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Prior studies have reported pulmonary function tests (PFT) before and after related and unrelated allogeneic hematopoietic stem cell transplantation (HSCT). However, limited data exist on the evaluation of lung function after haploidentical stem cell transplantation (HAPLO) with post-transplant cyclophosphamide (PTCY). We retrospectively reported the evaluation of PFTs at screening before HAPLO in 80 patients at 100 days and 1 year of follow-up. The proportion of surviving patients with available PFTs at 100 days and 1 year were 86% and 68%, respectively. During the follow-up period, four patients met the criteria for bronchiolitis obliterans syndrome and two for interstitial lung disease. Overall survival was 73% (95% CI 62–82%) at 2 years. We observed a significant reduction in diffusing capacity of the lungs for carbon monoxide (DLCO) corrected for the most recent hemoglobin concentration (DLCOc) at 100 days after HAPLO. However, an overall substantial stable pulmonary function was observed at 1 year. [ABSTRACT FROM AUTHOR]

We assessed the outcomes associated with thiotepa, busulfan and fludarabine (TBF) conditioning regimen in a cohort of 29 consecutive patients allografted for myelofibrosis (MF). The median age was 56 (range 42–70) years. According to the refined Dynamic International Prognostic Scoring System (DIPSS-plus), 15 (52%) patients were classified as high risk. Graft source was peripheral blood stem cells in 27 patients. Donor type was HLA-matched related (n = 5), matched unrelated (n = 16), mismatched unrelated (n = 1), and haploidentical (n = 7). All but 2 patients engrafted. The cumulative incidence (CI) of grade II–IV acute graft-versus-host disease (GVHD) was 21% (95% CI, 10–42) at day 100. The CI of chronic GVHD was 39% (95% CI, 23–65) at 3 years. The median follow-up period was 39 (range 14–60) months. Overall survival was 69% (95% CI, 50–83) at 3 years. No relapse was observed. TBF is a valid conditioning strategy in patients with MF. [ABSTRACT FROM AUTHOR]

Chemotherapy treatment and autologous and allogeneic cell transplantations are often complicated by the onset of metabolic and endocrine disorders. Autoimmune disorders, metabolic diseases, and hormonal dysfunctions are some of the endocrine complications observed during or after treatment with immunotherapy (mostly novel agents) and/or chemotherapy conditioning for transplantation. Although successful treatment of the underlying hematological condition often improves the dysfunction, endocrinopathies can have an impact on prognosis and are associated with poor survival; therefore, it is important to detect and treat them as early as possible. An increased incidence of cardiovascular diseases and metabolic syndrome has been observed after transplantation mostly in longterm survivors. In addition, chemotherapy and radiation along with the prolonged use of corticosteroids can contribute to the onset of thyroid and gonadal dysfunctions. The aim of this article is to describe metabolic dysfunctions occurring in patients who underwent allogeneic cell transplantation. [ABSTRACT FROM AUTHOR]

Summary: Allogeneic haematopoietic stem cell transplantation is still the only available curative option for Familial Haemophagocytic Lymphohistiocytosis (FHLH). Most studies report outcomes after bone marrow or peripheral blood stem cell transplantation. We analysed the outcomes of 118 children with FHLH undergoing single‐unit umbilical cord blood transplantation performed from 1996 to 2014. Myeloablative conditioning regimen was given to 90% of the patients, and was mostly busulfan‐based (n = 81, 76%), including anti‐thymocyte globulin or alemtuzumab (n = 102, 86%). The cumulative incidence of Day 60 neutrophil engraftment was 85%; and that of non‐relapse mortality and acute graft‐versus‐host disease (GvHD) was 21% and 33% at 100 days, respectively. The 6‐year cumulative incidence of chronic GvHD was 17% and the 6‐year probability of overall survival was 55%. In multivariate analysis, children receiving a graft with a total nucleated cell dose greater than 9·9 × 107/kg had a better overall survival (hazard ratio [HR]: 0·49, 95% CI: 0·27–0·88, P = 0·02). Degree of human leucocyte antigen (HLA) matching was associated with improved disease‐free survival (5/6 vs. 6/6 HR: 2·11, 95% confidence interval [CI]: 1·01–4·4, P = 0·05 and ≤4/6 vs. 6/6, HR: 2·82, CI: 1·27–6·23, P = 0·01). Umbilical cord blood transplantation with a high cell dose and good HLA match is a suitable alternative option to haematopoietic stem cell transplantation in children with FHLH who lack a HLA‐matched donor. [ABSTRACT FROM AUTHOR]

