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Introduction: New diagnostic methods and antifungal strategies may improve prognosis of mucormycosis. We describe the diagnostic value of metagenomic next⁃generation sequencing (mNGS) and identify the prognostic factors of mucormycosis. Methods: We conducted a retrospective study of hematologic patients suffered from mucormycosis and treated with monotherapy [amphotericin B (AmB) or posaconazole] or combination therapy (AmB and posaconazole). The primary outcome was 84-day all-cause mortality after diagnosis. Results: Ninety-five patients were included, with “proven” (n = 27), “probable” (n = 16) mucormycosis confirmed by traditional diagnostic methods, and “possible” (n = 52) mucormycosis with positive mNGS results. The mortality rate at 84 days was 44.2%. Possible + mNGS patients and probable patients had similar diagnosis processes, overall survival rates (44.2% vs 50.0%, p = 0.685) and overall response rates to effective drugs (44.0% vs 37.5%, p = 0.647). Furthermore, the median diagnostic time was shorter in possible + mNGS patients than proven and probable patients (14 vs 26 days, p < 0.001). Combination therapy was associated with better survival compared to monotherapy at six weeks after treatment (78.8% vs 53.1%, p = 0.0075). Multivariate analysis showed that combination therapy was the protective factor (HR = 0.338, 95% CI: 0.162–0.703, p = 0.004), though diabetes (HR = 3.864, 95% CI: 1.897–7.874, p < 0.001) and hypoxemia (HR = 3.536, 95% CI: 1.874–6.673, p < 0.001) were risk factors for mortality. Conclusions: Mucormycosis is a life-threatening infection. Early management of diabetes and hypoxemia may improve the prognosis. Exploring effective diagnostic and treatment methods is important, and combination antifungal therapy seems to hold potential benefits. [ABSTRACT FROM AUTHOR]

Introduction: Scarce real-life data exists for COVID-19 management in hematologic disease (HD) patients in the Omicron era. Purpose: To assess the current clinical management and outcome of SARS-CoV-2 infection diagnosed, identify the risk factors for severe outcomes according to the HD characteristics and cell therapy procedures in a real-world setting. Methods: A retrospective observational registry led by the Spanish Transplant Group (GETH-TC) with 692 consecutive patients with HD from December 2021 to May 2023 was analyzed. Results: Nearly one-third of patients (31%) remained untreated and presented low COVID-19-related mortality (0.9%). Nirmatrelvir/ritonavir was used mainly in mild COVID-19 cases in the outpatient setting (32%) with a low mortality (1%), while treatment with remdesivir was preferentially administered in moderate-tosevere SARS-CoV-2 infection cases during hospitalization (35%) with a mortality rate of 8.6%. The hospital admission rate was 23%, while 18% developed pneumonia. COVID-19-related mortality in admitted patients was 14%. Older age, autologous hematopoietic stem cell transplantation (SCT), chimeric antigen receptor T-cell therapy, corticosteroids and incomplete vaccination were factors independently associated with COVID-19 severity and significantly related with higher rates of hospital admission and pneumonia. Incomplete vaccination status, treatment with prior anti-CD20 monoclonal antibodies, and comorbid cardiomyopathy were identified as independent risk factors for COVID-19 mortality. Conclusions: The results support that, albeit to a lower extent, COVID-19 in the Omicron era remains a significant problem in HD patients. Complete vaccination (3 doses) should be prioritized in these immunocompromised patients. The identified risk factors may help to improve COVID-19 management to decrease the rate of severe disease, ICU admissions and mortality. [ABSTRACT FROM AUTHOR]

Immunocompromised patients with hematologic diseases may experience life-threatening infections with rather uncommon manifestations. Laryngitis has been described as a potential infection in such vulnerable patients and may result in major complications, ranging from impending airway obstruction to total laryngeal necrosis. Immediate laryngoscopy is of paramount importance, as it provides quantification of laryngeal edema and evidence of necrosis. Documentation of the causative pathogen is usually feasible through tissue culture. In the literature, 14 cases of necrotizing laryngitis have already been published. Here, we present the case of a 38-year-old male with a recent diagnosis of multiple myeloma, who received the first cycle of therapy a few days before admission. The patient presented with neutropenic fever, diarrhea, and multiple organ dysfunction. His course was complicated with hemophagocytic lymphohistiocytosis and stridor. A diagnosis of necrotizing laryngitis attributed to Acinetobacter baumannii invasion of the larynx was established. This manuscript highlights that the management of patients with hematologic disease and necrotizing laryngitis should be coordinated in highly specialized centers and clinicians should have a high level of clinical suspicion and act promptly. [ABSTRACT FROM AUTHOR]

Jianzhong Hu,1 Martin Chandler,1 Christopher Matthew Manuel,1 Jorge Caicedo,2 Michael Denne,2 Bruce Ewenstein,2 Ali G Mokdad,2 Shan Xing,3 Michael Recht1,4,5 1American Thrombosis and Hemostasis Network, Rochester, NY, USA; 2Rare Diseases and Hematology, Takeda Pharmaceuticals, U.S.A., Inc, Lexington, MA, USA; 3US Value and Evidence Generation, Takeda Pharmaceuticals, U.S.A., Inc, Lexington, MA, USA; 4Yale Center for Bleeding and Clotting Disorders, Yale University School of Medicine, New Haven, CT, USA; 5National Bleeding Disorders Foundation, New York, NY, USACorrespondence: Michael Recht, National Bleeding Disorders Foundation, 7 Penn Plaza, New York, NY, 10001, USA, Tel +1 800-360-2846, Email mrecht@hemophilia.orgIntroduction: Intracranial hemorrhage (ICH), a serious complication in persons with hemophilia A (PWHA), causes high rates of mortality and morbidity. Identified ICH risk factors from patient data spanning 1998– 2008 require reassessment in light of changes in the current treatment landscape.Aim and methods: PWHA identified in the ATHNdataset were evaluated retrospectively to assess incidence of ICH and determine the association between ICH risk and key characteristics using time-to-event analyses (Cox proportional-hazards models, survival curves, and sensitivity analyses).Results: Over a median follow-up time of 10.7 patient-years, 135 of 7837 PWHA over 2 years of age in the ATHNdataset (1.7%) experienced an ICH. Stratification by prophylaxis status and inhibitor status resulted in an incidence rate (IR) ratio (IRR) (IR+/IR−) of 0.63 (95% confidence interval [CI], 0.43– 0.94; P=0.020) and 1.76 (95% CI, 0.97– 3.20; P=0.059), respectively. Characteristics associated with greater risk of developing ICH include being aged 2– 12 years; being covered by Medicaid; having had HIV, hepatitis C, or hypertension; and never having received factor VIII or prophylactic treatment. In multivariable analysis with interaction, the estimated hazard ratio for PWHA never receiving prophylaxis was 7.67 (95% CI, 2.24– 26.30), which shrunk to 2.03 (95% CI, 1.30– 9.12) in bootstrapping analysis and 3.09 in the highest-penalty ridge-regression analysis but was still significant. Inhibitor status was found not to be statistically associated with ICH in all analyses.Conclusion: These results align with previous studies demonstrating that prophylaxis confers a protective effect against ICH. Previously, inhibitor positivity had been shown to increase risk for ICH; however, this study did not corroborate those findings.Keywords: bleeding, factor VIII, health insurance, hematologic disease, prophylaxis, risk factors

