Underwent Hematopoietic Stem Cell Transplantation Research Articles

Oral Mucositis in a Patient With Aplastic Anemia Who Underwent Hematopoietic Stem Cell Transplantation: A Case Report

Oral Mucositis in a Patient With Aplastic Anemia Who Underwent Hematopoietic Stem Cell Transplantation: A Case Report

Conditioning Regimens in Patients with β-Thalassemia Who Underwent Hematopoietic Stem Cell Transplantation: A Scoping Review.

The success of transplant procedures in patients with beta-thalassemia major (β-thalassemia) goes hand-in-hand with improvements in disease knowledge, better supportive care, discoveries in immunogenetics, increase in stem cell sources, and enhancement of conditioning regimens. The aim of this scoping review was to report the evolution of conditioning regimes for β-thalassemia hematopoietic stem cell transplantation. We performed a systematic search for all relevant articles published before July 2021, using the following Medical Subject Headings: “bone marrow transplantation”, “stem cell transplantation”, “allogeneic”, “thalassemia”, “β-thalassemia”, and “thalassemia major”. The final analysis included 52 studies, published between 1988 and 2021, out of 3877 records. The most common conditioning regimen was a combination of busulfan and cyclophosphamide, with successive dose adjustments or remodulation based on patient characteristics. Pre-transplant treatments, reductions in cyclophosphamide dosage, or the adoption of novel agents such as treosulphan all improved overall survival and thalassemia-free survival in transplant-related mortality high-risk patients. Conditioning regimes were modulated for those without a suitable fully matched sibling or unrelated donor, with encouraging results. Hematopoietic stem cell transplantation with haploidentical donors is currently available to virtually all patients with β-thalassemia. However, disparities in outcome are still present around the world. In developing and limited-resource countries, where most diagnoses are focused, transplants are not always available. Therefore, more efforts are needed to close this treatment gap.

Systematic Review and Meta-Analysis of Health-Related Quality of Life in Patients with β-Thalassemia Underwent Hematopoietic Stem Cell Transplantation

Background: β-Thalassemia major (β-TM) represents one of the most important hemoglobinopathies worldwide. Remarkable improvements have been achieved in supportive therapy, based on blood transfusions and iron chelation, and nowadays this approach is capable to assure a long life in these patients in industrialized countries. The only curative treatment is represented by hematopoietic stem cell transplantation (HSCT). However, this treatment may be burdened by deterioration in the Health-Related Quality of Life (HRQoL). The aim of this paper was to evaluate with a systematic review and meta-analysis the role of HRQoL in transplanted β-TM patients. Methods: PubMed database, Web of Science and Scopus were systematically searched for studies published between January 1st, 2000 to September, 2020; the following terms were entered in the database queries: β-thalassemia, HRQoL and HSCT. The study was carried out according to the Preferred Reporting Items for Systematic and Meta-analyses (PRISMA) statement. Results: We identified a total of 33 potential studies. Among these, 10 were finally considered in the systematic review and 5 in the meta-analysis. Overall, good scores in the principal domains of HRQoL were reported by transplanted patients. These data were confirmed by results of meta-analysis that showed significant difference between transplanted and β-TM patients treated with a conventional therapy in Physical an Emotional Function dimension, with a medium effect size (d=0.65, (95% CI (0.29-1.02) z = 3.52, p =0.0004, I2=75% and d=0.59, (95% CI (0.43-0.76) z = 6.99, p <0.00001, I2=0% respectively). Conclusion: HRQoL is generally good in β-TM transplanted patients and may give a significant contribute the difficult choice whether transplant or not a β-TM patient treated with conventional therapy.

Thrombotic Microangiopathy: Multi-Institutional Review of Pediatric Patients Who Underwent HSCT.

