Autologous Stem Cell Transplantation Research Articles

Induction Chemotherapy-Related Covert Cardiac Remodeling in Pre-Autologous Hematopoietic Stem Cell Transplantation for Multiple Myeloma: A Retrospective Observational Study.

Autologous hematopoietic stem cell transplantation (ASCT) has emerged as a cornerstone in multiple myeloma (MM) management, offering the prospect of prolonged disease control. However, the induction chemotherapy drugs required prior to ASCT carry cardiovascular toxicity (CVT), potentially leading to a range of cardiovascular complications. This retrospective observational study, conducted at Guangdong Provincial People's Hospital from January 2020 to December 2023, analyzed 47 of the initial 173 patients who met the criteria. The cohort, comprising 22 males (46.81%) and 25 females (53.19%), had a mean age of 55.68 ± 11.38 years. They underwent various induction chemotherapy regimens, receiving a median of 5 (4-6) cycles of the course over an average duration of 7.10 ± 2.46 months. Before ASCT treatment following induction chemotherapy, echocardiographic findings indicated reductions in left ventricular end-systolic dimension, right atrial diameter, E-wave velocity, E/e' ratio, and the E/A ratio. The latter altered from a pretreatment value greater than 1 to posttreatment less than 1, marking diastolic dysfunction emergence or aggravation in 51.06% of patients. The electrocardiographic data indicate a reduced heart rate and prolonged P-wave duration and P-R duration, with an increase in arrhythmia incidence to 19.15% following induction chemotherapy. Induction chemotherapy, administered prior to ASCT in patients with MM, can lead to the emergence or aggravation of cardiac diastolic dysfunction and increase the incidence of arrhythmic events. Therefore, it is crucial to emphasize the importance of balancing the benefits and risks of induction chemotherapy to maximize its efficacy while minimizing CVT.

Clinical efficacy and safety of parenteral nutrition in hematopoietic stem cell transplantation: results of a single-center randomized controlled study

BACKGROUND: Hematopoietic stem cell transplantation frequently necessitates nutritional support. However, the safety and tolerance of parenteral nutrition have not been adequately examined, except for its association with infectious complications. AIM: To evaluate the impact of parenteral nutrition on nutritional status and its correlation with complications during the early period following hematopoietic stem cell transplantation. MATERIALS AND METHODS: From 2019–2020, 125 recipients of autologous (n=38) and allogeneic (n=87) hematopoietic stem cell transplantation were included in the study with a median age of 20 years (2–65). The chosen cohort included patients with leukemia (n=67), solid tumors (n=32), dysplasia (n=15) and lymphoproliferation (n=11). The control group (n=55) was composed of patients who did not depend on nutritional support. Parenteral nutrition was administered to the primary group (n=70) using second-generation (n=22) and third-generation (n=24) fat emulsions. In the event of contraindications, parenteral nutrition was administered without fat emulsions (n=24). To mitigate the risk of side effects, the initial dose of parenteral nutrition was 50%, with the achievement of the complete dose by the third day of therapy. RESULTS: In 95.7% of cases, mucositis was the indication for parenteral nutrition. The duration of parenteral nutrition was 2–31 days (median, 11 days). In 58.6% of cases, parenteral nutrition was initiated against the background of nausea and vomiting. During the escalation of the parenteral nutrition dose, increased nausea was observed in 7.1% of patients. Side effects were noted in 22.9% of cases: hyperglycemia (n=7), vomiting (n=6) and hypertriglyceridemia (n=3). Four patients developed complications associated with parenteral nutrition, and nutritional therapy was discontinued in 20% of cases (n=14). After hematopoietic stem cell transplantation, the primary clinical efficacy criterion, body mass index, was observed to decline in each observation group. However, it recovered by day 28 and did not differ between the comparison groups as follows: the control group — from 22.7±6.4 to 21.7±6.1 kg/m2, (p=0.42); group “2 in 1” — from 23.4±6.1 to 21.6±4 kg/m2, (p=0.56); group provided with parenteral nutrition employing second-generation fat emulsions — from 18,8±4,9 to 18.7±4.4 kg/m2, (p=0.72); group administered parenteral nutrition with third-generation fat emulsions — from 18.6±4.2 to 18.3±3.5 kg/m2, (p=0.84). The febrile neutropenia rate was 75.2% and did not vary in the control and comparison groups (p=0.54). The incidence of sepsis was 24.8% and was not influenced by the use of parenteral nutrition (p=0.07). Furthermore, sepsis risk was higher when administering parenteral nutrition “2 in 1” compared to fat emulsions-containing parenteral nutrition (p=0.002). The overall frequency of acute graft versus host disease was 16.1% and was similar in the comparison groups (p=0.65). CONCLUSION: In the cohort of patients treated with hematopoietic stem cell transplantation, parenteral nutrition regimens supplemented with second- and third-generation fat emulsions with a stepwise dose increase varied in acceptable tolerance, infection-related complications, and immunological safety, as well as effectively maintained nutritional status parameters.

