosed with Wiskott-Aldrich syndrome, who did not have an HLA-matched related or unrelated donor and was treated successfully with a hematopoietic stem cell transplant (HSCT) from a haploidentical family donor. Graft-versus-host disease (GvHD) prophylaxis included post-transplant cyclophosphamide (PT-Cy). At day +30, the peripheral blood-nucleated cell chimerism was 100% and the WAS protein had a normal expression. Currently, at month 32 post-transplant, the patient has hematological and immune reconstitution and complete donor chimerism without evidence of GvHD. HSCT with PT-Cy was a feasible and safe option for this patient with WAS, in which an HLA matched donor was not available.


Subject(s)
Humans , Male , Infant , Wiskott-Aldrich Syndrome/diagnosis , Wiskott-Aldrich Syndrome/genetics , Wiskott-Aldrich Syndrome/therapy , Hematopoietic Stem Cell Transplantation/adverse effects , Graft vs Host Disease/etiology , Bone Marrow Transplantation/adverse effects , Cyclophosphamide
5.
JOURNAL OF RARE DISEASES ; (4): 30-35, 2024.
文章 在 英语 | WPRIM | ID: wpr-1006914

摘要

Renal light chain amyloidosis (AL amyloidosis) had poor prognosis before the 21st century. However, the treatment of AL amyloidosis has made great progress in the last decade. We reviewed traditional treatments of AL amyloidosis such as alkylating agents, proteasome inhibitors, and recent advances such as monoclonal antibodies. Bortezomib improved the hematological response and survival effectively of the patients, and the combination of Daratumumab brings faster and deeper hematological response, increasing the response rate of target organs such as the kidneys and heart. The renal response was significant higher in the patients with the therapy of Daratumumab, part of them could achieve very good partial response or better renal response. Autologous hematopoietic stem cell transplantation(auto-HSCT)improves hematological as well as organ response, and could be the first choice among eligible patients. Kidney transplantation is a feasible option for those with good hematological response.

6.
文章 在 中文 | WPRIM | ID: wpr-1006420

摘要

Hepatic sinusoidal obstruction syndrome (HSOS), also known as hepatic veno-occlusive disease, is hepatic vascular disease of hepatic sinusoidal obstruction and hepatic venular occlusion and fibrosis due to various causes. This article systematically elaborates on the research advances in HSOS from the aspects of understanding and naming, etiology and pathogenesis, clinical manifestation, diagnosis and differential diagnosis, prevention, and treatment. HSOS can occur in patients receiving bone marrow hematopoietic stem cell transplantation, radiotherapy/chemotherapy, and medication containing pyrrolidine alkaloids, and the common clinical manifestations of HSOS include abdominal distension, distending pain in the liver area, ascites, jaundice, and hepatomegaly. The diagnostic criteria for HSOS vary with etiology, and it needs to be differentiated from other diseases such as drug-induced liver diseases and hepatic venous outflow tract obstruction. Defibrotide and low-molecular-weight heparin have a therapeutic effect on HSOS associated with hematopoietic stem cells and pyrrolidine alkaloids, respectively, and there are currently no effective drugs for HSOS caused by oxaliplatin chemotherapy.

7.
文章 在 中文 | WPRIM | ID: wpr-1024093

摘要

Cytomegalovirus(CMV)pneumonia is one of the common complications of hematopoietic stem cell transplantation,and is also a significant cause leading to patient death.Due to the poor prognosis of CMV pneumo-nia,intervention measures are necessary to prevent CMV reactivation and progress to CMV pneumonia.At present,clinical medication mainly relies on preemptive treatment strategies,and the timing of medication depends on the timeliness of early diagnosis.However,feasible methodology and measures for the early diagnosis of CMV pneumo-nia in clinical practice are relatively limited.Meanwhile the diagnostic gold standard operation is invasive,causing trauma to a certain degree,and the detection timeliness is poor.This review summarizes the clinical status and ad-vances in the diagnosis and drug prophylactic treatment of CMV pneumonia after hematopoietic stem cell transplan-tation,and explores possible development directions and trends in the future.

