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A randomized phase 2 trial of oral vitamin A for graft-versus-host disease in children and young adults
Khandelwal, P., Langenberg, L., Luebbering, N., Lake, K., Butcher, A., Werling, K., Ramos, K. N., Taggart, C., Choe, H. K., Vasu, S., et al
Blood. 2024
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Editor's Choice
Abstract
Vitamin A plays a key role in the maintenance of gastrointestinal homeostasis and promotes a tolerogenic phenotype in tissue resident macrophages. We conducted a prospective randomized double-blinded placebo-controlled clinical trial in which 80 hematopoietic stem cell transplant recipients were randomized 1:1 to receive pre-transplant high-dose vitamin A or placebo. A single oral dose of vitamin A of 4000 I.U/kg, maximum 250,000 I.U was given prior to conditioning. Primary endpoint was incidence of acute GVHD at day+100. In an intent-to-treat analysis, incidence of acute GVHD was 12.5% in vitamin A arm and 20% in placebo (p=0.5). Incidence of acute GI GVHD was 2.5% in the vitamin A arm (p=0.09) and 12.5% in placebo at day+180. Incidence of chronic GVHD was 5% in the vitamin A arm and 15% in placebo (p=0.02) at 1 year. In an "as treated" analysis, cumulative incidence of acute GI GVHD at day+180 was 0% in vitamin A recipients and 12.5% in placebo (p=0.02) and chronic GVHD incidence 2.7% in the vitamin A recipients and 15% in placebo (p=0.01). The only possibly attributable toxicity was asymptomatic grade 3 hyperbilirubinemia in one vitamin A recipient at day+30, which self-resolved. Absolute CCR9+ CD8+effector memory T-cells, reflecting gut T-cell trafficking, were lower in the vitamin A arm at day+30 after HSCT (p=0.01). Levels of serum amyloid A-1, a vitamin A transport protein with pro-inflammatory effects, were lower in the vitamin A arm. The vitamin A arm had lower IL-6, IL-8, ST2 levels and likely more favorable gut microbiome and short chain fatty acids. Pre-HSCT oral vitamin A is inexpensive, has low toxicity and reduces GVHD (clinicaltrials.gov NCT03202849).
PICO Summary
Population
Children aged 12 months and over with pre transplant vitamin A levels below 75th centile of normal range for age, from a single centre in USA (n=80)
Intervention
A single oral dose of vitamin A of 4000 I.U/kg, maximum 250,000 I.U prior to conditioning pre-transplant (n=40)
Comparison
Placebo (n=40)
Outcome
In an intent-to-treat analysis, incidence of acute GVHD was 12.5% in vitamin A arm and 20% in placebo. Incidence of acute GI GVHD was 2.5% in the vitamin A arm and 12.5% in placebo at day+180. Incidence of chronic GVHD was 5% in the vitamin A arm and 15% in placebo at 1 year. In an "as treated" analysis, cumulative incidence of acute GI GVHD at day+180 was 0% in vitamin A recipients and 12.5% in placebo and chronic GVHD incidence 2.7% in the vitamin A recipients and 15% in placebo. The only possibly attributable toxicity was asymptomatic grade 3 hyperbilirubinemia in one vitamin A recipient at day+30, which self-resolved. Absolute CCR9+ CD8+effector memory T-cells, reflecting gut T-cell trafficking, were lower in the vitamin A arm at day+30 after HSCT. Levels of serum amyloid A-1, a vitamin A transport protein with pro-inflammatory effects, were lower in the vitamin A arm. The vitamin A arm had lower IL-6, IL-8, ST2 levels and likely more favorable gut microbiome and short chain fatty acids.
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Reconstitution of Natural Killer cells after allogeneic hematopoietic stem cell transplantation is facilitated by Huiyang-Guben decoction through activating the Smad7/Stat3 signal pathway
Gao, X., Wang, Q., He, H., Yang, T., Zhang, H., Zhao, J., Yao, X.