Characteristics and outcomes of therapy-related acute leukemia following autologous transplant (Auto-HCT) for multiple myeloma.

With a prolonging duration of survivorship, patients with multiple myeloma (MM) who receive high-dose chemotherapy and autologous hematopoietic stem cell transplantation (auto-HCT) have an increased risk of secondary malignancy, most concerning acute leukemia. We retrospectively reviewed the records of all patients with MM who underwent auto-HCT between January 1, 2010, and January 1, 2023, who later developed therapy-related acute leukemia (t-AL). Of 1770 patients with MM who underwent auto-HCT, 18 (1.01%) developed t-AL at a mean interval of 60.0 ± 41.3 months after auto-HCT. The patients with t-AL consisted of 9 (50%) with B-cell acute lymphoblastic leukemia (B-ALL), 8 (44.4%) with acute myeloid leukemia (AML), and 1 (5.6%) with acute promyelocytic leukemia (APML). All patients had received an alkylating agent as part of induction, and the majority received lenalidomide as maintenance therapy. Genetic abnormalities of t-AL were consistent with prior reports. Median overall survival from diagnosis of t-AL was 19.5 months. In patients with t-AL who entered CR, long term survival was common. Further research on predisposing conditions to developing t-AL in patients with MM undergoing auto-HCT is warranted.

Ulcerative Jejunitis in Celiac Disease: A Thirty-Year U.S. Experience.

Ulcerative jejunitis (UJ) or ulcerative enteritis (UE) is a rare complication of celiac disease (CeD). Guidelines regarding diagnosis and management are missing and these cases have seldom been reported in the United States. Single center case-series of CeD in which UE developed at a large academic center in the USA. Clinical presentation, diagnosis, treatment, and evolution of disease were collected. Eight cases were identified (6M/2F, mean age 59.5 (38-77) years). Presentations included intestinal obstruction (n=3), GI hemorrhage (n=3), and malabsorption (n=2). Ulcers were present in the duodenum in 4 patients, and exclusively past the angle of Treitz in only 4 cases, which makes the term ulcerative enteritis (UE) more appropriate than UJ. Six out of eight had T-cell receptor (TCR) clonal gene rearrangements and two had definite aberrant T cells. Corticosteroids were tried in all patients without improvement and 5 underwent surgical resection. Three patients received cladribine. One patient received an autologous stem cell transplant, followed by ruxolitinib. Two were subsequently diagnosed with enteropathy-associated T-cell lymphoma (EATL), including one with cerebral EATL, and 1 died from hemophagocytic syndrome. Two are still alive, including one only on GFD and two were lost to follow-up after surviving at least 30 months post treatment. UE seems a more appropriate term to describe an ulcerative complication of CeD at high risk of obstruction or bleeding. Steroids were not effective. Treatment outcomes were variable, but with a 50% death rate.