Objectives: Whether pre-exposure prophylaxis (PrEP) with tixagevimab/cilgavimab 150 mg/150 mg (T/C) in individuals with hematologic disease (HD) may lead to a reduced risk of SARS-CoV-2 breakthrough infection (BTI)/hospitalization, or death in the Omicron era remains to be established. Methods: An observational study included participants with HD who received PrEP. BTIs were defined as SARS-CoV-2 positivity by reverse transcription-polymerase chain reaction. The incidence of BTIs (95% CI) and of BTIs/hospitalization/death was calculated using the Kaplan-Meier method and as the number of BTIs per 100 person-years of follow-up according to the circulating variant of concern (VoC). A Poisson regression model was used to evaluate the association between the rate of incidence and circulating VoCs after controlling for demographics and clinical factors. Results: We included 550 HD patients: 71% initiated T/C PrEP when BA.5 was the most prevalent, followed by XBB/EG, BA.2, and BA.1 (19%, 7%, and 3%, respectively). Overall, the 1-year incidence estimate of BTIs/hospitalization/death was 24% (18.7-29.4%). A greater risk of incident infections was observed when BA.5 and XBB/EG sub-lineages circulated (aRR 5.05 [2.17, 11.77]; P < .001 and 3.82 [1.50, 9.7]; P = 0.005, compared to BA.1, respectively). Conclusions: The 1-year incidence of SARS-CoV-2 BTIs/hospitalization/death was 24% which is in line with what was observed in other similar studies. The risk appeared to be higher when more recent Omicron sub-lineages were circulating suggesting a reduction of in vitro neutralization.

IntroductionScarce real-life data exists for COVID-19 management in hematologic disease (HD) patients in the Omicron era.PurposeTo assess the current clinical management and outcome of SARS-CoV-2 infection diagnosed, identify the risk factors for severe outcomes according to the HD characteristics and cell therapy procedures in a real-world setting.MethodsA retrospective observational registry led by the Spanish Transplant Group (GETH-TC) with 692 consecutive patients with HD from December 2021 to May 2023 was analyzed.ResultsNearly one-third of patients (31%) remained untreated and presented low COVID-19-related mortality (0.9%). Nirmatrelvir/ritonavir was used mainly in mild COVID-19 cases in the outpatient setting (32%) with a low mortality (1%), while treatment with remdesivir was preferentially administered in moderate-to-severe SARS-CoV-2 infection cases during hospitalization (35%) with a mortality rate of 8.6%. The hospital admission rate was 23%, while 18% developed pneumonia. COVID-19-related mortality in admitted patients was 14%. Older age, autologous hematopoietic stem cell transplantation (SCT), chimeric antigen receptor T-cell therapy, corticosteroids and incomplete vaccination were factors independently associated with COVID-19 severity and significantly related with higher rates of hospital admission and pneumonia. Incomplete vaccination status, treatment with prior anti-CD20 monoclonal antibodies, and comorbid cardiomyopathy were identified as independent risk factors for COVID-19 mortality.ConclusionsThe results support that, albeit to a lower extent, COVID-19 in the Omicron era remains a significant problem in HD patients. Complete vaccination (3 doses) should be prioritized in these immunocompromised patients. The identified risk factors may help to improve COVID-19 management to decrease the rate of severe disease, ICU admissions and mortality.

Immunocompromised patients with hematologic diseases may experience life-threatening infections with rather uncommon manifestations. Laryngitis has been described as a potential infection in such vulnerable patients and may result in major complications, ranging from impending airway obstruction to total laryngeal necrosis. Immediate laryngoscopy is of paramount importance, as it provides quantification of laryngeal edema and evidence of necrosis. Documentation of the causative pathogen is usually feasible through tissue culture. In the literature, 14 cases of necrotizing laryngitis have already been published. Here, we present the case of a 38-year-old male with a recent diagnosis of multiple myeloma, who received the first cycle of therapy a few days before admission. The patient presented with neutropenic fever, diarrhea, and multiple organ dysfunction. His course was complicated with hemophagocytic lymphohistiocytosis and stridor. A diagnosis of necrotizing laryngitis attributed to Acinetobacter baumannii invasion of the larynx was established. This manuscript highlights that the management of patients with hematologic disease and necrotizing laryngitis should be coordinated in highly specialized centers and clinicians should have a high level of clinical suspicion and act promptly.

Genotype-phenotype association studies often combine phenotype data from multiple studies to increase statistical power. Harmonization of the data usually requires substantial effort due to heterogeneity in phenotype definitions, study design, data collection procedures, and data-set organization. Here we describe a centralized system for phenotype harmonization that includes input from phenotype domain and study experts, quality control, documentation, reproducible results, and data-sharing mechanisms. This system was developed for the National Heart, Lung, and Blood Institute's Trans-Omics for Precision Medicine (TOPMed) program, which is generating genomic and other -omics data for more than 80 studies with extensive phenotype data. To date, 63 phenotypes have been harmonized across thousands of participants (recruited in 1948-2012) from up to 17 studies per phenotype. Here we discuss challenges in this undertaking and how they were addressed. The harmonized phenotype data and associated documentation have been submitted to National Institutes of Health data repositories for controlled access by the scientific community. We also provide materials to facilitate future harmonization efforts by the community, which include 1) the software code used to generate the 63 harmonized phenotypes, enabling others to reproduce, modify, or extend these harmonizations to additional studies, and 2) the results of labeling thousands of phenotype variables with controlled vocabulary terms.

Rosai Dorfman disease is generally defined as a massive bilateral painless cervical lymphadenopathy accompanied with both fever and leukocytosis with neutrophilia. Additionally, it may possibly be associated with polyclonal hypergammaglobulinemia, reversal of CD4/CD8 ratio, the elevated erythrocyte sedimentation rate (ESR), microcytic anemia, and thrombocytosis. Rosai-Dorfman disease is known as a benign self-limiting disease, so no treatment is required in many cases, although it causes death in some cases by involving vital organs like kidney. The treatment is required when there is a life-threatening situation such as airway obstruction or involvement of vital organs such as kidney, liver, and lower respiratory tract. The required treatment choices include steroid therapy, chemotherapy, radiotherapy, and surgery. Surgical treatment is performed for bulk removal to resolve the obstruction caused by the mass as well as taking biopsy for the definite histopathologic diagnosis of disease. A 26-year-old man was referred to oral and maxillofacial surgery (OMFS) clinic of Taleghani hospital with chief complaints of pain and swelling of left submandibular space. According to the patient himself, the swelling had been started three months earlier. After rejecting dental source of the lesion, we decided to remove the mass by excisional biopsy concerning the patient’s discomfort. Histopathology report verified Rosai Dorfman disease as definite diagnosis of the mass.