Objective Methods Results Conclusions This retrospective single center study aims to describe changes in the severity and organ‐specific distribution of GvHD, by comparing the outcomes of 3 distinct GvHD prophylaxis approaches.Between January 2012 and June 2022, 226 patients underwent allogeneic hematopoietic stem cell transplantation from HLA‐matched or 1‐allele mismatched related or unrelated donors. Fifty‐eight (26%) received prophylaxis with calcineurin inhibitor in combination with mycophenolate mofetil or a short course of methotrexate (Cohort‐1), 87 (38%) tacrolimus plus sirolimus (Cohort‐2), and 81 (36%) post‐transplant cyclophosphamide (PTCy) plus tacrolimus (Cohort‐3).The incidence of grade II‐IV aGvHD was 69% vs. 41.4% vs. 27.2%; p < .01. The most significant reduction with PTCy was observed in both stage 3–4 skin and lower gastrointestinal (GI) involvement (p < .01). The incidence of moderate‐to‐severe cGvHD at 12 months was 34.5% vs. 34.5% vs. 6.2%; p < .01. Moderate‐to‐severe skin and GI cGvHD was less common after PTCy (p < .01). The 1‐year GvHD‐free/relapse‐free survival was higher with PTCy (p < .01).Our study indicates that PTCy‐based GvHD prophylaxis reduces the frequency and severity of both acute and chronic GvHD, with a notable decrease in severe GI and cutaneous manifestations. The higher GRFS may result in lower GvHD‐related mortality, leading to an improved quality of life among survivors. [ABSTRACT FROM AUTHOR]

Background: Patients with hematological malignancies receiving hematopoietic cell transplantation (HCT) or chimeric antigen receptor (CAR) T-cell therapy are at risk of developing serious clinical complications after discharge., Objective: The aim of the TEL-HEMATO study was to improve our telehealth platform for the follow-up of patients undergoing HCT or CAR T-cell therapy during the first 3 months after discharge with the addition of wearable devices., Methods: Eleven patients who received autologous (n=2) or allogeneic (n=5) HCT or CAR T-cell therapy (n=4) for hematological malignancies were screened from November 2022 to July 2023. Two patients discontinued the study after enrollment. The telehealth platform consisted of the daily collection of vital signs, physical symptoms, and quality of life assessment up to 3 months after hospital discharge. Each patient received a clinically validated smartwatch (ScanWatch) and a digital thermometer, and a dedicated smartphone app was used to collect these data. Daily revision of the data was performed through a web-based platform by a hematologist or a nurse specialized in HCT and CAR T-cell therapy., Results: Vital signs measured through ScanWatch were successfully collected with medium/high adherence: heart rate was recorded in 8/9 (89%) patients, oxygen saturation and daily steps were recorded in 9/9 (100%) patients, and sleeping hours were recorded in 7/9 (78%) patients. However, temperature recorded manually by the patients was associated with lower compliance, which was recorded in 5/9 (55%) patients. Overall, 5/9 (55%) patients reported clinical symptoms in the app. Quality of life assessment was completed by 8/9 (89%) patients at study enrollment, which decreased to 3/9 (33%) at the end of the third month. Usability was considered acceptable through ratings provided on the System Usability Scale. However, technological issues were reported by the patients., Conclusions: While the addition of wearable devices to a telehealth clinical platform could have potentially synergic benefits for HCT and CAR T-cell therapy patient monitoring, noncomplete automation of the platform and the absence of a dedicated telemedicine team still represent major limitations to be overcome. This is especially true in our real-life setting where the target population generally comprises patients of older age with a low digital education level., (©Lidia Hurtado, Melinda Gonzalez Concepcion, Aida Flix-Valle, Marina Ruiz-Romeo, Sonia Gonzalez-Rodriguez, Marta Peña, Annalisa Paviglianiti, Maria Angeles Pera Jambrina, Anna Sureda, Cristian Ochoa-Arnedo, Alberto Mussetti. Originally published in JMIR Formative Research (https://formative.jmir.org), 04.06.2024.)