Thrombotic microangiopathy (TMA) is a rare but serious complication of hematopoietic stem cell transplantation (HSCT). The purpose of our study is to estimate the incidence, prevalence, and analyze the risk factors and outcome of TMA in children receiving HSCT. Patients under the age of 21 who underwent HSCT at one of the 42 Pediatric Health Information System (PHIS) hospitals from 2000–2012 were analyzed, including demographics, hospitalizations, TMA, and other HSCT-related complications. From 2000 to 2012, a total of 12,369 unique pediatric patients who received HSCT were identified. Among these, 93 (0.8%) children were identified to have the diagnosis of TMA. TMA was significantly associated with allogeneic HSCT, peripheral blood stem cell trasnplants (PBSCT), cytomegalovirus (CMV), human herpes virus 6 (HHV6), fungal infection, graft-versus-host disease (GVHD), and veno-occlusive disease (VOD) (p = 0.01). Multivariate logistic regression analysis of mortality showed only HHV6 was an independent risk factor associated with increased mortality in patients with TMA (hazard ratio: 2.86 [1.01, 8.39], p = 0.05). The prevalence of TMA in our study is 0.8% with a mortality in our pediatric TMA cohort of 30%, which is in contrast to the higher mortality reported in previously published, small-case series. HHV6 emerged as not only a risk factor for TMA but also as associated with increased mortality in these patients.

Electronic Symptom Assessment in Children and Adolescents With Advanced Cancer Undergoing Hematopoietic Stem Cell Transplantation.

Background/Purpose: Effective symptom assessment and management for children with advanced cancer undergoing hematopoietic stem cell transplantation (HSCT) is critical to minimize suffering. The purpose of this subanalysis was to compare feasibility of electronic data collection data and symptom prevalence, frequency, severity, and distress from children with advanced cancer undergoing HSCT with a non-HSCT cohort. Method: An abbreviated Pediatric Quality of Life and Evaluation of Symptoms Technology Memorial Symptom Assessment Scale was electronically administered every 2 weeks to children with advanced cancer. A subanalysis was conducted for the cohort of children who received autologous or allogeneic HSCT. Results: Forty-six participants completed 563 symptom assessments during the study. However, 11 of these 46 children received HSCT and completed 201 symptom assessments. The median age in the HSCT cohort was 12.7 years, 73% were female, and most children had a hematologic (45%) or solid tumor (45%) malignancy. Pain (35%), nausea (30%), sleeping difficulty (29%), and fatigue (22%) were the most commonly reported symptoms in children receiving HSCT. Children in the HSCT cohort had similar total, subscale, and individual symptom scores compared with children who did not receive HSCT. Certain domains of gastrointestinal symptoms (nausea, lack of appetite, and diarrhea) were higher for children receiving HSCT compared with children with advanced cancer not receiving HSCT (p < .05). Conclusion: Elicitation of patient-reported symptom experiences using electronic methods improves nurses' understanding of the symptom experience for children with advanced cancer undergoing HSCT and may promote timely assessment and treatment of distressing symptoms.

A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456

A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456

Electronic Symptom Assessment in Children and Adolescents with Advanced Cancer Undergoing Hematopoietic Stem Cell Transplantation

Background Effective symptom assessment and management for children with advanced cancer undergoing hematopoietic stem cell transplantation (HSCT) is critical to minimize suffering. Most literature specific to children receiving HSCT is based upon medical record reviews and parent report, and to a lesser extent, patient self-report. Objectives The purpose of this study was to describe symptom frequency, severity and level of distress in children and adolescents undergoing HSCT using patient self-report via electronic data collection. Methods A modified Memorial Symptom Assessment Scale (MSAS) was electronically administered to children with advanced cancer every two weeks. Clinical data was collected at corresponding time points. A sub-analysis was conducted for the cohort of children with advanced cancer who received autologous or allogeneic HSCT. Results Forty-seven children with advanced cancer were enrolled and completed 563 surveys during the course of the study. Eleven of these 47 children received HSCT and completed 201/563 surveys. The median age of children in the HSCT cohort was 12.7 years, 73% were female, and most children had a hematologic (45%) or solid tumor (45%) malignancy. Ninety-six percent of surveys administered to children receiving HSCT were completed, and 64% of surveys were completed at home. Pain (35%), nausea (30%), sleeping difficulty (29%) and fatigue (22%) were the most commonly reported symptoms in children receiving HSCT. Children in the HSCT cohort had higher total and physical subscale mean MSAS scores, and higher pain, nausea, fatigue, and lack of appetite mean scores compared to children with advanced cancer not receiving HSCT (p Conclusion Children with advanced cancer receiving HSCT may have increased symptom burden with higher levels of distress as compared to those who do not receive HSCT. Increased understanding of the symptom experience for children with advanced cancer undergoing HSCT may promote timely assessment and treatment of distressing symptoms.