8.
文章 在 中文 | WPRIM | ID: wpr-1024976

摘要

【Objective】 To explore the risk factors for the production of anti-HLA antibodies in patients with hematological diseases before hematopoietic stemcell transplantation. 【Methods】 The results and clinical data of 1 008 patients with hematological diseases in our hospital who underwent anti-HLA antibody testing were collected by using Luminex technology platform before transplantation from 2016 to 2018 for statistical analysis. 【Results】 The total positive rate of anti-HLA antibodies in 1 008 patients was 24.08%. Multivariate analysis showed that independent risk factors associated with the production of anti-HLA antibodies included age≥30 years old(P=0.046, OR1.467, 95%CI1.007-2.136), time from disease diagnosis to antibody testing≥41 days(P=0.000, OR1.830, 95%CI1.306-2.565), initial platelet count<20×109/L(P=0.020, OR1.543, 95%CI1.072-2.220), prior pregnancy(P=0.000, OR5.187, 95%CI3.689-7.293), transfusions before admission(P=0.001, OR1.762, 95%CI1.257-2.470)and total platelet transfusion volumes after admission≥30 U(P=0.000, OR2.352, 95%CI1.638-3.376). Age ≥30 years old(P=0.023, OR=1.839, 95%CI1.088-3.108)and prior pregnancy(P=0.042, OR=5.258, 95%CI1.062-26.038)are associated with the production of anti-HLA class Ⅰ and class Ⅱ antibodies, respectively. The time from disease diagnosis to antibody testing≥41 days(P=0.000, OR=2.873, 95%CI1.612-5.119), initial platelet count<20×109/L(P=0.008, OR=2.164, 95%CI1.225-3.822), prior pregnancy(P=0.002, OR=6.734, 95%CI1.993-22.751), transfusions before admission(P=0.001, OR=2.746, 95%CI1.531-4.925)and total platelet transfusion volumes after admission>30 U(P=0.006, OR=3.459, 95%CI1.416-8.451)are associated with the production of anti-HLA class Ⅰ and Ⅱ antibodies. 【Conclusion】 Older age, longer course of disease, lower PLT count, history of pregnancy and blood transfusion, and higher total amount of PLT transfusion are risk factors which affect the production of anti-HLA antibodies.Therefore, it is advisable to test for anti-HLA antibodies according to the situation before transplantation, which is of great value in guiding donor selection, monitoring antibody changes and improving transplant prognosis.

9.
文章 在 中文 | WPRIM | ID: wpr-1027396

摘要

Objective:To investigate the effects of sleep disorders (SD) on the radiation injury of hematopoietic stem cells (HSCs) in bone marrow (BM).Methods:Totolly 56 C57BL/6J male mice aged 6-8 weeks were enrolled in this study. They were subjected to whole body irradiation of 60Co γ-rays with doses of 5.0 and 7.5 Gy. A SD model was established using a SD device. According to the random number table method, the mice were divided into seven groups: the control group (Con group), the SD group, the mere radiation group (IR group), the group of post-irradiation SD (IR+ SD group), the group of post-irradiation SD treated with phosphate buffer solution (IR+ SD+ PBS group), the group of post-irradiation SD treated with GSK2795039 (IR+ SD+ GSK group), and the group of post-irradiation SD treated with N-acetylcysteine (IR+ SD+ NAC group), with in eight mice each group. The changes in the peripheral blood of the mice after 5.0 Gy irradiation were detected using the collected tail venous blood, and the survival rates of the mice after 7.5 Gy irradiation were observed. The changes in the density and count of bone marrow cells were observed using hematoxylin and eosin (HE) staining. The number of hematopoietic stem cells in bone marrow (LSK cells), as well as their apoptosis level and changes in cell cycle, were detected using flow cytometry. Furthermore, indicators of LSK, such as reactive oxygen species(ROS) and mitochondrial-derived reactive oxygen species (mtROS), were analyzed. Nicotinamide adenine dinucleotide phosphate (NADP+ /NADPH) and glutathione (GSSG/GSH) were detected using an enzyme microplate reader in order to observe the oxidative stress level of LSK. Furthermore, flow cytometry was employed to sort the LSK cells from the mice, and flow cytometry was used to detect the expression of NADPH oxidase 2(NOX2) and cysteinyl aspartate specific proteinnase-1(Caspase-1), and polymerase chain reaction (PCR) was used to detect the expression of inflammatory factors such as NOX1-4, interleukin 1β (IL-1β), interleukin 6 (IL-6), interleukin 18 (IL-18), and tumor necrosis factor α (TNF-α). Results:Compared to the IR group, the IR+ SD group exhibited significantly slower recovery of white blood cells (WBC) and platelets (PLT) ( t = 4.39, 6.37, P < 0.05), the bone marrow cell count decreasing from (2.14 ± 0.38) × 10 7 to (3.59 ± 0.29) × 10 7 ( t = 8.55, P < 0.05), significantly decreased proportion of G 0-phase LSK cells, significantly increased proportion of apoptotic cells ( t = 7.53, 8.21, P < 0.05), and significantly increased DCFH-DA, MitoSOX, and NADP+ /NADPH ( t = 22.99, 29.47, 3.77, P<0.05). In the case of IR, SD further promoted the activation of NOX2 and led to increases in the mRNA expression of downstream inflammatory factors such as IL-1β, IL-6, IL-18, and TNF-α ( t = 6.95, 6.01, 8.39, 4.91, 5.56, P < 0.05). Inhibition of NOX2-ROS could prevent the SD-induced aggravation of post-irradiation hematopoietic injury. This significantly reduced the apoptotic rate of LSK cells and the expression of inflammatory factors, ultimately accelerating the hematopoietic recovery of LSK cells ( t = 9.24, 3.92, P < 0.05). Conclusions:SD can aggravate the IR-induced injury of hematopoietic stem cells in bone marrow, primarily by activating the NOX2-ROS-Caspase-1 axis. This will increase the levels of intracellular inflammatory factors and ROS, promote cell apoptosis, and ultimately inhibit the hematopoietic recovery of bone marrow.