Background: Human metapneumovirus (hMPV) epidemiology, clinical characteristics and risk factors for poor outcome after allogeneic stem cell transplantation (allo-HCT) remain a poorly investigated area., Methods: This retrospective multicenter cohort study examined the epidemiology, clinical characteristics, and risk factors for poor outcomes associated with human metapneumovirus (hMPV) infections in recipients of allo-HCT., Results: We included 428 allo-HCT recipients who developed 438 hMPV infection episodes between January 2012 and January 2019. Most recipients were adults (93%). hMPV infections were diagnosed at a median of 373 days after allo-HCT. The infections were categorized as upper respiratory tract disease (URTD) or lower respiratory tract disease (LRTD), with 60% and 40% of cases, respectively. Patients with hMPV LRTD experienced the infection earlier in the transplant course and had higher rates of lymphopenia, neutropenia, corticosteroid use, and ribavirin therapy. Multivariate analysis identified lymphopenia and corticosteroid use (>30 mg/d) as independent risk factors for LRTD occurrence. The overall mortality at day 30 after hMPV detection was 2% for URTD, 12% for possible LRTD, and 21% for proven LRTD. Lymphopenia was the only independent risk factor associated with day 30 mortality in LRTD cases., Conclusions: These findings highlight the significance of lymphopenia and corticosteroid use in the development and severity of hMPV infections after allo-HCT, with lymphopenia being a predictor of higher mortality in LRTD cases., Competing Interests: Potential conflicts of interest. P. B. declares research grants from Neovii, Riemser, and Medac (to institution); advisory board membership for Novartis, Cellgene, Amgen, Medac, and Servier (personal and to institution); speakers bureau for Miltenyi, Jazz, Riemser, Novartis, and Amgen (to institution); and patent und royalties from Medac. All other authors report no potential conflicts. All authors have submitted the ICMJE Form for Disclosure of Potential Conflicts of Interest. Conflicts that the editors consider relevant to the content of the manuscript have been disclosed., (© The Author(s) 2023. Published by Oxford University Press on behalf of Infectious Diseases Society of America. All rights reserved. For permissions, please e-mail: journals.permissions@oup.com.)

Multiple myeloma (MM) accounts for about 1.8% of all cancers and slightly over 17% of hematologic malignancies. Despite improvements in outcomes in recent years, currently, there is still no cure for this disease. Although allogeneic stem cell transplantation (Allo-SCT) is a potentially curative treatment, given the armamentarium of highly effective therapeutic options and a pipeline of novel agents, many opinion leaders sustain that there is no longer a role for this approach. Of note, several studies that did not include novel agents reported long-term molecular remissions, and possibly a cure, in a subset of patients after reduced-intensity conditioning/nonmyeloablative (RIC/NM) Allo-SCT. However, there are no current data supporting upfront Allo-SCT. Prospective trials combining the so-called "graft-versus-myeloma" effect and new drugs are an unmet medical need in high-risk patients. Early relapse after first-line treatment, which identifies patients with poor prognosis independently of other prognostic factors, could become a clinical indication.

Although allogeneic stem cell transplantation is not a standard therapy for multiple myeloma, some patients can benefit from this intense therapy. There are few reports on outcomes after umbilical cord blood transplantation in multiple myeloma, and investigation of this procedure is warranted. We retrospectively analyzed 95 patients, 85 with multiple myeloma and 10 with plasma cell leukemia, receiving single or double umbilical cord blood transplantation from 2001 to 2013. Median follow up was 41 months. The majority of patients received a reduced intensity conditioning. The cumulative incidence of neutrophil engraftment was 97%±3% at 60 days, and that of 100-day acute graft-versus-host disease grade II-IV was 41%±5%. Chronic graft-versus-host disease at two years was 22%±4%. Relapse and non-relapse mortality was 47%±5% and 29%±5% at three years, respectively. Three-year progression-free survival and overall survival were 24%±5% and 40%±5%, respectively. Anti-thymocyte globulin was associated with decreased incidence of acute graft-versus-host disease, higher non-relapse mortality, decreased overall and progression-free survival. Patients with high cytogenetic risk had higher relapse, and worse overall and progression-free survival. In conclusion, umbilical cord blood transplantation is feasible for multiple myeloma patients., (Copyright© Ferrata Storti Foundation.)