610 - Frequency of Veno-Occlusive Disease in Children with B-Thalassemia Major Who Underwent Hematopoietic Stem Cell Transplantation

610 - Frequency of Veno-Occlusive Disease in Children with B-Thalassemia Major Who Underwent Hematopoietic Stem Cell Transplantation

Quality of Life and Psychopathology in Adults Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) in Childhood: A Qualitative and Quantitative Analysis.

Background: Patients who undergo pediatric Hematopoietic Stem Cell Transplantation (HSCT) may experience long-term psychological sequelae and poor Quality of Life (QoL) in adulthood. This study aimed to investigate subjective illness experience, QoL, and psychopathology in young adults who have survived pediatric HSCT.Method: The study involved patients treated with HSCT in the Hematology-Oncology Department between 1984 and 2007. Psychopathology and QoL were investigated using the SCL-90-R and SF-36. Socio-demographic and medical information was also collected. Finally, participants were asked to write a brief composition about their experiences of illness and care. Qualitative analysis of the texts was performed using T-LAB, an instrument for text analysis that allows the user to highlight the occurrences and co-occurrences of lemma. Quantitative analyses were performed using non-parametric tests (Spearman correlations, Kruskal-Wallis and Mann-Whitney tests).Results: Twenty-one patients (9 males) participated in the study. No significant distress was found on the SCL-90 Global Severity Index, but it was found on specific scales. On the SF-36, lower scores were reported on scales referring to bodily pain, general health, and physical and social functioning. All the measures were significantly (p < 0.05) associated with specific socio-demographic and medical variables (gender, type of pathology, type of HSCT, time elapsed between communication of the need to transplant and effective transplantation, and days of hospitalization). With regard to the narrative analyses, males focused on expressions related to the body and medical therapies, while females focused on people they met during treatment, family members, and donors. Low general health and treatment with autologous HSCT were associated with memories about chemotherapy, radiotherapy, and the body parts involved, while high general health was associated with expressions focused on gratitude (V-Test ± 1.96).Conclusion: Pediatric HSCT survivors are more likely to experience psychological distress and low QoL in adulthood compared with the general population. These aspects, along with survivors' subjective illness experience, show differences according to specific medical and socio-demographic variables. Studies are needed in order to improve the care and long-term follow-up of these families.

A Reduced-Intensity Conditioning Regimen for Patients with Dyskeratosis Congenita Undergoing Hematopoietic Stem Cell Transplantation

Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative option for progressive marrow failure, myelodysplastic syndrome, or leukemia associated with dyskeratosis congenita (DC). HSCT for DC is limited by a high incidence of treatment-related mortality, thought to be related to underlying chromosomal instability and sensitivity to chemotherapy and radiation. We report our experience in 7 patients with DC who underwent allogeneic transplantation using a reduced-intensity conditioning (RIC) preparative regimen that contained chemotherapy only (no radiation). This RIC regimen, designed specifically for patients with DC, contained alemtuzumab, fludarabine, and melphalan (with melphalan at 50% reduced dosing), with the goal of decreasing toxicity and improving outcome. All 7 patients engrafted, with none developing mixed chimerism or rejection. Two patients experienced acute graft-versus-host disease (GVHD) and 1 went on to develop limited chronic GVHD of the skin. Five patients remain alive and well at a median follow-up of 44 months (range, 14 to 57 months). We conclude that a radiation-free RIC regimen results in durable engraftment, acceptable toxicity, and improved overall survival in patients with DC undergoing allogeneic HSCT.