10.
文章 在 中文 | WPRIM | ID: wpr-1028680

摘要

Objective:To verify the predictive value of the Second Revision of the International Staging System (R2-ISS) in newly diagnosed patients with multiple myeloma (MM) who underwent first-line autologous hematopoietic stem cell transplantation (ASCT) in a new drug era in China.Methods:This multicenter retrospective cohort study enrolled patients with newly diagnosed MM from three centers in China (Beijing Chao-Yang Hospital, Capital Medical University; the First Affiliated Hospital, Sun Yat-Sen University, and the Second Affiliated Hospital of Naval Medical University) from June 2008 to June 2018. A total of 401 newly diagnosed patients with MM who were candidates for ASCT were enrolled in this cohort, all received proteasome inhibitor and/or immunomodulator-based induction chemotherapy followed by ASCT. Baseline and follow-up data were collected. The patients were regrouped using R2-ISS. Progression-free survival (PFS) and overall survival (OS) were analyzed. The Kaplan-Meier method was used to analyze the survival curve and two survival curves were compared using the log-rank test. Cox regression analysis were performed to analyze the relationship between risk factors and survival.Results:The median age of the patients was 53 years (range 25-69 years) and 59.5% (240 cases) were men. Newly diagnosed patients with renal impairment accounted for 11.5% (46 cases). According to Revised-International Staging System (R-ISS), 74 patients (18.5 %) were diagnosed with stage Ⅰ, 259 patients (64.6%) with stage Ⅱ, and 68 patients (17.0%) with stage Ⅲ. According to the R2-ISS, the distribution of patients in each group was as follows: 50 patients (12.5%) in stage Ⅰ, 95 patients (23.7%) in stage Ⅱ, 206 patients (51.4%) in stage Ⅲ, and 50 patients (12.5%) in stage Ⅳ. The median follow-up time was 35.9 months (range, 6-119 months). According to the R2-ISS stage, the median PFS in each group was: 75.3 months for stage Ⅰ; 62.0 months for stage Ⅱ, 39.2 months for stage Ⅲ, and 30.3 months for stage Ⅳ; and the median OS was not reached, 86.6 months, 71.6 months, and 38.5 months, respectively. There were statistically significant differences in PFS and OS between different groups (both P<0.001). Multivariate Cox regression analysis showed that stages Ⅲ and Ⅳ of the R2-ISS were independent prognostic factors for PFS ( HR=2.37, 95% CI 1.30-4.30; HR=4.50, 95% CI 2.35-9.01) and OS ( HR=4.20, 95% CI 1.50-11.80; HR=9.53, 95% CI 3.21-28.29). Conclusions:The R2-ISS has significant predictive value for PFS and OS for transplant-eligible patients with MM in the new drug era. However, the universality of the R2-ISS still needs to be further verified in different populations.