Simple Summary: Appendicitis is a common condition necessitating emergency surgery. Individuals with hematologic disorders often have abnormalities in blood counts, which can act as risk factors for surgical complications. This study explores surgical risks in appendicitis patients with hematologic disorders, using a retrospective analysis from January 2000 to June 2021. Among the 131 identified patients, 89 were studied; 75 underwent surgery, while 14 received non-surgical treatments. No significant relationship was found between clinical characteristics, hematologic disease risk, severity of appendicitis, and surgical complications. Improved preoperative absolute neutrophil count and platelet counts were higher than those of initial findings in patients without surgical complications. Lower preoperative absolute neutrophil counts and platelet counts were correlated with extended hospital stays. The findings suggest that for hematologic patients with appendicitis, meticulous preoperative laboratory evaluations followed by minimally invasive appendectomy could be a safer approach without increasing the risk of severe complications compared to non-surgical management. Appendicitis is a prevalent surgical emergency. Although appendectomy has traditionally been the go-to treatment, recent studies suggest antibiotics can be equally effective for uncomplicated cases. However, evidence is scant regarding patients with hematologic disorders. This study delves into the surgical risks tied to appendicitis in patients with underlying hematologic conditions. A retrospective analysis was carried out on patients diagnosed with appendicitis and hematologic disorders from January 2000 to June 2021. Patients were pinpointed using ICD-10 diagnostic codes, and surgical procedures were identified based on the hospital's surgical fee codes. Hematologic conditions were sorted into risk levels, and patient treatments were scrutinized. Among the 131 initially identified patients, 89 were included in the study. Out of these, 75 underwent surgical procedures, while 14 received non-surgical treatments. The surgical group displayed better preoperative laboratory outcomes. Clinical characteristics, hematologic disease risk, and severity of appendicitis appeared not to be related to surgical complications. Patients without surgical complications showed improvement in preoperative absolute neutrophil count (ANC) and platelet counts. Lower preoperative ANCs and platelet counts were associated with extended hospital stays. For patients with hematologic disorders diagnosed with appendicitis, thorough preoperative laboratory evaluations followed by minimally invasive appendectomy appear to be a safe route without heightening the risk of severe complications compared to non-surgical management. [ABSTRACT FROM AUTHOR]

Thalassemia is an inherited hematologic disease in which the body makes an abnormal form of hemoglobin. Patients are diagnosed with thalassemia major due to severe anemia and clinical symptoms. Thalassemia major is a common disease in the Turkish Republic of Northern Cyprus (TRNC) and other Mediterranean countries. Thalassemia major is a chronic and severe health problem. Thalassemia major damages organs due to intense iron accumulation and deep anemia. It negatively affects the quality of life and shortens life. Also, due to chronic disease, patients experience psychological and social problems. In this review, we present thalassemia major in the TRNC. The rate of patients has decreased with prevention programs and no sick baby has been born after 2001 in the TRNC. However, existing thalassemia patients have many physical and psychosocial problems and the treatment processes the patients are challenging. Since TRNC is an island that receives immigration, it has a sociological texture that includes different cultures, which can increase the dimensions of this problem. Therefore, this review focuses on the thalassemia process and the psychosocial problems caused by the disease in the TRNC.

Abstract Objectives The Antibiotic Stewardship Team of Meander Medical Centre (Meander MC) instigated a revaluation of its treatment protocol for hematologic patients admitted with febrile neutropenia. The current hospital protocol advises administering meropenem for 72 hours, followed by antibiotic therapy guided by microbiological cultures. In order to responsibly adjust the current empiric regimen, this study aimed to determine the frequency of bacteria resistant to alternative antibiotics, namely ceftazidime and piperacillin/tazobactam, in both surveillance and diagnostic cultures. Methods This retrospective, observational, single-centre study included adult patients with a hematologic malignancy and febrile neutropenia admitted between October 2018 and June 2021. Collected metadata included patient characteristics, surveillance and diagnostic culture results, and antibiotic use. Results A total of 100 patients were included. One or more bacteria resistant to ceftazidime or piperacillin/tazobactam were identified in blood and urine cultures in seven (7%) and one (1%) patients respectively. Conclusions Our results support the safe reduction of the use of meropenem by changing the empiric treatment protocol for patients with hematologic malignancy and febrile neutropenia. As this study showed a lower resistance frequency to piperacillin/tazobactam than to ceftazidime, this antibiotic is the recommended alternative.

Rosai Dorfman disease is generally defined as a massive bilateral painless cervical lymphadenopathy accompanied with both fever and leukocytosis with neutrophilia. Additionally, it may possibly be associated with polyclonal hypergammaglobulinemia, reversal of CD4/CD8 ratio, the elevated erythrocyte sedimentation rate (ESR), microcytic anemia, and thrombocytosis. Rosai-Dorfman disease is known as a benign self-limiting disease, so no treatment is required in many cases, although it causes death in some cases by involving vital organs like kidney. The treatment is required when there is a life-threatening situation such as airway obstruction or involvement of vital organs such as kidney, liver, and lower respiratory tract. The required treatment choices include steroid therapy, chemotherapy, radiotherapy, and surgery. Surgical treatment is performed for bulk removal to resolve the obstruction caused by the mass as well as taking biopsy for the definite histopathologic diagnosis of disease. A 26-year-old man was referred to oral and maxillofacial surgery (OMFS) clinic of Taleghani hospital with chief complaints of pain and swelling of left submandibular space. According to the patient himself, the swelling had been started three months earlier. After rejecting dental source of the lesion, we decided to remove the mass by excisional biopsy concerning the patient’s discomfort. Histopathology report verified Rosai Dorfman disease as definite diagnosis of the mass. [ABSTRACT FROM AUTHOR]