Quality of life and depression during hospitalization for hematopoietic stem cell transplantation to predict quality of life and post-traumatic stress disorder symptoms at 6 months post-transplant.

215 Background: Patients undergoinghematopoietic stem cell transplantation (HCT) experience a steep deterioration in quality of life (QOL) and mood during hospitalization for HCT. The impact of this deterioration on patients’ long-term QOL and post-traumatic stress disorder (PTSD) symptoms is unknown. Methods: We conducted a prospective longitudinal study of patients hospitalized for HCT. At baseline (day-6), day+1, day+8, and 6 months post-HCT, we assessed QOL (Functional Assessment of Cancer Therapy-Bone Marrow Transplantation [FACT-BMT]) and mood (Hospital Anxiety and Depression Scale [HADS]). We used the PTSD Checklist to assess for PTSD symptoms at 6 months. We used multivariable linear regression models to identify predictors of QOL and PTSD symptoms at 6 months post-HCT. Results: We enrolled 97% (90/93) of consecutively eligible patients undergoing autologous (n = 30), myeloablative allogeneic (n = 30), or reduced intensity allogeneic (n = 30) HCT. Overall, patients’ QOL at 6 months (mean FACT-BMT: 110, 95%CI [104-116]) recovered to baseline pre-transplant values (mean FACT-BMT: 110, 95% CI [107-115]). At 6 months, 28.4% of participants met provisional diagnostic criteria for PTSD, and 43.3% had clinically significant depression. In multivariable regression analyses adjusting for baseline QOL, mood, other covariates, change in QOL and depression scores during hospitalization for HCT predicted impaired QOL (DQOL β = 1.13, P &lt; 0.0001, D HADS-depression β = 2.51, P = 0.001) and PTSD symptoms (DQOL β = 0.50, P &lt; 0.0001, DHADS-depression β = 1.22, P &lt; 0.0001) at 6 months post-HCT. Conclusions: While patients’ overall QOL at 6 months post-HCT returned to baseline values, a significant proportion met provisional diagnostic criteria for PTSD and depression. The decline in QOL and increase in depressive symptoms during hospitalization for HCT were the most important predictors of long-term QOL impairment and PTSD symptoms. Future studies should evaluate whether interventions to improve QOL and reduce psychological distress during HCT may improve long-term QOL and reduce the risk of PTSD symptoms.

Psychological distress during hospitalization for hematopoietic stem cell transplantation to predict lower quality of life and high post-traumatic stress disorder symptoms at 6 months post-transplant.

9557 Background: Patients undergoinghematopoietic stem cell transplantation (HCT) experience a steep deterioration in quality of life (QOL) and mood during hospitalization for HCT. The impact of this deterioration on patients’ long-term QOL and post-traumatic stress disorder (PTSD) symptoms is unknown. Methods: We conducted a prospective longitudinal study of patients hospitalized for HCT. At baseline (day-6), day+1, day+8, and 6 months post-HCT, we assessed QOL (Functional Assessment of Cancer Therapy-Bone Marrow Transplantation [FACT-BMT]) and mood (Hospital Anxiety and Depression Scale [HADS]). We used the PTSD Checklist to assess for PTSD symptoms at 6 months. We used multivariable linear regression models to identify predictors of QOL and PTSD symptoms at 6 months post-HCT. Results: We enrolled 97% (90/93) of consecutively eligible patients undergoing autologous (n = 30), myeloablative allogeneic (n = 30), or reduced intensity allogeneic (n = 30) HCT. Overall, patients’ QOL at 6 months (mean FACT-BMT...