Salvage Treatment for Extragonadal Germ Cell Tumours: High-Dose Chemotherapy and Autologous Stem Cell Transplantation Outcomes-A Single-Centre Experience.

Objective: Extragonadal germ cell tumours have a more unfavourable prognosis than gonadal germ cell tumours. We aimed to evaluate the survival analysis, response rates, and factors affecting responses to high-dose chemotherapy (HDCT) and autologous stem cell transplantation (ASCT) in patients with relapsed/refractory extragonadal germ cell tumours. Methods: This study included patients diagnosed with extragonadal germ cell tumours who underwent HDCT + ASCT between November 2016 and January 2023 at Gülhane Training and Research Hospital. Clinical characteristics and follow-up data from patient records and the hospital's electronic system were retrospectively analysed. Patients under 18 years of age and those without medical records were excluded. Patient characteristics, post-HDCT progression-free survival (PFS), overall survival (OS) data, and factors affecting survival were examined. The relationship between clinical factors and OS/PFS was analysed. Results: Twenty-five patients were included in this study. Complete response (CR) was observed in seven patients (28%), partial response (PR) was observed in nine patients (36%), stable disease (SD) was observed in one patient, and progressive disease (PD) was observed in eight patients (32%) after HDCT + ASCT. The median follow-up period was 25.4 months. The median PFS and OS after HDCT + ASCT were calculated as 6.1 months and 12.2 months, respectively. Conclusions: Salvage HDCT + ASCT is an option in the treatment of extragonadal germ cell tumours, offering the potential for prolonged survival and curing.

BACE (Bendamustine, Cytarabine, Cyclophosphamide and Etoposide) conditioning regime for patients with lymphoma undergoing autologous stem cell transplant

BACE (Bendamustine, Cytarabine, Cyclophosphamide and Etoposide) conditioning regime for patients with lymphoma undergoing autologous stem cell transplant

Outcomes in patients with light chain cardiac amyloidosis undergoing heart transplantation: a single centre UK study

Abstract Background Light chain cardiac amyloidosis (AL-CA) results in restrictive cardiomyopathy associated with progressive heart failure and poor survival. Traditionally, outcomes following heart transplantation in patients with AL-CA have been worse than in other heart failure cohorts and consequently, many centres do not offer this population transplantation. Purpose and Methods This was a retrospective observational study of 18 patients with AL-CA (age 52.7years +/- 8.3, male 66%) referred to our centre in the UK for assessment for heart transplantation from 2008 to 2023. Seven patients (39%) were listed for transplant, and all received orthotopic heart transplantation (OHT). This study compared pre-transplantation characteristics in those offered cardiac transplantation with those not transplanted. We also examined post-transplantation outcomes in this group (n=7) compared with the overall heart transplantation population (n=95) at our centre. Results Patients with AL-CA who were not offered transplantation (n=11) were older (mean age 54.3 vs 50.3 years), were more likely male (72% vs 57%) but had similar baseline renal function to those transplanted (median eGFR 60.4 vs 54.2ml/min). Reasons against offering OHT included extensive multisystem or renal amyloid deposition (n=4), insufficient haematological response (n=2), heart failure not advanced enough for OHT (n=1) and malignancy (n=1). Pre-transplant right heart catheterisation of patients undergoing OHT demonstrated a mean transpulmonary gradient of 7.8mmHg (+/- 1.9) and cardiac output of 2.94/min (+/- 0.3). Five patients (71%) had a complete and two (29%) a partial haematological response to clonally directed chemotherapy. Three patients were bridged to transplant with biventricular assist devices for 9, 10 and 51 days, respectively. Five patients developed episodes of histologically confirmed rejection of grade 2R that resolved with medical management. One patient experienced first degree atrioventricular block and had a pulseless electrical activity arrest 19 days after transplantation before being fitted with a permanent pacemaker. Two patients went on to receive autologous stem cell transplantation. Patients with AL-CA undergoing OHT had a lower mortality rate compared with non-transplanted patients over a median follow up of 9 months (HR 0.3, 95%CI 0.07-1.3, p = 0.10). Two patients with AL-CA died at 42 days and 7.7 years after OHT. Five patients (71%) are alive under follow up at 4, 7.5, 9, 10 months and 7.7 years post OHT. There was no difference in survival between patients who underwent heart transplantation for AL-CA and patients who underwent heart transplantation for all other indications (HR 1.94, 95%CI 0.3 – 12, p=0.45). Conclusions In selected patients with AL-CA, OHT offers favourable outcomes. After clonally directed therapy to control the underlying plasma cell dyscrasia, heart transplantation should be considered in the absence of widespread systemic disease.