Molecular and cellular biochemistry. 2023
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Editor's Choice
Abstract
Natural Killer (NK) cell is the first batch of re-constructed cell populations after allogeneic hematopoietic stem cell transplantation (allo-HSCT), and its delayed reconstitution inevitably causes poor outcome. The traditional Chinese medicine Huiyang-Guben decoction (HYGB) has been clinically used in patients undergoing allo-HSCT, but its effect on NK cell reconstruction is still unclear. 40 patients with allo-HSCT therapy were randomly divided into the control group and the HYGB group, and were given oral administration of normal saline or HYGB for 4 weeks before allo-HSCT, respectively. NK cells were cultured and treated with transforming growth factor β (TGF-β) and HYGB in vitro, and cell viability, cell apoptosis, and the function of NK cells were evaluated. Functional verification experiments were performed by knocking down signal transduction molecule 7 (Smad7) in NK cells before TGF-β and HYGB treatment. Clinical data suggested that HYGB intervention decreased the incidence of acute graft-versus-host disease after allo-HSCT, and increased the proportion of NK cell population. Meanwhile, HYGB improved cell viability, restrained apoptotic cell death, and enhanced cell killing activity of NK cells in patients with allo-HSCT. Notably, we found that HYGB significantly increased the expression level of Smad7 and the phosphorylation level of signal transducer and activator of transcription 3 (Stat3) in NK cells from patients with allo-HSCT. Moreover, HYGB alleviated TGF-β-induced NK cell impairment and re-activated the Smad7/Stat3 signaling in vitro, while silencing Smad7 reversed the protective effect of HYGB on TGF-β-treated NK cells. HYGB promotes NK cell reconstruction and improves NK cell function after allo-HSCT through activating the Smad7/Stat3 signaling pathway.
PICO Summary
Population
People with haematological malignancies underoing first HSCT aged 14-55 years from a single centre in China (n=40)
Intervention
The traditional Chinese medicine Huiyang-Guben decoction four weeks prior to transplant (HYGB, n=20)
Comparison
Normal saline four weeks prior to transplant (control, n=20)
Outcome
Clinical data suggested that HYGB intervention decreased the incidence of acute graft-versus-host disease after allo-HSCT, and increased the proportion of NK cell population. Meanwhile, HYGB improved cell viability, restrained apoptotic cell death, and enhanced cell killing activity of NK cells in patients with allo-HSCT. Notably, we found that HYGB significantly increased the expression level of Smad7 and the phosphorylation level of signal transducer and activator of transcription 3 (Stat3) in NK cells from patients with allo-HSCT. Moreover, HYGB alleviated TGF-β-induced NK cell impairment and re-activated the Smad7/Stat3 signaling in vitro, while silencing Smad7 reversed the protective effect of HYGB on TGF-β-treated NK cells.
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Randomized Double-Blind Phase II Trial of Fecal Microbiota Transplantation Versus Placebo in Allogeneic Hematopoietic Cell Transplantation and AML
Rashidi, A., Ebadi, M., Rehman, T. U., Elhusseini, H., Kazadi, D., Halaweish, H., Khan, M. H., Hoeschen, A., Cao, Q., Luo, X., et al
Journal of clinical oncology : official journal of the American Society of Clinical Oncology. 2023;:Jco2202366
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Editor's Choice
Abstract
PURPOSE Gut microbiota injury in allogeneic hematopoietic cell transplantation (HCT) recipients and patients with AML has been associated with adverse clinical outcomes. Previous studies in these patients have shown improvements in various microbiome indices after fecal microbiota transplantation (FMT). However, whether microbiome improvements translate into improved clinical outcomes remains unclear. We examined this question in a randomized, double-blind, placebo-controlled phase II trial. METHODS Two independent cohorts of allogeneic HCT recipients and patients with AML receiving induction chemotherapy were randomly assigned in a 2:1 ratio to receive standardized oral encapsulated FMT versus placebo upon neutrophil recovery. After each course of antibacterial antibiotics, patients received a study treatment. Up to three treatments were administered within 3 months. The primary end point was 4-month all-cause infection rate. Patients were followed for 9 months. RESULTS In the HCT cohort (74 patients), 4-month infection density was 0.74 and 0.91 events per 100 patient-days in FMT and placebo arms, respectively (infection rate ratio, 0.83; 95% CI, 0.48 to 1.42; P = .49). In the AML cohort (26 patients), 4-month infection density was 0.93 in the FMT arm and 1.25 in the placebo arm, with an infection rate ratio of 0.74 (95% CI, 0.32 to 1.71; P = .48). Unique donor bacterial sequences comprised 25%-30% of the fecal microbiota after FMT. FMT improved postantibiotic recovery of microbiota diversity, restored several depleted obligate anaerobic commensals, and reduced the abundance of expanded genera Enterococcus, Streptococcus, Veillonella, and Dialister. CONCLUSION In allogeneic HCT recipients and patients with AML, third-party FMT was safe and ameliorated intestinal dysbiosis, but did not decrease infections. Novel findings from this trial will inform future development of FMT trials.