11.
International Eye Science ; (12): 895-898, 2024.
文章 在 中文 | WPRIM | ID: wpr-1030815

摘要

Allogeneic hematopoietic stem cell transplantation(allo-HSCT)is a treatment for many malignant tumors of blood system and severe immunodeficiency, and chronic graft-versus-host disease(cGVHD)is one of the major complications after transplantation, which seriously affects the life quality of patients. The cGVHD can attack all the target tissues of the eye. The more common ones are lacrimal gland, eyelid, conjunctiva, cornea and limbus, meibomian gland, etc. The pathophysiological process is inflammation and fibrosis. Although many researchers at home and abroad have gradually begun to explore the disease and obtained many new findings, its pathology and pathogenesis are still not fully understood, and there is no unified and effective treatment. Clinically, the treatment of the disease is usually when symptoms have appeared. At this time, the target tissue is likely to have irreversible and permanent damage, resulting in a protracted and refractory situation. Therefore, this thesis mainly clarifies the research progress of the pathology and pathogenesis of ocular cGVHD in recent years, in order to provide new guidance for the treatment of the disease.

12.
Journal of Modern Urology ; (12): 122-125, 2024.
文章 在 中文 | WPRIM | ID: wpr-1031666

摘要

【Objective】 To improve the understanding and diagnosis and treatment level of ALK negative anaplastic large cell lymphoma (ALK-ALCL) by sharing the diagnosis and treatment process of a patient with ALK-ALCL treated in Hangzhou Bay Hospital of Ningbo. 【Methods】 The clinical data and diagnosis and treatment process of the patient were retrospectively analyzed, and relevant literature was reviewed. 【Results】 The patient was a young male, with recurrent gross hematuria and right low back pain as the initial symptoms.Imaging examination indicated bladder tumor.After resection, the tumor was reduced and confirmed to be ALK-ALCL.After chemotherapy and autologous hematopoietic stem cell transplantation, the patient’s condition continued to improve.During the follow-up, no recurrence was observed. 【Conclusion】 Primary ALK-ALCL in the bladder is very rare and prone to misdiagnosis and missed diagnosis in clinical practice.The successful diagnosis and treatment experience of this patient can provide clinical reference.

13.
JOURNAL OF RARE DISEASES ; (4): 30-35, 2024.
文章 在 中文 | WPRIM | ID: wpr-1032063

摘要

Renal light chain amyloidosis (AL amyloidosis) had poor prognosis before the 21st century. However, the treatment of AL amyloidosis has made great progress in the last decade. We reviewed traditional treatments of AL amyloidosis such as alkylating agents, proteasome inhibitors, and recent advances such as monoclonal antibodies. Bortezomib improved the hematological response and survival effectively of the patients, and the combination of Daratumumab brings faster and deeper hematological response, increasing the response rate of target organs such as the kidneys and heart. The renal response was significant higher in the patients with the therapy of Daratumumab, part of them could achieve very good partial response or better renal response. Autologous hematopoietic stem cell transplantation(auto-HSCT)improves hematological as well as organ response, and could be the first choice among eligible patients. Kidney transplantation is a feasible option for those with good hematological response.

14.
Organ Transplantation ; (6): 449-455, 2024.
文章 在 中文 | WPRIM | ID: wpr-1016911

摘要

<b>Objective</b> To evaluate clinical efficacy of lung transplantation for lung chronic graft-versus-host disease (cGVHD) after hematopoietic stem cell transplantation (HSCT). <b>Methods</b> Clinical data of 12 patients undergoing lung transplantation for lung cGVHD were retrospectively analyzed. Preoperative clinical manifestations and involved organs of patients were analyzed. The lung function before and after lung transplantation was compared, and the survival of patients after lung transplantation was analyzed. <b>Results</b> Eleven patients underwent HSCT due to primary hematological malignancies, including 9 cases of leukemia, 1 case of myelodysplastic syndrome, 1 case of lymphoma. And 1 case underwent HSCT for systemic lupus erythematosus. Among 12 cGVHD patients, skin involvement was found in 8 cases, oral cavity involvement in 5 cases, gastrointestinal tract involvement in 4 cases and liver involvement in 3 cases. All 12 patients developed severe respiratory failure caused by cGVHD before lung transplantation, including 9 cases of typeⅡ respiratory failure and 3 cases of type Ⅰ respiratory failure. Two patients underwent right lung transplantation, 2 cases of left lung transplantation and 8 cases of bilateral lung transplantation. The interval from HSCT to lung transplantation was 75 (19-187) months. Upon the date of submission, postoperative follow-up time was 18 (7-74) months. Ten patients survived, 1 died from severe hepatitis at postoperative 22 months, and 1 died from gastrointestinal bleeding at postoperative 6 months. No recurrence of primary diseases was reported in surviving patients. <b>Conclusions</b> Lung transplantation is an efficacious treatment for lung cGVHD after HSCT, which may prolong the survival time and improve the quality of life of the recipients.