High-Dose Chemotherapy and Autologous or Allogeneic Transplantation in Aggressive B-Cell Lymphoma-Is There Still a Role?

Novel therapies such as CAR-T, BTK inhibitors and PD-1 inhibitors have changed the management of aggressive B-cell lymphomas. Nonetheless, these novel therapies have their own risk of late toxicities including second malignancies. They also create a subgroup of patients with relapse, treatment failure, or indefinite maintenance. We discuss the current role of autologous and allogeneic stem cell transplantation in this context. In patients with recurrent diffuse large B-cell lymphoma, CAR-T cell treatment has largely replaced autologous transplant. Autologous transplant should be considered in patients with late relapses and in selected patients with T-cell-rich B-cell lymphoma, where CAR-T cell therapy may be less effective. It also remains the treatment of choice for consolidation of patients with primary CNS lymphoma. In mantle cell lymphoma, intensive chemotherapy combined with BTK inhibitors and rituximab results in excellent outcomes, and the role of autologous transplantation is declining. In Hodgkin's lymphoma, autologous transplant consolidation remains the standard of care for patients who failed initial chemotherapy. Allogeneic transplantation has lower relapse rates but more complications and higher non-relapse mortality than autologous transplantation. It is usually reserved for patients who fail autologous transplantation or in whom autologous stem cells cannot be collected. It may also have an important role in patients who fail CAR-T therapies. The increasing complexity of care and evolving sequencing of therapies for patients with aggressive B-cell lymphomas only emphasizes the importance of appropriate patient selection and optimal timing of stem cell transplantation.

A plain language summary of the final analysis of the GRIFFIN study of daratumumab plus lenalidomide, bortezomib, and dexamethasone for people with newly diagnosed multiple myeloma.

This summary describes the final analysis of the GRIFFIN study. In this study, participants were newly diagnosed with a type of blood and bone marrow cancer called multiple myeloma, had never received any treatment, and were able to undergo an autologous stem cell transplant. The GRIFFIN study looked at adding the drug daratumumab (D) to a combination of standard treatments called RVd (lenalidomide [R], bortezomib [V], and dexamethasone [d]) during the treatment phases induction and consolidation, followed by daratumumab and lenalidomide (D-R) maintenance. Participants also received an autologous stem cell transplant to further help reduce multiple myeloma. The GRIFFIN study looked at whether D-RVd followed by D-R maintenance was better at killing multiple myeloma cells compared with RVd on its own followed by R maintenance on its own, and if treatments were safe. This summary also describes results from 2 other GRIFFIN publications: one that looked at participants with certain multiple myeloma characteristics or demographic factors that are associated with worse outcomes, and another that looked at how treatments impacted the participants' quality of life. At the time of the final analysis of GRIFFIN, participants who were treated with D-RVd followed by D-R maintenance had very low (undetectable) levels of multiple myeloma cells and multiple myeloma markers (biological signs) and were more likely to be alive without the multiple myeloma getting worse or coming back compared with participants who received standard RVd treatment followed by R maintenance. There was also a pattern of similar benefits achieved by participants who were at risk for worse outcomes. Additionally, participants who received D-RVd treatment followed by D-R maintenance reported less pain, less fatigue (extreme tiredness), and greater improvements in their ability to conduct daily physical activities. While some side effects (unwanted or unexpected effects of treatment) were higher with D-RVd, side effects in both groups were as expected, and adding daratumumab did not reduce a participant's ability to handle treatment. Results of the GRIFFIN study showed that D-RVd treatment followed by D-R maintenance was better at treating multiple myeloma than the standard treatment of RVd followed by R maintenance in adults with a new diagnosis of multiple myeloma who were able to receive an autologous stem cell transplant, with no unexpected side effects of treatment.Clinical Trial Registration: NCT02874742 (GRIFFIN) (ClinicalTrials.gov).