Worachaya Pengthina,1 Pirun Saelue2 1Pharmaceutical Care Training Center, Department of Pharmaceutical Care, Faculty of Pharmacy, Chiang Mai University, Chiang Mai, Thailand; 2Clinical Hematology Unit, Division of Internal Medicine, Faculty of Medicine, Prince of Songkla University, Hat Yai, Songkhla, ThailandCorrespondence: Pirun Saelue, Clinical Hematology Unit, Division of Internal Medicine, Faculty of Medicine, Prince of Songkla University, Hat Yai, Songkhla, 90110, Thailand, Tel +66 7445 1481 ; +66 81543 3031, Fax +66 7428 1457, Email pirun2118@hotmail.comBackground: Bone marrow transplantation, antithymocyte globulin/cyclosporine and eltrombopag are recommended as first-line therapy of severe aplastic anemia (SAA). However, androgens could be considered as front-line treatment among any patients ineligible for better methods although unsatisfactory efficacy is presented.Objective: This retrospective study aimed to evaluate response and survival rate of practical-based treatment with oxymetholone.Patients and Methods: This constituted an analysis of patients receiving a diagnosis of acquired aplastic anemia (AA) at the age of 15 or over and receiving oxymetholone between January 2004 and December 2018. Propensity Score Analysis (PSA) 1:1 matching was performed, according to sex, age and interval from first symptom to treatment. The primary outcome was one-year overall response (OR).Results: Seventy-four patients were successfully matched by PSA. The 1-year OR of oxymetholone in the nonsevere AA (nSAA) and SAA/very severe AA (vSAA) groups was 54.1 and 13.5%, respectively (P < 0.001). With median follow-up 2.7 years, the overall survival was 59.5% in nSAA and 37.8% in SAA/vSAA (P = 0.051). Median survival in nSAA and SAA/vSAA were 7.0 years and 1.8 years, respectively (P = 0.045). However, the responders of SAA/vSAA had longer survival than nonresponders of the nSAA group.Conclusion: These results revealed longer survival among the responders of patients with AA, even in the SAA/vSAA group. However, close monitoring of therapeutic responses is still performed. Switching therapy is necessary when remission is undetected after 6 months of oxymetholone treatment.Keywords: androgen, hematologic disease, real world practice, survival

(1) Background: We compared the diagnostic and prognostic performance of serum amyloid A (SAA), procalcitonin (PCT), delta neutrophil index (DNI), and C-reactive protein (CRP) in patients with hematologic diseases; (2) Methods: We retrospectively collected the remaining serum samples from patients with hematologic diseases, analyzed their clinical data, and measured the levels of PCT, DNI, CRP, and SAA. Performances for infection diagnosis were evaluated using a receiver operating characteristic curve analysis, and 90-day mortality was analyzed using Kaplan–Meier estimation; (3) Results: The levels of all markers were significantly higher in the infected group (N = 27) than those in the uninfected group (N = 100) (p < 0.0001 for all markers). The areas under the curve for diagnosing infection for PCT, DNI, CRP, and SAA were 0.770, 0.817, 0.870, and 0.904, respectively. Increased PCT levels were associated with higher mortality (p = 0.0250); this association was not observed with other examined markers; (4) Conclusions: CRP and SAA exhibited greater discriminative power for infection than PCT. However, only PCT levels were positively associated with 90-day mortality. Herein, we evaluated the diagnostic performance of the four markers. Additional studies are needed to confirm the findings of the present study and validate the potential of these markers in clinical practice. [ABSTRACT FROM AUTHOR]

Background: The selective leukoreduction protocol (SLP) is limited in that patients who require it can be overlooked. We estimated SLP compliance (SLPC) using the Observational Medical Outcomes Partnership common data model (CDM). Methods: Patients were classified into eight groups: pre- and post-hematology disease (A and B), pre- and post-solid organ transplantation (C and D), solid cancer (E), immunodeficiency (F), anticancer therapy (G), and cardiovascular surgery (H). We examined the red blood cell (RBC) transfusion history from three hospital datasets comprising approximately three million patients over 20 years using CDM-based analysis. SLPC was calculated as the percentage of patients who received only leukoreduced RBCs in total patients transfused RBCs. Results: In total, 166,641 patients from three hospitals were enrolled in this study. From 2001 to 2021, SLPC in all groups, except H, tended to increase, although there were differences among the hospitals. Based on the most recent values (2017-2021), the SLPC in groups A, B, D, and G was maintained at =75% until 1,095 days before or after diagnosis or treatment. Groups E, F, and H had < 50% SLPC one day after diagnosis and treatment. Conclusions: CDM analysis supports the review of large datasets for SLPC evaluation. Although SLPC tended to improve in most patient groups, additional education and monitoring are needed for groups that continue to show low SLPC. [ABSTRACT FROM AUTHOR]

The COVID-19 pandemic has already reaped thousands of lives, although many scientific studies already showed the possibility of this scenario. Currently, further attention is provided to patients depicting comorbidities such as respiratory or immunocompromised diseases, hypertension, and diabetes, as these individuals show a worse prognosis. Cell therapies using stem cells and/or defense cells, combined or not with traditional treatment, could be an outstanding strategy for COVID-19 management since these treatments can act by modulating the immune system, reducing proliferation, and favoring the complete elimination of the virus. In this review, we highlight the main molecular characteristics of this novel coronavirus, as well as the main pathognomonic signs of COVID-19. Furthermore, possible cell therapies are pointed out to show alternative treatments against COVID-19 and its sequels.

• The 1-year incidence estimate of breakthrough infections (BTIs)/hospitalization/death was 24%. • A greater risk of incident BTIs was observed with BA.5 and XBB/EG. • The search for a new prophylaxis is urgently needed. Whether pre-exposure prophylaxis (PrEP) with tixagevimab/cilgavimab 150 mg/150 mg (T/C) in individuals with hematologic disease (HD) may lead to a reduced risk of SARS-CoV-2 breakthrough infection (BTI)/hospitalization, or death in the Omicron era remains to be established. An observational study included participants with HD who received PrEP. BTIs were defined as SARS-CoV-2 positivity by reverse transcription-polymerase chain reaction. The incidence of BTIs (95% CI) and of BTIs/hospitalization/death was calculated using the Kaplan-Meier method and as the number of BTIs per 100 person-years of follow-up according to the circulating variant of concern (VoC). A Poisson regression model was used to evaluate the association between the rate of incidence and circulating VoCs after controlling for demographics and clinical factors. We included 550 HD patients: 71% initiated T/C PrEP when BA.5 was the most prevalent, followed by XBB/EG, BA.2, and BA.1 (19%, 7%, and 3%, respectively). Overall, the 1-year incidence estimate of BTIs/hospitalization/death was 24% (18.7-29.4%). A greater risk of incident infections was observed when BA.5 and XBB/EG sub-lineages circulated (aRR 5.05 [2.17, 11.77]; P <.001 and 3.82 [1.50, 9.7]; P = 0.005, compared to BA.1, respectively). The 1-year incidence of SARS-CoV-2 BTIs/hospitalization/death was 24% which is in line with what was observed in other similar studies. The risk appeared to be higher when more recent Omicron sub-lineages were circulating suggesting a reduction of in vitro neutralization. [ABSTRACT FROM AUTHOR]

(1) Background: We compared the diagnostic and prognostic performance of serum amyloid A (SAA), procalcitonin (PCT), delta neutrophil index (DNI), and C-reactive protein (CRP) in patients with hematologic diseases; (2) Methods: We retrospectively collected the remaining serum samples from patients with hematologic diseases, analyzed their clinical data, and measured the levels of PCT, DNI, CRP, and SAA. Performances for infection diagnosis were evaluated using a receiver operating characteristic curve analysis, and 90-day mortality was analyzed using Kaplan–Meier estimation; (3) Results: The levels of all markers were significantly higher in the infected group (N = 27) than those in the uninfected group (N = 100) (p < 0.0001 for all markers). The areas under the curve for diagnosing infection for PCT, DNI, CRP, and SAA were 0.770, 0.817, 0.870, and 0.904, respectively. Increased PCT levels were associated with higher mortality (p = 0.0250); this association was not observed with other examined markers; (4) Conclusions: CRP and SAA exhibited greater discriminative power for infection than PCT. However, only PCT levels were positively associated with 90-day mortality. Herein, we evaluated the diagnostic performance of the four markers. Additional studies are needed to confirm the findings of the present study and validate the potential of these markers in clinical practice.