15.
Journal of Leukemia & Lymphoma ; (12): 189-192, 2024.
文章 在 中文 | WPRIM | ID: wpr-1017402

摘要

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a curative method for various hematological diseases. With the optimization of transplantation technology, the clinical application of allo-HSCT is more and more mature. Post-transplant liver injury is one of the common postoperative complications, which seriously affects the quality of life and long-term survival of patients. The causes of liver injury after allo-HSCT can be divided into non-infectious and infectious factors, which show similar clinical manifestations and different treatment principles. Timely diagnosis of post-transplant liver injury and the identification of the disease cause will be beneficial for early prevention or targeted treatment, thereby improving patients' prognosis. This review focuses on the etiology, clinical features, and treatment options of liver injury after allo-HSCT, aiming to deepen the understanding of hematologists on liver injury after allo-HSCT.

16.
Journal of Army Medical University ; (semimonthly): 311-318, 2024.
文章 在 中文 | WPRIM | ID: wpr-1017563

摘要

Objective To analyze the clinical outcomes of early invasive fungal disease(IFD)in patients after allogenetic hematopoietic stem cell transplantation(allo-HCST)with metagenomic next-generation sequencing(mNGS).Methods A retrospective analysis was conducted on patients undergoing allo-HCST in our Bone Marrow Transplantation Center between July 2021 and October 2022.These patients experienced one of the following conditions within 100 d after transplantation:① Patients with persistent fever and negative blood culture after empiric antimicrobial therapy for 72 h or longer;② Hyperpyrexia of unknown origin occurred again after effective anti-infection in the past;③ Symptoms in lower respiratory tract associated with lung lesions on CT scan,and empiric anti-infective therapy was ineffective.Peripheral blood or bronchoscopic alveolar lavage fluid were tested with mNGS,and overall survival(OS)and non-relapse mortality(NRM)were analyzed.Results There were 60 patients enrolled in this study.For the peripheral blood samples of 47 cases and bronchoalveolar lavage fluid samples of 13 cases,mNGS found that 19 cases were negative to pathogens,30 cases were non-fungal positive,and 11 case were fungal positive,including 3 cases of aspergillus,5 cases of mucor,2 cases of Candida tropicalis,and 1 case of Trichosporon asahii.Of the 11 patients with fungal positive,8 achieved complete remission after antifungal therapy according to the mNGS results.The 1-year OS and NRM of the 60 patients were 70.0%(95%CI:64.1%~75.9%)and 20.0%(95%CI:11.9%~32.5%),respectively,while those of the fungal infection patients were 54.5%(95%CI:49.5%~69.5%)and 36.4%(95% CI:15.5%~70.3%),respectively.No significant differences were seen in 1-year OS(P=0.487)and 1-year NRM(P=0.358)among the negative,fungal infection and non-fungal infection patients,neither OS(P=0.238)and NRM(P=0.154)between the fungal infection and the non-fungal infection patients.Conclusion mNGS can rapidly diagnose the early IFD after allo-HSCT,which is helpful for timely and effective treatment and improves the prognosis of patients.