Why are Higher CD34+ Cell Doses Associated with Improved Outcomes among Pediatric Patients Undergoing Autologous Hematopoietic Stem Cell Transplant for Central Nervous System Tumors – But Not for High-Risk Neuroblastoma?

Why are Higher CD34+ Cell Doses Associated with Improved Outcomes among Pediatric Patients Undergoing Autologous Hematopoietic Stem Cell Transplant for Central Nervous System Tumors – But Not for High-Risk Neuroblastoma?

Long-term outcome of autologous haematopoietic stem cell transplantation in patients with systemic sclerosis: a comparison with patients treated with rituximab and with traditional immunosuppressive agents

BackgroundAutologous haematopoietic stem cell transplantation (AHSCT) is more effective than conventional immunosuppressive therapies (CIT) in improving the outcome of patients with rapidly progressive diffuse cutaneous systemic sclerosis (dcSSc). So far, there is still a paucity of data comparing AHSCT with rituximab (RTX). Aim of the study is to retrospectively compare, in patients with dcSSc, the effectiveness of AHSCT with that of RTX and CIT.MethodsThirty-five dcSSc AHSCT-treated patients were compared with 29 and 36 matched cases treated with RTX and CIT, respectively. The patients were followed up for 5 years by assessing selected outcome measures every year. Overall survival, modified Rodnan skin score (mRSS), lung function tests (FVC and DLCO), and the revised EUSTAR Activity Index (REAI) were the outcome measures chosen to evaluate the therapy efficacy.ResultsAHSCT was significantly more effective than RTX and CIT in prolonging survival, inducing a rapid reduction of the mRSS and REAI and maintaining the baseline level of lung function tests for a longer time. RTX therapy was also superior to CIT in reducing REAI, mRSS and in saving lung function.ConclusionAHSCT is more effective than both RTX and CIT in prolonging survival and inducing prolonged remission in patients with rapidly progressive dcSSc.

The evolving role of checkpoint inhibitors in the treatment of Hodgkin lymphoma.

Since their initial approval as single agent therapy for multiply relapsed/refractory Hodgkin lymphoma (HL), the PD-1 inhibitors nivolumab and pembrolizumab have been incorporated into second-line salvage regimens, and they are being investigated in upfront therapy of newly diagnosed patients. As second-line therapy in combination with brentuximab vedotin or multi-agent chemotherapy, nivolumab and pembrolizumab provide high complete remission rates and durable progression-free survival after consolidative autologous stem cell transplant. Incorporation of these agents into frontline chemotherapy regimens is feasible, and early results from a Phase III trial of nivolumab-AVD compare favorably with the existing standard for advanced stage HL, brentuximab vedotin plus AVD. As nivolumab and pembrolizumab move into earlier lines of HL therapy, open research questions include the efficacy of checkpoint inhibitor regimens in patients who relapse after frontline exposure to nivolumab or pembrolizumab; the selection of patients with relapsed HL who can achieve durable remissions without autologous stem cell transplant; and the efficacy of the PD-1 inhibitors in the frontline therapy of patients with early stage Hodgkin lymphoma.

Cellular therapy in older adults with relapsed/refractory diffuse large B-cell lymphoma.