배경: Candidemia is the most common form of invasive candidiasis and is still associated with high mortality rates in immunocompromised patients. It is known that the distribution of Candida species can be varied according to the region and patient group. Recently, active antifungal prophylaxis has been increased in patients with hematologic diseases, which might influence the characteristics of candidemia. Therefore, we aimed to assess the characteristics of candidemia including species distribution, antifungal resistance, and outcomes of patients. 방법: A retrospective data review was performed at the Catholic Hematology Hospital from August 2011 to November 2021. Candidemia was defined as at least one positive blood culture for Candida sp. Different episodes of candidemia in the same patient were counted independently. Only the initial Candida isolate was included within an episode. Two or more documented species of Candida were all counted independently. 결과: A total of 155 candidemia episodes were identified from 150 patients and demographics are shown in Table 1. 62 Patients (41.3%) were neutropenic state (ANC <500cells/µL) when diagnosed as candidemia and catheter-related origin was the most common secondary cause with 46 patients (30.7%). Candida species found are reported in Table 2, 38 cases (24.5%) of candidemia were caused by C. albicans and 117 cases (75.5%) by non-albicans species. Among non-Candida albicans sp., C. tropicalis was most common (36.8%). Most common species was C. tropicalis (36.8%), followed by C. albicans (24.5%), C. glabrata (15.5%), C. krusei (10.3%). The results of antifungal susceptibility are shown in Table 3. Overall mortality rate at 28-day was 40%. 결론: In this study, C. tropicalis is the most common species in patients with hematologic disease and non-albicans Candida sp. accounted for three-fourths of candidemia. Mortality rate is still high despite treatment and appropriate treatment strategy should be decided regarding the distribution and resistance pattern. [ABSTRACT FROM AUTHOR]

OBJECTIVE Prophylaxis with posaconazole (PP) is effective in the prevention of invasive fungal infections in immunocompromised adult patients. However, evaluation of its effectiveness and safety in children is limited. The aim of the study was to describe the use of posaconazole as antifungal prophylaxis in children. METHODS We reviewed the medical records of immunocompromised patients younger than 13 years with hematologic diseases and post hematopoietic stem cell transplant (HSCT) who received antifungal PP at the Instituto Nacional de Salud del Niño San Borja (INSN-SB) in Lima, Peru, from January 2014 to December 2018. RESULTS Fifty-six courses of PP were identified in 47 patients with a median age of 7.5 years (IQR, 4-10), 51.6% (n = 24) of whom were female. The main underlying medical conditions were aplastic anemia (n = 19, 33.9%), acute lymphoblastic leukemia (n = 18, 32.1%), acute myeloid leukemia (n = 14, 25.0%), and 34.1% had undergone HSCT. The median dose of posaconazole was 13.62 mg/kg/day (IQR, 12.0-16.8), and the median duration of PP was 24 days (IQR, 16-82). Gastrointestinal symptoms included abdominal pain (17.9%), nausea (16.1%), diarrhea (7.1%), and vomiting (3.6%). Elevated alanine aminotransferase and aspartate aminotransferase levels were observed in 9/35 patients (25.7%) and 10/51 (19.6%) patients, respectively. Five cases of breakthrough fungal infection were identified (8.9%). CONCLUSIONS Patients younger than 13 years who received PP showed an increase in transaminase values, and the development of breakthrough fungal infections. [ABSTRACT FROM AUTHOR]

Purpose: We performed a retrospective study to determine the effectiveness and feasibility of articulating linear stapler in laparoscopic total splenectomy (LTS) and laparoscopic partial splenectomy (LPS), focusing on technical laparoscopic skills that could help pediatric surgeons to avoid intra- and postoperative complications. Methods: Retrospective cohort study of children younger than 18 years who underwent laparoscopic spleen surgery between January 2008 and March 2020. Age, sex, indication for surgery, operative time (OT), intra- and postoperative complications, and postoperative length of hospital stay (LHS) were analyzed. Data from parenchymal resection and vessels sealing techniques were obtained. Results: Thirty patients, 19 LTS and 11 LPS, were included. The mean age of the patients was 10.9 years, and 16 patients were male and 14 were female. For hematologic diseases, LTS was the elective surgery, associated with cholecystectomy in 5 cases. LPS was the common procedure for splenic cysts. The stapler was used in LTS to close the hilum vessels and in LPS for parenchymal resection. No statistically significant differences in OT were observed comparing LTS and LPS. Two conversions occurred in LTS; none in LPS. The mean LHS was 6 days in both groups. No recurrence or major complications appeared in both groups at 1-12 years of follow-up. In particular for LPS, there are no relapse of cyst neither reduction in splenic function. Conclusions: This study shows the effectiveness, feasibility, and safety of mechanic stapler in splenic surgery both for hilum vessels sealing and for parenchymal resection. The use of this device can reduce risk of hemorrhagic recurrences or major surgical complications improving the safety of the operation. [ABSTRACT FROM AUTHOR]

Partial, or subtotal, splenectomy (PS) has become an accepted alternative to total splenectomy (TS) for management of hematologic disorders in children, but little is known about its long-term outcomes. Here, we present our institutional experience with partial splenectomy, to determine rate of subsequent TS or cholecystectomy and identify if any factors affected this need. All patients who underwent partial splenectomy at a single tertiary children's hospital were retrospectively reviewed from 2002 through 2019 after IRB approval. Primary outcome of interest was rate of reoperation to completion splenectomy (CS) and rate of cholecystectomy. Secondary outcome were positive predictor(s) for these subsequent procedures. Twenty-four patients underwent PS, at median age 6.0 years, with preoperative spleen size of 12.7 cm by ultrasound. At median follow up time of 8.0 years, 29% of all patients and 24% of hereditary spherocytosis (HS) patients underwent completion splenectomy at median 34 months and 45 months, respectively. Amongst HS patients who did not have a cholecystectomy with or prior to PS, 39% underwent a delayed cholecystectomy following PS. There were no significant differences in age at index procedure, preoperative splenic volume, weight of splenic specimen removed, transfusion requirements, preoperative or postoperative hematologic parameters (including hemoglobin, hematocrit, total bilirubin, and reticulocyte count) amongst patients of all diagnoses and HS only who underwent PS alone compared to those who went on to CS. There were no cases of OPSS or deaths. Partial splenectomy is a safe alternative to total splenectomy in children with hematologic disease with theoretical decreased susceptibility to OPSS. However, families should be counseled of a 29% chance of reoperation to completion splenectomy, and, in HS patients, a 39% chance of delayed cholecystectomy if not performed prior to or with PS. Further studies are needed to understand predictors of these outcomes. [ABSTRACT FROM AUTHOR]