17.
Journal of Army Medical University ; (semimonthly): 319-325, 2024.
文章 在 中文 | WPRIM | ID: wpr-1017564

摘要

Objective To investigate the effects of anti-HLA donor-specific antibodies(DSA)and desensitization for DSA+patients on engraftment of haploidentical hematopoietic stem cell transplantation(haplo-HSCT).Methods The patients who underwent haplo-HSCT and examinations for HLA antibodies and DSA in our department from March 2017 to July 2023 were recruited in this study.The effects of desensitization measure on engraftment in the DSA+patients after haplo-HSCT were analyzed.Results Among the 70 patients who underwent haplo-HSCT and test for HLA antibodies,15(21.4%)patients were DSA positive,including 7(46.7%)cases of strong positive,3(20.0%)cases of moderate positive,and 5(33.3%)cases of weak positive.The median duration for neutrophil implantation was significantly extended in the DSA+patients than the negative patients(P=0.027).For the 6 patients developed graft failure(GF),4 were DSA+which was statistically higher than the DSA-patients(P=0.025).Multivariate regression analysis showed that DSA was an independent factor affecting GF(HR=9.273,95%CI:1.505~57.124,P=0.016).Among the 10 patients(7 strong positive and 3 moderate positive DSA)received desensitization therapy,4 patients received combination desensitization,with a 100%rate of successful transplantation,and 6 received single desensitization,with 4(66.7%)experiencing GF,so the GF rate was obviously lower in the combination than the single desensitization(P=0.008).Conclusion In haplo-HSCT patients,DSA is an important factor leading to implantation delay and GF.While,single desensitization treatment has limited efficacy.In combined DSA desensitization therapy,the decrease of antibody titer should be dynamically monitored to ensure the successful implantation of stem cells and reduce GF rate.

18.
Journal of Army Medical University ; (semimonthly): 331-339, 2024.
文章 在 中文 | WPRIM | ID: wpr-1017566

摘要

Objective To preliminarily investigate the association between changes in intestinal microbiota and oral microbiota of allogeneic hematopoietic stem cell transplantation(allo-HSCT)patients and early gastrointestinal acute graft versus host disease(aGVHD),and try to explore potentially effective biomarkers and provide theoretical basis for early prediction and intervention of gastrointestinal aGVHD.Methods Ten acute leukemia patients who developed gastrointestinal aGVHD within 1 month after receiving allo-HSCT in Department of Hematology of Sichuan Provincial People's Hospital from September 2021 to June 2023 were enrolled,and their fecal samples and saliva samples before and after the aGVHD were collected.16S rRNA sequencing analysis was applied for the differential changes in intestinal and oral microbiota before and after the development of early gastrointestinal aGVHD.Results ① A decrease in Bacteroides spp.and an increase in Enterococcus spp.and Enterobacteriaceae spp.in the intestinal microbiota were positively correlated with the occurrence of early upper gastrointestinal aGVHD(P<0.05),whereas no significant difference was observed in the overall microbial diversity of the oral microbiota(P>0.05).② LEfSe analysis of the intestinal microbiota before and after gastrointestinal aGVHD revealed an increase in Klebsiella spp.and Enterococcus spp.and a decrease in Escherichia coli;In the oral microbiota,LEfSe analysis revealed 10 microbial markers with significant difference,of which Gamma proteobacteria was the most significant.③ The difference in β-diversity of the intestinal microbiota was significant(P=0.03),whereas there was no significant difference in the α-and β-diversity of the oral microbiota.Conclusion Significant differences are found in intestinal microbiota before and after the occurrence of early gastrointestinal aGVHD in patients after Allo-HSCT,and the occurrence may have a correlation with the chang in oral microbiota.

19.
文章 在 中文 | WPRIM | ID: wpr-1017720

摘要

Acute leukemia(AL)is a common hematological malignancy in children and adolescents. Chemotherapy is currently the primary treatment for AL.Alternative therapies,such as hematopoietic stem cell transplantation(HSCT),targeted therapy,and immunotherapy also offer greater hope for the survival of refractory/relapsed patients. Chemotherapeutic drugs,radiotherapy,targeted drugs and immunotherapeutic drugs are well-applied clinically,meanwhile posing threats to non-target systems. The adverse effects on the reproductive system may lead to the dilemma of infertility,thus reducing the long-term quality of life. As the survival rate of AL patients keeps increasing continuously,the influence of different treatments on the gonad function needs to be clarified. With the help of targeted fertility prevention,the patient′s quality of life can be enhanced in parallel with life span. This article aims to review the impact of AL treatment on ovarian function in female children and adolescents and provide ideas for the long-term fertility protection of leukemia patients.

20.
文章 在 中文 | WPRIM | ID: wpr-1020436

摘要

This paper provided a comprehensive review of the concept, current situation and influencing factors of survivors returning to work after hematopoietic stem cell transplantation, both in domestic and international contexts. The research analyzed and elaborates on four aspects of individual factors, disease-related factors, occupational factors, and social support, in order to provide references for constructing an intervention program for the return to work of hematopoietic stem cell transplantation survivors based on the cultural background in China.

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