Relapsed/Refractory (R/R) diffuse large B-cell lymphoma (DLBCL) is an aggressive disease with poor prognosis and limited therapeutic options. High-dose chemotherapy with autologous hematopoietic stem cell transplantation (autoHCT) was historically the curative-intent treatment for patients who demonstrated chemosensitivity to salvage therapy. However, a significant portion of patients do not make it autoHCT due to disease progression or overall fitness and eligibility. This is of particular concern in the older adult population. In recent years, significant advances in cellular therapies including chimeric antigen receptor (CAR) T-cells and bispecific antibodies, in addition to improvement in autoHCT tolerability, have allowed for additional treatment options for patients with R/R DLBCL. These novel therapies offer the potential for durable remissions and cure, and should be considered in older patients. We present a review focused on the safety and efficacy of cellular therapies in the older adult population with R/R DLBCL.

A systematic review on the epidemiology and treatment options of multiple Myeloma in Asia

Multiple myeloma (MM) accounts for almost 15 % of all neoplastic malignancies around the globe. This systematic review intends to analyse data on the treatment and management of MM in selected regions in Asia to identify and prioritize areas that need attention. A comprehensive review of original articles, published in English from 2005 to 2022, derived from the PubMed/MEDLINE database was conducted based on the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. There were 98 studies from select regions of Asia (China, India, Taiwan, Hong Kong, and Singapore) on newly diagnosed MM and relapsed/refractory MM. This review evaluated the trends in disease outcomes with the gradual shift in treatment regimens from doublet to triplet. Additionally, this review also explored autologous stem cell transplant outcome and anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T-cell therapy in MM patients. This is the first systematic review attempting to collect data on the utility and comparison of innovative agents and modifications in treatment regimens in the context of the Asian population. This review established that the body of evidence for the management of MM was generally of poor quality and there is a need for more versatile studies in the region. Novel and innovative drug regimens may help in combating the illness but consorted efforts by researchers, industry partners, policymakers, and the government are key factors in the long-term survival of MM patients. In the current systematic review, the authors have tried to give a comprehensive account of the available treatments, trends in MM management and prognosis for MM in Asia.

Maintenance therapy promotes profound organ and haematologic response in light-chain amyloidosis patients not undergoing autologous stem cell transplantation

Background Evidence on maintenance therapy following frontline induction is sparse in primary light-chain amyloidosis (AL), especially for those who do not undergo autologous haematopoietic stem cell transplantation (ASCT). Methods We enrolled primary AL patients who achieved at least haematologic very good partial response (VGPR) by the 4th month at the frontline from December 2008 to June 2023 at Zhongshan Hospital, Fudan University. Those who followed maintenance therapy were identified as the maintenance group (n = 44), whereas those entering the observational phase were classified as the observational group (n = 24). Results After 7.2(interquartile range, 4.7–18.6) months of maintenance therapy, 9(20.5%), 14(31.8%) and 5 (11.4%) patients achieved improvement in haematologic, cardiac and renal response respectively. Five (11.4%) patients had minimal residual disease (MRD) response conversion from positive to negative based on maintenance therapy. In the observation group, none of the patients had haematologic response improvement, with 3(12.5%) and 1(4.2%) patient showing cardiac and renal response improvement during follow-up. Conclusion This study identified the clinical benefits of maintenance therapy in patients with AL who did not undergo ASCT in real-world practice.