Bacteremia during Neutropenic Episodes in Children Underwent Hematopoietic Stem Cell Transplantation Receiving Ciprofloxacin and Penicillin Prophylaxis

Bacteremia during Neutropenic Episodes in Children Underwent Hematopoietic Stem Cell Transplantation Receiving Ciprofloxacin and Penicillin Prophylaxis

Eosinophils from Hematopoietic Stem Cell Recipients Suppress Allogeneic T Cell Proliferation

Eosinophilia has been associated with less severe graft-versus-host disease (GVHD), but the underlying mechanism is unknown. We hypothesized that eosinophils diminish allogeneic T cell activation in patients with chronic GVHD. The capacity of eosinophils derived from healthy subjects and hematopoietic stem cell (HSC) transplant recipients, with or without chronic GVHD, to reduce allogeneic T cell proliferation was evaluated using a mixed leukocyte reaction. Eosinophil-mediated inhibition of proliferation was observed for the eosinophils of both healthy subjects and patients who underwent HSC transplantation. Eosinophils from patients with and without chronic GVHD were equally suppressive. Healthy eosinophils required cell-to-cell contact for their suppressive capacity, which was directed against CD4+ T cells and CD8+ T cells. Neither eosinophilic cationic protein, eosinophil-derived neurotoxin, indoleamine 2,3-dioxygenase, or increased numbers of regulatory T cells could account for the suppressive effect of healthy eosinophils. Real-time quantitative PCR analysis revealed significantly increased mRNA levels of the immunoregulatory protein galectin-10 in the eosinophils of both chronic GVHD patients and patients without GVHD, as compared with those from healthy subjects. The upregulation of galectin-10 expression in eosinophils from patients suggests a stimulatory effect of HSC transplantation in itself on eosinophilic galectin-10 expression, regardless of chronic GVHD status. To conclude, eosinophils from HSC transplant recipients and healthy subjects have a T cell suppressive capacity.

Autophagy Is Required For Long-Term Hematopoietic Stem Cell (HSC) Function and G-CSF-Induced HSC Mobilization