Introduction: Scarce real-life data exists for COVID-19 management in hematologic disease (HD) patients in the Omicron era., Purpose: To assess the current clinical management and outcome of SARS-CoV-2 infection diagnosed, identify the risk factors for severe outcomes according to the HD characteristics and cell therapy procedures in a real-world setting., Methods: A retrospective observational registry led by the Spanish Transplant Group (GETH-TC) with 692 consecutive patients with HD from December 2021 to May 2023 was analyzed., Results: Nearly one-third of patients (31%) remained untreated and presented low COVID-19-related mortality (0.9%). Nirmatrelvir/ritonavir was used mainly in mild COVID-19 cases in the outpatient setting (32%) with a low mortality (1%), while treatment with remdesivir was preferentially administered in moderate-to-severe SARS-CoV-2 infection cases during hospitalization (35%) with a mortality rate of 8.6%. The hospital admission rate was 23%, while 18% developed pneumonia. COVID-19-related mortality in admitted patients was 14%. Older age, autologous hematopoietic stem cell transplantation (SCT), chimeric antigen receptor T-cell therapy, corticosteroids and incomplete vaccination were factors independently associated with COVID-19 severity and significantly related with higher rates of hospital admission and pneumonia. Incomplete vaccination status, treatment with prior anti-CD20 monoclonal antibodies, and comorbid cardiomyopathy were identified as independent risk factors for COVID-19 mortality., Conclusions: The results support that, albeit to a lower extent, COVID-19 in the Omicron era remains a significant problem in HD patients. Complete vaccination (3 doses) should be prioritized in these immunocompromised patients. The identified risk factors may help to improve COVID-19 management to decrease the rate of severe disease, ICU admissions and mortality., Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2024 Piñana, Vazquez, Heras, Aiello, López-Corral, Arroyo, Soler-Espejo, García-Cadenas, Garcia-Gutierrez, Aroca, Chorao, Olave, Lopez-Jimenez, Gómez, Arellano, Cuesta-Casas, Avendaño-Pita, González-Santillana, Hernández-Rivas, Roldán-Pérez, Mico-Cerdá, Guerreiro, Morell, Rodriguez-Galvez, Labrador, Campos, Cedillo, Vidal, Martino and Solano.)

Background and Objectives: There are conflicting studies on the prevalence of mediastinal lymphadenopathy (LAP) and its relationship to the prognosis of COVID-19 disease. The prevalence varied from 3.4 to 66 percent and more prevalent in patients who died. This study aimed to investigate the mediastinal lymphadenopathy and the disease progression in COVID-19 patients. Materials and Methods: In this case-control study, 195 COVID-19 patients were divided into two groups, with the mediastinal lymphadenopathy and without it. In these groups, demographic characteristics, underlying diseases, laboratory results, and outcomes were compared. Results: The median age in the LAP group was higher than the opposite group (62 vs. 58.5; p= 0.037). SpO2 (85% vs. 90%; P <0.001), lymphocyte count (760 vs. 969; p= 0.02), Neutrophil-to-Lymphocyte Ratio (5.53 vs. 4.41; p= 0.02), and ESR (36 vs. 29; p= 0.03) were significantly correlated with the presence of lymphadenopathy, using the Mann-Whitney Wilcoxon rank test. ICU admission (65.71% vs. 36.87; p= 0.003), mechanical ventilation (31.42% vs. 13.75%; p= 0.022), disease severity (65.71% vs. 40%; p <0.01), length of hospital stay (9 vs. 7; p= 0.039) and mortality rate (40% vs. 21.25%; p= 0.034) were more predominantly observed in the LAP group, using the χ2 test. There was no apparent difference in sex and the underlying diseases among the two groups. Conclusion: This observation showed a relatively high prevalence of mediastinal lymphadenopathy in COVID-19 patients, which was more common in the elderly with low oxygen saturation. Therefore, LAP may lead to further intensive care needs, more use of mechanical ventilation, high severity of disease, and mortality rate. [ABSTRACT FROM AUTHOR]

Background and Objectives: There are conflicting studies on the prevalence of mediastinal lymphadenopathy (LAP) and its relationship to the prognosis of COVID-19 disease. The prevalence varied from 3.4 to 66 percent and more prevalent in patients who died. This study aimed to investigate the mediastinal lymphadenopathy and the disease progression in COVID-19 patients. Materials and Methods: In this case-control study, 195 COVID-19 patients were divided into two groups, with the mediastinal lymphadenopathy and without it. In these groups, demographic characteristics, underlying diseases, laboratory results, and outcomes were compared. Results: The median age in the LAP group was higher than the opposite group (62 vs. 58.5; p= 0.037). SpO2 (85% vs. 90%; P

Programed death-1 (PD-1) is an immune checkpoint pathway used by cancer cells to evade the anti-cancer activity of T cells. If this pathway is active, inhibitory signals create an unresponsive state, bringing about the tumor growth. Nowadays, the PD-1/PD-L1, L2 inhibitor therapy shows a new way of treatment to clinicians who are working with hemato-oncologic cancers. Some diseases, such as classic Hodgkin lymphoma (cHL) and primary mediastinal large-B-cell lymphoma, that express large amounts of programed death-ligand 1(PD-L1), may be a good target of such a therapy. In hematology, the anti-PD-1 therapy is used successfully and safely in cHL. Other studies are limited, or the results are not available yet. Although most of the diseases, except chronic lymphocytic leukemia and multiple myeloma, show meaningful responses, when using these drugs, we must carefully monitor autoimmune and rare, but serious side effects. In this paper, we emphasize the use of PD-1 inhibitors in hematology.

The role of antithymocyte globulin (ATG) in patients with hematologic diseases undergoing umbilical cord blood transplantation (UCBT) remains controversial. This systematic review and meta‐analysis was conducted to comprehensively evaluate this issue. PubMed, Embase, and the Cochrane Library were systematically searched. Clinical studies reporting the impact of ATG‐ vs non‐ATG‐containing conditioning regimens on transplantation outcomes were identified. Twenty‐five studies were included. ATG significantly prevented grade II‐IV and grade III‐IV acute graft‐vs‐host disease (GVHD) (11 studies, 5020 patients, HR: 0.49, 95% CI: 0.42‐0.56, P <.001; 5 studies, 5490 patients, HR: 0.60, 95% CI: 0.46‐0.80, P <.001) but not chronic GVHD (8 studies, 5952 patients, HR: 0.78, 95% CI: 0.51‐1.20, P =.266). However, use of ATG was associated with increased transplantation‐related mortality and inferior overall survival (9 studies, 4244 patients, HR: 1.79, 95% CI: 1.38‐2.33, P <.001; 8 studies, 5438 patients, HR: 1.96, 95% CI: 1.56‐2.46, P <.001). Our study did not recommend routine use of ATG in UCBT. Individualizing the ATG timing and dose based on patient characteristics to retain the prophylactic effects of ATG on GVHD without compromising the survival of UCBT recipients may be reasonable. [ABSTRACT FROM AUTHOR]