PICO Summary
Population
Adults undergoing HSCT (n=74) or suffering from AML (n=26), and enrolled in an RCT in a single centre in USA
Intervention
Standardized oral encapsulated faecal microbiota transplantation (HSCT cohort: n=49)
Comparison
Placebo (HSCT cohort: n=25)
Outcome
In the HCT cohort, 4-month infection density was 0.74 and 0.91 events per 100 patient-days in FMT and placebo arms, respectively (infection rate ratio, 0.83; 95% CI, 0.48 to 1.42). Unique donor bacterial sequences comprised 25%-30% of the fecal microbiota after FMT. FMT improved postantibiotic recovery of microbiota diversity, restored several depleted obligate anaerobic commensals, and reduced the abundance of expanded genera Enterococcus, Streptococcus, Veillonella, and Dialister.
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A phase II trial of netupitant/palonosetron for prevention of chemotherapy-induced nausea/vomiting in patients receiving BEAM prior to hematopoietic cell transplantation
Bubalo, J. S., Radke, J. L., Bensch, K. G., Chen, A. I., Misra, S., Maziarz, R. T.
Journal of oncology pharmacy practice : official publication of the International Society of Oncology Pharmacy Practitioners. 2023;:10781552231173863
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Editor's Choice
Abstract
Objective: The purpose of this study was to investigate the efficacy and safety of netupitant/palonosetron (NEPA) for the prevention of chemotherapy-induced nausea and vomiting (CINV) for hematopoietic cell transplantation (HCT) patients receiving BEAM therapy. Study Design: This phase II, prospective, intention-to-treat, single-center, single-arm study involved 43 adult patients who received NEPA and dexamethasone for the prevention of CINV due to BEAM conditioning chemotherapy. An interim analysis, performed after 13 patients, determined utility versus futility, and supported continuation to full enrollment. Descriptive statistics were used to report complete response (CR), complete protection, incidence of emesis, and administration of rescue agents. A Kaplan-Meier curve depicted time to first emesis and first rescue medication. Patients self-reported levels of daily nausea descriptively via a CINV Questionnaire. Results: By study end, 13 of 43 patients achieved a CR with an average of 10.6 emesis-free days (SD 0.95) over the 11-day observation period, with no emetic events in any patient during the acute/chemotherapy phase. Nausea was well-controlled throughout the acute therapy phase (Day 1-6) and increased during the delayed phase (Day 7-11) with a peak mean level of 2.79/10 at Day 10. Aside from lower grade (≤2), headaches, constipation, and diarrhea were the most widely reported adverse effects. Conclusion: The combination of NEPA and dexamethasone is safe and effective for the prevention of CINV in patients receiving BEAM conditioning therapy prior to HCT. The regimen demonstrated greater effectiveness in the acute phase versus the delayed phase, with low levels of nausea throughout the study period and complete emesis prevention during chemotherapy.
PICO Summary
Population
Adults from a single centre in USA undergoing BEAM conditioning chemotherapy before transplant (n=43)
Intervention
Netupitant/palonosetron (NEPA) and dexamethasone for the prevention of chemotherapy induced nausea and vomiting (CINV)
Comparison
None
Outcome
By study end, 13 of 43 patients achieved a CR with an average of 10.6 emesis-free days (SD 0.95) over the 11-day observation period, with no emetic events in any patient during the acute/chemotherapy phase. Nausea was well-controlled throughout the acute therapy phase (Day 1-6) and increased during the delayed phase (Day 7-11) with a peak mean level of 2.79/10 at Day 10. Aside from lower grade (≤2), headaches, constipation, and diarrhea were the most widely reported adverse effects.
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Frontline-matched sibling donor transplant of aplastic anemia patients using primed versus steady-state bone marrow grafts
El Fakih, R., Alfraih, F., Alhayli, S., Ahmed, S. O., Shaheen, M., Chaudhri, N., Alsharif, F., Hanbali, A., Alshaibani, A., Alotaibi, A. S., et al
Annals of hematology. 2021
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Editor's Choice
Abstract
Priming donors with G-CSF before BM harvest is reported to improve engraftment and GvHD in recipients. These effects are highly desirable when transplanting patients with non-neoplastic hematologic diseases, particularly AA patients. Here we retrospectively report the outcomes of 39 AA patients receiving a primed BM graft from MSD to 43 patients receiving a steady-state BM graft from MSD, otherwise transplanted using a uniform transplant platform. The graft had higher TNC and CD34 cell concentrations in the primed group (p?0.001), and that was reflected in higher TNC and CD34 doses per kilogram of recipient in the primed group (p?=?0.004 and 0.03, respectively). The OS for primed BM graft recipients was 97.4% and 78.9% for the steady-state BM graft recipients, p-value?=?0.01. The cumulative incidence of death without GF was 2.6% in the primed group and 16.3% in the steady-state group, p-value?=?0.03. There was no difference in GvHD incidence between the two groups. We confirm that priming improved the TNC and CD34 graft concentration and cell dose; this evidence along with other reported studies constitute reasonable evidence to prove that BM priming improve engraftment. We observed no increase in GvHD using primed BM graft.