Hematopoiesis originates from a rare pool of hematopoietic stem cells (HSC) that are uniquely capable of both self-renewal and terminal differentiation into lineage-committed progenitor cells. Autophagy is a process of cytoplasmic protein recycling which maintains cellular homeostasis and protects the cell during periods of metabolic stress and nutrient deprivation and has an established role in the survival and function of immunological cells. Recent publications have linked autophagy with preservation of normal long term HSCs (LT-HSCs) during aging (Nature; 2013: 494:323-7). We therefore sought to determine the role of autophagy in LT-HSC function at homeostasis and during the clinically relevant stress of G-CSF-induced HSC mobilization.Using single cell imaging flow cytometry to monitor autophagosome formation in LC3-GFP transgenic mice (LC3-GFP punctae formation is a reporter of autophagy activity) we demonstrated autophagic activity in HSC populations but not in committed myeloid progenitors. In line with this, inhibition of autophagy degradation by chloroquine administration resulted in the accumulation of autophagy related protein p62 in purified HSCs compared to myeloid progenitors. To determine the contribution of autophagy to HSC development and function, we analyzed mice deficient in Atg5, a protein essential for autophagosome formation. Autophagy was not required for fetal liver (FL) HSC development, however, Atg5-/- FL HSCs showed mildly reduced long-term repopulating function in bone marrow (BM) transplantation assays (16 weeks peripheral blood (PB) engraftment Atg5-/- 83.45% vs. WT 93.10%, n=12, p<0.001). Importantly, Atg5-/- LT-HSCs (Lin-Sca-1+c-kit+Flk2-CD150+CD48-) were markedly reduced in congenic recipients compared to WT FL LT-HSCs (Atg5-/- 0.02% vs. WT 0.04%, n=11, p<0.001). Secondary competitive transplantation was used to determine the effect of autophagy loss on LT-HSC function in vivo. Atg5-/- LT-HSCs exhibited a profound impairment in the repopulation of secondary congenic recipients (Atg5-/-4.65% vs. WT 32.3%, n=5, p<0.01).Mechanistically, microarray analysis of purified LT-HSCs from Atg5-/- vs. WT FL chimeras demonstrated clear differences in gene expression by unsupervised hierarchical clustering. Differentially expressed genes included Cxcl12, Sdc2 and Apex1, regulators of HSC fate. Gene Ontology enrichment analysis demonstrated that autophagy deficient LT-HSCs had impaired metabolism, enhanced cellular differentiation, enforced proliferation and increased apoptosis. Validating these findings, there was a loss of quiescence in the Atg5-/- compared to WT LT-HSC (quiescent Atg5-/- 30% vs. WT 39%, n=16 p<0.05) and Atg5-/- FL HSCs exhibited enhanced apoptosis after culture in cytokine enriched media (Atg5-/-21.2% vs. WT 14.2%, n=5, p<0.01).Given the requirement for autophagy in homeostasis of LT-HSC and its role in proliferation and metabolic stress, we next investigated whether autophagy participated in the HSC response to G-CSF. G-CSF is commonly used to ameliorate neutropenia in patients treated with chemotherapy and is also used to mobilize HSCs for patients undergoing HSC transplantation. Using single cell imaging flow cytometry, autophagosome formation was enhanced in HSCs after 6 days of G-CSF mobilization (10mcg/day). G-CSF treatment efficiently mobilized PB neutrophils and colony forming units (CFU) in WT chimeras, however Atg5-/- chimeras showed a striking reduction in G-CSF-induced neutrophil (Atg5-/- 4.95 x106/mL vs. WT 10.46 x106/mL, n=20, p<0.0001) and PB CFU mobilization post-G-CSF (Atg5-/- 47/100µL vs. WT 126/100µL, n=5, p<0.01). BM CFU numbers were similar in Atg5-/- and WT FL chimeras both pre- and post-G-CSF mobilization. Atg5-/-neutrophils demonstrated increased apoptosis after G-CSF treatment suggesting that autophagy limits PB neutrophil survival, but not BM neutrophil development during G-CSF mobilization.These data demonstrate that autophagy is an active process in LT-HSCs and that genetic deletion of Atg5 results in the failure of adult LT-HSC maintenance and function. Autophagic activity is augmented by G-CSF-induced HSC stress and is required for G-CSF-induced HSC mobilization. These findings are particularly relevant to HSC transplantation and hematopoietic function in the context of a rapid rise in the clinical use of agents that have modulatory effects on autophagy. Disclosures:No relevant conflicts of interest to declare.

Concise Review: Hematopoietic Stem Cell Transplantation: Targeting the Thymus

Allogeneic hematopoietic stem cell (HSC) transplantation can cure patients suffering from diverse genetic and acquired diseases as well as cancers. Nevertheless, under conditions where T-cell reconstitution is critical, the entry of donor progenitors into the thymus remains a major bottleneck. It is assumed that following the intravenous injection of HSC, they first home to the BM. More committed progenitors can then be exported to the thymus in response to a myriad of signals regulating thymus seeding. Notably although, the thymus is not continually receptive to the import of hematopoietic progenitors. Furthermore, as stem cells with self-renewing capacity do not take up residence in the thymus under physiological conditions, the periodic colonization of the thymus is essential for the sustained differentiation of T lymphocytes. As such, we and others have invested significant efforts into exploring avenues that might foster a long-term thymus-autonomous differentiation. Here, we review strategic approaches that have resulted in long-term T-cell differentiation in immunodeficient (SCID) mice, even across histocompatibility barriers. These include the forced thymic entry of BM precursors by their direct intrathymic injection as well as the transplantation of neonatal thymi. The capacity of the thymus to support hematopoietic progenitors with renewal potential will hopefully promote the development of new therapeutic strategies aimed at enhancing T-cell differentiation in patients undergoing HSC transplantation.

