Role of extracorporeal photopheresis in the management of children with graft-vs-host disease.

INTRODUCTION: Graft-vs-host disease (GVHD) is a frequent cause of morbidity and mortality in allogeneic stem cell transplants. Extracorporeal photopheresis (ECP) is one of the most accepted second-line treatments, but technical issues of ECP in children might be prohibitive. MATERIALS AND METHODS: Patients under 18 y of age with corticodependant or corticorefractory GVHD receiving ECP at our hospital were included in this retrospective study. ECP was performed with an in-line system (CellExTherakos) in 2013-2014 and with an off-line system (Spectra Optia) from 2015 onwards. Cumulative incidence curves were obtained to compare ECP efficacy among patients grouped by different baseline, apheresis, and disease characteristics. Significant variables on univariate analysis (Gray's test) were pooled into a multivariate analysis (Fine-Gray proportional hazard regression for competing events). RESULTS: A total of 701 ECP sessions were performed on 33 patients between October 2013 and December 2021. In total, 97% of the sessions could be executed. In 8% of the sessions an incident was detected, most of them mild and related to catheter dysfunction. With a median follow-up for alive patients of 33.6 mo (range, 8-95), the composite partial and complete response cumulative incidence was 70% (95% confidence interval, 51%-82%) and the median time to maximal response was 2.8 mo (range, 0.25-9.8). Significantly lower response ratios were found in patients with hepatic, gastrointestinal, acute, or severe GVHD. The only variable that influenced response on multivariate analysis was GVHD severity. DISCUSSION: ECP is feasible, safe, and effective for pediatric patients with corticorefractory or corticodependant GVHD, offering a less toxic and nonimmunosuppressive treatment option.

[Immunotherapy in multiple myeloma]. / Inmunoterapia en el mieloma múltiple.

Patients with multiple myeloma who present with refractory disease or relapse after receiving the main classes of available drugs -immunomodulators, proteasome inhibitors and antibodies against CD38- do not have satisfactory therapeutic alternatives. New treatments based on the redirection of T lymphocytes to act directly against tumor cells, such as bispecific antibodies and T cells with chimeric antigen receptors, are changing this scenario. The published information confirms unprecedented antitumor activity of these agents in patients with refractory myeloma and they will certainly represent the backbone of the treatment of these patients in the immediate future. However, these therapies also present specific characteristics and medium or long-term toxicities that pose new healthcare challenges. In this review, we address the current results and future challenges of the administration of these treatments in patients with relapsed or refractory multiple myeloma.

Single-Unit Transfusion Policy in Autologous Hematopoietic Stem Cell Transplantation: Less is Not Worse.

Single-unit red blood cell (1-RBC) transfusion policy has shown to effectively reduce transfusion burden while maintaining comparable clinical outcomes in hematological patients compared to the classical double-unit policy. However, its effects specifically after autologous stem cell transplantation (ASCT) have not been previously studied. We aimed to evaluate the impact of the 1-RBC policy on transfusion burden in a homogeneous cohort of patients undergoing ASCT. We retrospectively compared the transfusion requirements and the clinical outcomes of 187 patients transplanted from May 2019 to December 2022 under a 1-RBC policy, with a historical cohort of 153 patients transplanted from January 2016 to April 2019 under a double-unit policy. The 1-RBC policy was associated with a 32% reduction in RBC utilization and lower number of RBC transfusions at day 30 after transplantation (median 2 versus 3 units; P < .0001), with an odds ratio of 0.49 in multivariate analysis (P = .03). However, the number of transfusion episodes remained similar (median of 2 in both arms; P = .34). No significant differences in length of stay, hemoglobin levels at discharge or 30-day mortality were observed. In conclusion, transitioning to the 1-RBC represents a straightforward action in current practice that significantly reduces blood transfusions in patients undergoing ASCT, without negatively impacting clinical outcomes.

Extracorporeal Photopheresis in Graft-versus-Host Disease.

Graft-versus-host disease (GVHD) is a major cause of mortality and morbidity following allogeneic hematopoietic stem cell transplantation. Extracorporeal photopheresis (ECP), which exposes mononuclear cells to ultraviolet A irradiation in the presence of a photosensitizing agent, has shown efficacy in the treatment of GVHD. Recent observations in molecular and cell biology have revealed the mechanisms by which ECP can reverse GVHD, including lymphocyte apoptosis, differentiation of dendritic cells from circulating monocytes, and modification of the cytokine profile and T cell subpopulations. Technical innovations have made ECP accessible to a broader range of patients; however, logistical constraints may limit its use. In this review, we scrutinize the development of ECP from its origins to recent insights into the biology underlying ECP efficacy. We also review practical aspects that may complicate successful ECP treatment. Finally, we analyze how these theoretical concepts translate into clinical practice, summarizing the published experiences of leading research groups worldwide.

Real-World Experience in Extracorporeal Photopheresis for Adults with Graft-versus-Host Disease.

Extracorporeal photopheresis (ECP) has shown efficacy in treating graft-versus-host disease (GVHD). We aim to summarize eight years of real-world experience with off-line ECP in our institution, in order to validate this treatment schedule and analyze predictive factors. All consecutive adult patients with steroid-dependent or steroid-refractory GVHD undergoing off-line ECP were included in this single-center retrospective study. ECP was performed with a Spectra Optia device, processing 1 total blood volume, at a twice-weekly frequency for acute GVHD (aGVHD) and once weekly for chronic GVHD (cGVHD), and tapered individually according to clinical response. The cumulative incidence of response, including complete response (CR) and partial response (PR), were compared among patients grouped by different baseline, apheresis, and disease characteristics. Between January 2015 and May 2022, a total of 1382 ECP procedures were proposed for 82 patients. No incidents were reported in 97% of the ECP sessions. GVHD responded in 78% of patients (aGVHD: 57% CR and 4% PR; cGVHD, 39% CR and 48% PR). Overall survival was statistically greater for aGVHD patients who responded to ECP compared to those who did not respond (67.5% versus 26% at 1 year; P = 0.037). Severity was an independent predictor of response in aGVHD, whereas the absence of mouth involvement and lower lymphocyte counts in the apheresis product correlated with a higher response in cGVHD. Our findings support the effectiveness of this treatment schedule for GVHD. Further investigation is required to identify ECP-specific predictive factors, given that findings are not homogeneous across studies.

Cerebral calcium embolism.

A new-onset neurological deficit after calcified aortic valve replacement and an hyperdense image on the computed tomography raised suspicion of an stroke of unusual etiology.

Emicizumab-induced photosensitivity.

Emicizumab constitutes a novel and effective prophylaxis for hemophilia A patients with and without inhibitors. In this case report, we describe an emicizumab-induced photosensitivity that forced permanent sun-exposure suppression. To the best of our knowledge, this side effect had not been communicated until present.