A real-world pharmacovigilance study of tafasitamab: data mining of the US food and drug administration adverse event reporting system

ABSTRACT Background Tafasitamab is the first anti-CD19 monoclonal antibody approved for relapsed/refractory diffuse large B-cell lymphoma patients ineligible for autologous stem cell transplantation. The study was designed to evaluate tafasitamab-associated adverse events (AEs) by data mining the United States Food and Drug Administration Adverse Event Reporting System (FAERS). Research design and methods A disproportionality analysis was performed to assess the safety profile of tafasitamab based on the reports from the FAERS database between 2020Q3 and 2023Q3. Proportional reporting ratio (PRR) and empirical Bayesian geometric mean (EBGM) were used to identify the signals of AEs in patients receiving tafasitamab. Results A total of 529 reports with tafasitamab as the primary suspect drug were collected, including 1,262 AEs. Of these, 28 repeated AEs were identified using two algorithms. After excluding events unrelated to drug therapy, the top five repeated AEs by intensity ranking were cytopenia, immunosuppression, neutropenic sepsis, blood lactate dehydrogenase increased, and hematotoxicity. Unexpected significant AEs included polyneuropathy, splenomegaly, hemophagocytic lymphohistiocytosis, hypercalcemia, and ascites. Conclusions This study provides additional evidence for risk identification of tafasitamab in the real world, which could help clinicians and pharmacists increase vigilance and improve the safety of tafasitamab in clinical practice.

Multicenter randomized phase III study of high-dose therapy with autologous stem cell transplantation versus observation for patients with newly diagnosed peripheral T-cell lymphoma who achieved complete metabolic response after induction therapy (JCOG2210, TRANSFER study).

Patients with peripheral T-cell lymphoma demonstrated a poor prognosis after obtaining a complete response with induction treatment compared to those with B-cell lymphoma. Once it relapsed, curative treatment is frequently limited to invasive treatments with significant treatment-related mortality, including allogeneic stem cell transplantation. The limitations of these treatment choices indicate the necessity for developing optimal consolidation therapies to prevent relapse. This multicenter randomized phase III trial aims to confirm the superiority of the high-dose therapy with autologous stem cell transplantation over observation alone in terms of progression-free survival for patients with newly diagnosed peripheral T-cell lymphoma who achieved complete metabolic response after induction therapy. A total of 140 patients from 52 hospitals will be enrolled in Japan over 5.5years. This trial is registered in the Japan Registry of Clinical Trials as jRCTs031240169 (https://jrct.niph.go.jp/latest-detail/jRCTs031240169).

Hematopoietic stem cell transplantation therapy for refractory' Crohn disease: A systematic review and meta-analysis.

Despite the availability of numerous treatments for Crohn disease, there are patients who do not respond to any therapy, thereby diminishing their quality of life. The aim of this review is to analyze the efficacy and safety of autologous hematopoietic stem cell transplantation therapy for refractory Crohn disease. This work is a systematic review with meta-analysis conducted in accordance with the guidelines of the Preferred Reporting Items for Systematic Review and Meta-Analyses. Electronic databases such as PubMed, Scopus, Web of Science, and ClinicalTrials were consulted. The searches were carried out in August 2024. To evaluate the efficacy of autologous hematopoietic stem cell transplantation in inducing remission, the mean and standard deviation of the Crohn's Disease Activity Index pre- and post- treatment were used, and a fixed-effects meta-analysis was conducted. Additionally, to assess the efficacy in perianal fistulas, a random-effects meta-analysis was performed, collecting data on the number of subjects with fistulas at the beginning and end of the intervention. All 95% confidence intervals were calculated, and the I2 statistic was used to assess the heterogeneity of the outcome variables. A total of 609 records were identified from databases, with 12 studies selected for inclusion in the review. Immediate intervention proved effective in inducing a decrease in the Crohn Disease Activity Index compared to late intervention with conventional therapies. Moreover, the meta-analysis demonstrated efficacy for Crohn disease and associated fistulas with a mean decrease in the CDAI of -217.53 ± 14.3. When evaluating the efficacy of the procedure in perianal fistulas, a risk ratio of 0.47 with a 95% CI of [0.26, 0.86] was obtained. However, the procedure showed adverse effects, such as infections, acute renal failure or deaths. Systemic autologous hematopoietic stem cell transplantation has shown efficacy in patients who fail to achieve remission of their Crohn disease with conventional therapies. This procedure has also demonstrated efficacy in treating perianal fistulas. However, it is essential to carefully evaluate de implementation of this procedure due to the associated risks.