PICO Summary
Population
Patients with aplastic anaemia, undergoing matched sibling donor transplant (n=82)
Intervention
Bone marrow (BM) graft primed with G-CSF before bone marrow harvest (n=39)
Comparison
Steady state BM graft (n=43)
Outcome
The graft had higher TNC and CD34 cell concentrations in the primed group, and that was reflected in higher TNC and CD34 doses per kilogram of recipient in the primed group. The overall survival for primed BM graft recipients was 97.4% and 78.9% for the steady-state BM graft recipients. The cumulative incidence of death without graft failure was 2.6% in the primed group and 16.3% in the steady-state group. There was no difference in GvHD incidence between the two groups.
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The outcome and complications of total parenteral nutrition in pediatric hematopoietic stem cell transplantation
Alsalamah, S., Alramyan, R., Alakel, R., Altheyeb, F., Alrashed, R., Masuadi, E., Alyousif, G., Bin Sunaid, F. F., Alsultan, A., Essa, M. F.
Pediatric transplantation. 2021;:e14198
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Editor's Choice
Abstract
BACKGROUND Nutritional support posthematopoietic stem cell transplantation (HSCT) with total parenteral nutrition (TPN) or nasogastric tube feeding (NGT) in pediatric patients is associated with benefits and risks. METHODS We retrospectively reviewed the indication of TPN use in our pediatric HSCT patients and its impact on survival and possible related complications. RESULTS A total of 228 HSCTs were performed during the study period. TPN was used in 144 patients (63.2%) for a median of 14 days, while 8.8% had NGT feeding and 28% were able to tolerate oral feeding. Severe mucositis was seen in 104 TPN patients (72.2%) in comparison with 22 patients (26.2%) who were on Enteral Nutrition (EN) (p = <.001). Sinusoidal obstruction syndrome (SOS) was seen in 19 (13.2%) patients who had TPN compared to none in the patients who received EN (p = .001). The majority of patients who had SOS received myeloablative conditioning (MAC) therapy for hemoglobinopathy. Acute graft-versus-host disease (aGVHD) was seen in 24.8% of TPN patients and 9.1% of non-TPN patients (p = .01). However, there were no statistically significant differences in chronic GVHD, bacteremia, and patients' survival between both groups. CONCLUSIONS TPN is commonly used after pediatric HSCT in cases of severe mucositis. NGT feeding was found to be the least used nutritional support method. SOS and aGVHD were associated more frequently in TPN patients compared to EN patients. This suggests the possible disadvantages of TPN and importance of SOS preventative measures in high-risk patients.
PICO Summary
Population
Children under the age of 14 years who underwent HSCT at a single centre in Saudi Arabia (n=228)
Intervention
Total parenteral nutrition (TPN, n=144)
Comparison
Enteral nutrition (EN, n=84): either nasogastric tube feeding (n=20) or oral feeding (n=64)
Outcome
Severe mucositis was seen in 104 TPN patients (72.2%) in comparison with 22 patients (26.2%) who were on Enteral Nutrition (EN). Sinusoidal obstruction syndrome (SOS) was seen in 19 (13.2%) patients who had TPN compared to none in the patients who received EN. The majority of patients who had SOS received myeloablative conditioning therapy for hemoglobinopathy. Acute graft-versus-host disease (aGVHD) was seen in 24.8% of TPN patients and 9.1% of non-TPN patients. However, there were no statistically significant differences in chronic GVHD, bacteremia, and patients' survival between both groups.