High Incidence of Radiation-Induced Cavernous Hemangioma in Long-Term Survivors Who Underwent Hematopoietic Stem Cell Transplantation with Radiation Therapy during Childhood or Adolescence

Radiation-induced cavernous hemangioma (RICH) is a late complication of cerebral radiation therapy. Long-term survivors of hematopoietic stem cell transplantation (HSCT) who underwent radiation therapy could be at increased risk for RICH. We investigated records of 68 patients who underwent HSCT during childhood or adolescence and were assessed by magnetic resonance imaging (MRI), including T2*-weighted imaging of the brain, annually for 5 years over a range of 6 to 29 years after HSCT. We developed a scoring and grading system for RICH to monitor the process and the progress of radiologic changes. Among the 68 patients investigated, 28 (41.2%) were diagnosed with CH. All 28 patients had received total body irradiation as a conditioning treatment for HSCT and/or cranial radiation therapy before HSCT as part of the treatment of their primary disease. RICH was diagnosed in none of the patients who did not receive radiation (n = 19), in 46.2% of those who received 6 to 12 Gy (n = 39), and in all of those who received 18 to 36 Gy (n = 10). Total RICH scores were correlated with higher radiation doses. Careful and long-term evaluation with MRI, including T2*-weighted imaging, is necessary for HSCT recipients who received radiation therapy before and/or during HSCT.

Safety and Efficacy of CMX001 as Salvage Therapy for Severe Adenovirus Infections in Immunocompromised Patients

No therapeutic agent has yet been established as the definitive therapy for adenovirus infections. We describe the clinical experience of 13 immunocompromised patients who received CMX001 (hexadecyloxypropyl cidofovir), an orally bioavailable lipid conjugate of cidofovir, for adenovirus disease. We retrospectively analyzed 13 patients with adenovirus disease and viremia treated with CMX001; data were available for ≥ 4 weeks after initiation of CMX001 therapy. Virologic response (VR) was defined as a 99% drop from baseline or undetectable adenovirus DNA in serum. The median age of the group was 6 years (range, 0.92-66 years). One patient had severe combined immunodeficiency, 1 patient was a small bowel transplant recipient, and 11 were allogeneic stem cell transplant recipients. Adenovirus disease was diagnosed at a median of 75 days (range, 15-720 days) after transplantation. All patients received i.v. cidofovir for a median of 21 days (range, 5-90 days) before CMX001 therapy. The median absolute lymphocyte count at CMX001 initiation was 300 cells/μL (range, 7-1500 cells/μL). Eight patients (61.5%) had a ≥ 1 log10 drop in viral load after the first week of therapy. By week 8, 9 patients (69.2%) demonstrated a VR, with a median time to achieve VR of 7 days (range, 3-35 days). The change in absolute lymphocyte count was inversely correlated with the change in log10 viral load only at week 6 (r = -0.74; P = .03). Patients with VR had longer survival than those without VR (median 196 days versus 54.5 days; P = .04). No serious adverse events were attributed to CMX001 during therapy. CMX001 may be a promising therapeutic option for the treatment of severe adenovirus disease in immunocompromised patients.

Treatment Strategies for Invasive Aspergillosis in Neutropenic Patients: Voriconazole or Liposomal Amphotericin-B?

The proportion of patients with cancers who develop invasive fungal infections has increased dramatically over the past few decades. most of these infections are diagnosed in patients with hematological malignancies, mainly in patients with acute myeloid leukemia and those under-going allogeneic hematopoietic stem cell transplantation. for years deoxycolate amphotericin b has been considered the drug of choice for the treatment of invasive as-pergillosis, but it has been outclassed by its lipid formulations and new triazoles (i.e. voriconazole), that produced better response rates; nonetheless recovery from neutropenia remains the most important factor influencing outcome.

A Study on the Sexual Dysfunction of Female Recipients Who Underwent Hematopoietic Stem Cell Transplantation

A Study on the Sexual Dysfunction of Female Recipients Who Underwent Hematopoietic Stem Cell Transplantation