Comparative analysis of single versus tandem autologous stem cell transplantation in patients with multiple myeloma in Korea: the KMM2102 study

Tandem autologous stem cell transplantation can improve the prognosis of patients with multiple myeloma. However, the precise role of tandem transplantation remains debatable. We evaluated the clinical benefits of tandem transplantation retrospectively. Of the 655 included patients, 117 underwent tandem transplantation; the remaining were assigned to the control group. After a single transplantation, the tandem group achieved a complete remission (CR) rate of 24.8%, which increased to 46.2% after a second transplantation. The tandem group had a significantly longer median PFS than the control group in patients with International Staging System (ISS) III and high-risk cytogenetics (23.1 vs. 14.7 months, p = 0.007 for ISS III; 21.7 vs. 13.2 months, p = 0.042 for high-risk cytogenetics). The tandem group exhibited significantly superior PFS to the control group (20.3 vs. 12.6 months, p = 0.003) among patients who failed to achieve CR after a single transplantation. Tandem transplantation was associated with significantly improved PFS after adjusting for maintenance therapy in patients with ISS III, those with high-risk cytogenetics, and those who did not achieve CR after a single transplantation. Following propensity score matching, the tandem group exhibited significantly longer PFS than the control group (30.3 vs. 13.5 months, p = 0.028). Tandem transplantation should be considered in high-risk patients.

Clinical analysis of bleeding and thrombotic events in haematological-oncology patients with severe thrombocytopenia and a high risk of thrombosis

Background: Haematological patients with severe thrombocytopenia and high thrombotic risk face challenges related to balancing bleeding and thrombosis risks. This study investigated factors associated with bleeding and thrombosis in high-risk haematological oncology patients with severe thrombocytopenia not receiving anticoagulant therapy and characterized their clinical features when both events occurred. Methods: A total of 446 haematological oncology patients with Caprini scores ≥ 5 were included from July 2022 to June 2023 at Mianyang City Central Hospital. Those not receiving prophylactic anticoagulants due to an admission platelet count < 50 × 109/L were studied. Patients were categorized into bleeding/nonbleeding and thrombotic/nonthrombotic groups on the basis of hospital course. Relevant clinical data were collected, and univariate/multivariate logistic regression was used to analyse the influencing factors. The platelet count at admission was assessed via ROC curves for thrombosis prediction. Results: In the bleeding group, higher proportions of patients with leukaemia, myeloid tumours, lung infections, and a central venous catheter (CVC) with two lumens were observed, along with shorter catheter durations, lower initial and minimum platelet counts during hospitalization, and prolonged plasminogen times (all P < 0.05). The thrombotic group had a greater thrombosis history, initial platelet count, use of two venous catheter lumens, parenteral nutrition, sedation, and autologous haematopoietic stem cell transplantation (Auto-HSCT), with a lower leukaemia proportion (P < 0.05). Logistic regression identified lymphoma type and minimum platelet count as bleeding protective factors and the Charlson Comorbidity Index (CCI) score as an independent risk factor. Thrombosis history, two venous catheter lumens, and sedation were risk factors for thrombosis. The median platelet count was lower at bleeding and thrombosis than at admission (P = 0.007). The platelet count at admission had predictive value for thrombosis, especially severe thrombocytopenia, with an AUC of 0.735 (95% CI 0.613–0.858, P = 0.003) and a cut-off value of 42.5 × 109/L. Conclusions: For haematological neoplasm patients with a high risk of venous thromboembolism (VTE), severe thrombocytopenia and high CCI scores, risk prevention and control of bleeding take precedence over thrombosis prophylaxis. Prophylactic anticoagulation is still recommended for patients with lymphoma assessed at high risk for VTE and with platelet counts of at least 42.5 × 109/L.