• Infectious complications represent an important limitation for hematopoietic stem cell transplantation (HSCT) outcomes. • Host immunity and diagnostic limitations make latent tuberculosis infection (LTBI) an underestimated problem. • Recipients diagnosed with LTBI could benefit from pre-HSCT treatment. • Prospective data is required to define the best strategies for LTBI in the HSCT setting. Latent tuberculosis infection (LTBI) affects one-fourth of the world´s population. Hematopoietic stem cell transplantation (HSCT) recipients are at an elevated risk of developing active tuberculosis infection (ATBI). In this retrospective study of donors and HSCT recipients who underwent transplantation between February 2000 and June 2018, our aim was to determine the prevalence of LTBI and ATBI and to describe diagnostic and therapeutic strategies in an HSCT population in an endemic region. The cohort of 409 participants included 125 allogeneic HSCT (allo-HSCT) recipients, 165 autologous HSCT (auto-HSCT) recipients, and 119 HSCT donors. Patients were evaluated pre-HSCT with tuberculin skin test and thoracic imaging. LTBI was diagnosed in 26.2% of the cohort. Cases represented 20% of the auto-HSCT population, 20% of the allo-HSCT population, and 41.2% of the donor population. Pre-HSCT evaluation to rule out ATBI was performed in 62.6% of the cohort; all results were negative. Isoniazid was administered to 73.3% of those with LTBI. Within subgroups, 91.7% of HSCT recipients and 51% of donors received treatment. The median duration of therapy pre-HSCT was 70 days in recipients and 48 days in donors. The incidence of post-HSCT ATBI was 0 at 1-year follow-up. The incidence of LTBI in our population was higher than expected and still might have been underestimated owing to diagnostic test limitations. The absence of incident ATBI suggests that recipients, as opposed to donors, must receive LTBI treatment. Prevention of infectious complications in the HSCT population should be prioritized to improve clinical outcomes. Prospective data from collaborative working groups is needed to determine the best diagnostic and therapeutic approaches in this vulnerable patient population. [ABSTRACT FROM AUTHOR]

Objective: To compare the ultrasound- and fluoroscopy-guided subclavian vein catheterization in pediatric population with hematologic diseases.Methods: A randomized prospective study of subclavian vein catheterization in pediatric population with hematologic diseases was performed. After randomization, the patients were assigned to either ultrasound- or fluoroscopy-guided subclavian vein catheterization. The primary outcome was number of attempts at venous cannulation. Secondary outcomes included: catheterization success rate, fluoroscopy time, operation time, and surgical complications.Results: There were 170 children enrolled between February 2017 and July 2018. There was no difference between the two groups with regard to the demographic data. Success within 3 attempts was achieved in 82 cases (82/87, 92.0%) in the ultrasound (US) group vs. 65 cases (65/83, 78.3%) in the fluoroscopy group (P = 0.002). The average operation time was 10(7) min in US group vs. 10(6) min in fluoroscopy group (P = 0.722). There were 3 complications in the US group, while there were 6 complications in the fluoroscopy group (P = 0.321). There were 4 catheter-related thrombosis (CRTs) found in the US group during follow-up, however there was no CRT in the fluoroscopy group (P = 0.121).Conclusions: Ultrasound-guided venous puncture is a more accurate method of subclavian vein catheterization. However, the catheter tip can be placed more precisely by fluoroscopy. Thus, combined ultrasound-and fluoroscopy-guided technology is more efficient in subclavian vein catheterization of children with hematologic disease. [ABSTRACT FROM AUTHOR]

Blood disorders are defined as diseases related to the structure, function, and formation of blood cells. These diseases lead to increased years of life loss, reduced quality of life, and increased financial burden for social security systems around the world. Common blood disorder treatments such as using chemical drugs, organ transplants, or stem cell therapy have not yet approached the best goals, and treatment costs are also very high. RNA with a research history dating back several decades has emerged as a potential method to treat hematological diseases. A number of clinical trials have been conducted to pave the way for the use of RNA molecules to cure blood disorders. This novel approach takes advantage of regulatory mechanisms and the versatility of RNA-based oligonucleotides to target genes and cellular pathways involved in the pathogenesis of specific diseases. Despite positive results, currently, there is no RNA drug to treat blood-related diseases approved or marketed. Before the clinical adoption of RNA-based therapies, challenges such as safe delivery of RNA molecules to the target site and off-target effects of injected RNA in the body need to be addressed. In brief, RNA-based therapies open novel avenues for the treatment of hematological diseases, and clinical trials for approval and practical use of RNA-targeted are crucial., (Copyright © 2024. Published by Elsevier Inc.)

Programed death-1 (PD-1) is an immune checkpoint pathway used by cancer cells to evade the anti-cancer activity of T cells. If this pathway is active, inhibitory signals create an unresponsive state, bringing about the tumor growth. Nowadays, the PD-1/PD-L1, L2 inhibitor therapy shows a new way of treatment to clinicians who are working with hemato-oncologic cancers. Some diseases, such as classic Hodgkin lymphoma (cHL) and primary mediastinal large-B-cell lymphoma, that express large amounts of programed death-ligand 1(PD-L1), may be a good target of such a therapy. In hematology, the anti-PD-1 therapy is used successfully and safely in cHL. Other studies are limited, or the results are not available yet. Although most of the diseases, except chronic lymphocytic leukemia and multiple myeloma, show meaningful responses, when using these drugs, we must carefully monitor autoimmune and rare, but serious side effects. In this paper, we emphasize the use of PD-1 inhibitors in hematology. [ABSTRACT FROM AUTHOR]

Objective: To explore the clinical characteristics of hospitalized patients with hematologic diseases complicated with carbapenem-resistant organisms (CRO) infection and analyze the risk factors of 30-day all-cause mortality., Methods: The clinical data and laboratory test data of 77 hospitalized patients with hematologic diseases complicated with CRO infection in department of hematology of the Third Hospital of Shanxi Medical University from January 2015 to December 2020 were retrospectively analysed, the risk factors of 30-day all-cause mortality after CRO infection were analyzed by multivariate logistic regression., Results: Among the total of 77 patients with hematologic diseases complicated with CRO infection, 29 died and 48 survived within 30 days of infection, with a case fatality rate of 37.66%. A total of 93 strains of CRO were isolated from these patients, of which Acinetobacter baumannii had the highest detection rate (25.81%, 24/93), followed by Pseudomonas aeruginosa (18.28%, 17/93). The lung was the most common site of CRO infection. The detected pathogens were highly resistant to carbapenems, and 64.52% (60/93) of the pathogens were resistant to imipenem with minimum inhibitory concentration (MIC)≥16 μg/ml. The results of the univariate analysis showed that albumin concentration <25 g/L ( P =0.048), serum creatinine concentration≥120 μmol/L ( P =0.023), age-adjusted Charlson comorbidity index (ACCI) ( P =0.037) and primary treatments (supportive treatment, immunosuppressive therapy, chemotherapy, HSCT) ( P =0.048) were significantly associated with 30-day all-cause mortality after infection. The results of multivariate logistic regression analysis showed that when CRO infection confirmed, albumin concentration <25 g/L ( P =0.014, OR =6.171), serum creatinine concentration≥120 μmol/L ( P =0.009, OR =10.867) were independent risk factors for 30-day mortality of patients with hematologic diseases complicated with CRO infection., Conclusion: The mortality rate of CRO-infected patients with hematologic diseases is high. The detected pathogenic bacteria are highly resistant to imipenem. The albumin concentration <25 g/L and the serum creatinine concentration≥ 120 μmol/L at diagnosis of CRO infection were independent risk factors for 30-day mortality of the patients with hematologic diseases.

Hematopoietic stem cell transplantation (HSCT) is an effective therapy for acute leukemia (AL). Relapse represents the main cause of mortality. Isolated extramedullary relapse (iEMR) is atypical and has been related to better outcomes. Here we describe the clinical characteristics and outcomes of AL relapse after HSCT in our study population and analyze the impacts of different types of relapse on survival outcomes. This retrospective, multicenter study included 124 patients age ≥15 years with AL who underwent HSCT between 2004 and 2019. At diagnosis, 66.1% of the patients had lymphocytic AL, 19.7% presented with high-risk features, and 18.5% had extramedullary disease (EMD). At HSCT, 83.1% of the patients were in complete remission (CR), and 44.8% had negative measurable residual disease (MRD). The vast majority of donors were related (96%), including 48.4% HLA-matched and 47.6% haploidentical. Myeloablative conditioning was provided to 80.6% of patients. The median overall survival (OS) was 15 months (95% confidence interval [CI] 9.9 to 20.1 months). Factors associated with improved OS were adolescent and young adult (AYA) patient (P = .035), first or second CR (P = .026), and chronic graft-versus-host disease (GVHD) (P < .001). Acute GVHD grade III-IV (P = .009) was associated with increased mortality. The median relapse-free survival was 13 months (95% CI, 7.17 to 18.8 months); early disease status (P = .017) and chronic GVHD (P < .001) had protective roles. Sixty-eight patients (55%) relapsed after HSCT, with a median time to relapse of 6 months (95% CI, 3.6 to 8.4 months). iEMR was reported in 16 patients (23.5%). The most commonly involved extramedullary sites were the central nervous system and skin. Compared to patients with bone marrow relapse, all patients with iEMR had a diagnosis of acute lymphoid leukemia (P = .008), and 93.8% belonged to the AYA group; regarding pre-HSCT characteristics, iEMR patients had higher rates of negative MRD (P = .06) and a history of EMD (P = .009). Seventy-seven percent of relapsed patients received additional treatment with curative intent. The median OS after relapse (OSr) was 4 months (95% CI, 2.6 to 5.4 months). Factors related to increased OSr included lymphoid phenotype (P = .03), iEMR (P = .0042), late relapse (≥6 months) (P = .014), receipt of systemic therapy including second HSCT (P < .001), and response to therapy (P < .001). Rates of relapse and iEMR were higher than those previously reported in other studies. Advanced disease, reduced-intensity conditioning, and a diminished graft-versus-leukemia effect were factors influencing these findings. At relapse, presenting with iEMR after 6 months and receiving intensive therapy with adequate response were associated with better outcomes. Our results strongly suggest that a personalized approach to treating patients with HSCT is needed to counterbalance specific adverse factors and can positively impact clinical outcomes., (Copyright © 2023 The American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc. All rights reserved.)

Background: Iron overload (IOL) due to repetitive transfusions of packed red blood cells (pRBC) has a major impact on morbidity and mortality in patients with inherited bone marrow failure syndromes and hemoglobinopathies such as thalassemia and sickle cell disease. However, whether IOL influences the outcome of elderly patients with myeloid malignancies is not yet clear. Moreover, clinical trials have reported high drop-out rates during treatment with the oral iron chelator deferasirox (DFX).Aim: Here we report the results of a 2-year prospective observational study that aimed at describing the routine use of DFX in patients with hematological malignancies with regard to safety, efficacy and handling of the drug in a routine setting.Results: A total of 406 patients were included. 58% of the patients were male. Most of the patients had myelodysplastic syndromes (MDS) (68%) and myeloproliferative neoplasms (MPN) (14%). Median time from first transfusion to study enrollment was 1.1 years (0-25.5 years) and most patients were chelation naive (91%) at enrollment. With regard to transfusion burden, most of the patients were moderately or mildly transfusion-dependent with 53% receiving 2-4 and 27% receiving less than 2 units of pRBC per month. Serum ferritin decreased from a mean of 2305 μg/l (± 1449 μg/l) to a mean of 1910 μg/l (± 1529 μg/l) at 24 months. There was no substantial change in transfusion-dependence during the observation period. Dose adjustments were reported in 48% of the patients with dose-escalation strategies being the most frequent reason for dosage increases (49%). The median observation time was 355 days (5-1080 days). Median duration of exposure to DFX was 322 days (2-1078 days). Two-hundred and ninety (72%) patients discontinued the trial prematurely after a median time of 235 days (1-808 days). Death (29%) and adverse events (23%) were the main reasons for discontinuation. Eleven percent of the patients discontinued treatment due to sufficient decrease in serum ferritin. Most frequent adverse events were decrease in creatinine clearance (22%), increase in serum creatinine (18%) and diarrhea (16%).Conclusion: This descriptive trial confirms the efficacy of DFX in decreasing the serum ferritin. Moreover, the high drop-out rates seen in prospective trials are recapitulated in this study, which can be attributed to adverse events in a substantial proportion of patients. [ABSTRACT FROM AUTHOR]

OBJECTIVES: To determine the effects of a virtual reality intervention on pain and anxiety in patients undergoing a bone marrow aspiration and biopsy procedure. SAMPLE & SETTING: 97 adults in an outpatient cancer center in the midwestern United States. METHODS & VARIABLES: In this quasiexperimental study, participants were assigned to either the experimental group (use of virtual reality goggles) or the control group (standard treatment). Vital signs, pain, and anxiety were measured before and after the procedure. T tests and chi-square tests were used to compare the two groups based on demographic data, pain, anxiety, and total amount of medication used during the procedure. RESULTS: Participants who wore virtual reality goggles during a bone marrow aspiration and biopsy procedure did not experience a statistically significant decrease in pain and anxiety. However, the experimental and control groups showed a decrease in pain and anxiety levels from pre- to postprocedure. IMPLICATIONS FOR NURSING: Virtual reality goggles may be a feasible, noninvasive alternative to traditional pharmacologic treatment. The cost of any intervention would need to be considered. Virtual reality should be researched and adapted to benefit patients in appropriate settings. [ABSTRACT FROM AUTHOR]