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Geriatric nutritional risk index as a useful prognostic factor in second allogeneic hematopoietic stem cell transplantation
Kaito, S., Wada, A., Adachi, H., Konuma, R., Kishida, Y., Nagata, A., Konishi, T., Yamada, Y., Kumagai, T., Yoshifuji, K., et al
Annals of hematology. 2020
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Editor's Choice
Abstract
Second allogeneic hematopoietic stem cell transplantation (allo-HSCT) has a low survival outcome and a high non-relapse mortality (NRM) rate which is a major obstacle to this treatment. We hypothesized that the status of malnourishment after first allo-HSCT as represented by the geriatric nutritional risk index (GNRI) could be used as a prognostic factor to determine the outcomes of second allo-HSCT. A total of 108 patients with a median age of 42 (range, 17-69) years, who received second allo-HSCT for disease recurrence after first allo-HSCT from our institution, were included in this study. Low GNRI had a significant impact on NRM at 2 years after second allo-HSCT: 56.9% in patients with GNRI ≤ 92 compared with 27.5% in patients with GNRI > 92 (P = 0.002). In multivariate analysis, GNRI of ≤ 92 was the only significant factor for NRM (hazard ratio [HR] 2.29, 95% confidence interval [CI] 1.15-4.56, P = 0.018). High-risk disease status at second allo-HSCT (HR 2.74, 95% CI 1.46-5.14, P = 0.002) and GNRI of ≤ 92 (HR 1.70, 95% CI 1.02-2.82, P = 0.042) were identified as significant factors for overall survival (OS). A score of 1 was assigned to each factor, and the OS rate at 2 years after second allo-HSCT decreased according to the score: 53.0% in patients with score 0, 32.3% with score 1, and 2.5% with score 2 (P < 0.001). In conclusion, GNRI could be a useful predictor for the outcomes of second allo-HSCT. A prospective study in other cohorts is warranted to validate the findings of our study.
PICO Summary
Population
Patients receiving a second allo-HSCT for disease recurrence after first allo-HSCT (n=108)
Intervention
Patients with low score on the geriatric nutritional risk index (GNRI </= 92) (n=60)
Comparison
Patients with a high score on the geriatric nutritional risk index (GNRI >92) (n=48)
Outcome
Low GNRI had a significant impact on NRM at 2 years after second allo-HSCT: 56.9% in patients with GNRI </= 92 compared with 27.5% in patients with GNRI > 92. In multivariate analysis, GNRI of </= 92 was the only significant factor for NRM (hazard ratio [HR] 2.29). High-risk disease status at second allo-HSCT (HR 2.74) and GNRI of </= 92 (HR 1.70) were identified as significant factors for overall survival (OS). A score of 1 was assigned to each factor, and the OS rate at 2 years after second allo-HSCT decreased according to the score: 53.0% in patients with score 0, 32.3% with score 1, and 2.5% with score 2.
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Impact of a Replacement Algorithm for Vitamin D Deficiency in Adult Hematopoietic Stem Cell Transplant Patients
Kenny, S. A., Collum, K., Featherstone, C. A., Farooki, A., Jakubowski, A.
Journal of the advanced practitioner in oncology. 2019;10(2):109-118
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Editor's Choice
Abstract
Adults undergoing hematopoietic stem cell transplant (HSCT) are at risk for vitamin D deficiency. After HSCT, exposure to sunlight is restricted, and patients may experience poor nutrition and malabsorption from HSCT-related side effects. Vitamin D affects bone health and immunologic processes. The aim of this project is to establish a process for monitoring and treating vitamin D deficiency and to evaluate if therapeutic vitamin D levels are attainable posttransplant using an HSCT vitamin D replacement algorithm. A multidisciplinary group led by advanced practice providers established a workflow for monitoring and supplementing vitamin D and created an HSCT vitamin D replacement guideline. The medical records of 144 adult HSCT patients were reviewed, and the records of another 72 patients were reviewed a year later. Historical baseline data before the intervention found that 81% of patients were vitamin D deficient and 30% received supplementation. Postintervention and at 1-year follow-up, 76% and 65% of patients were vitamin D deficient before transplant and 97.1% and 100%, respectively, received supplementation for vitamin D deficiency. Post-HSCT compliance with monitoring demonstrated that approximately 91% of patients had a vitamin D level checked within 6 months of transplant. After implementation of the algorithm, there was a statistically significant difference (p < .001) between deficient vitamin D levels pretransplant (72.9%) and posttransplant (26.4%). Results demonstrate sustained compliance over a 2-year period with monitoring and supplementation of vitamin D pre- and peritransplant. Aggressive vitamin D repletion posttransplant decreased the incidence of vitamin D deficiency in HSCT patients. Further study is needed to investigate the long-term effects of vitamin D repletion on posttransplant complications.
PICO Summary
Population
Adults undergoing haematopoietic stem cell transplant (n=346)
Intervention
Vitamin D replacement guideline (n=162)
Comparison
Historical cohort prior to guideline implementation (n=184)
Outcome
Postintervention and at 1-year follow-up, 76% and 65% of patients were vitamin D deficient before transplant and 97.1% and 100%, respectively, received supplementation for vitamin D deficiency. Post-HSCT compliance with monitoring demonstrated that approximately 91% of patients had a vitamin D level checked within 6 months of transplant. After implementation of the algorithm, there was a statistically significant difference between deficient vitamin D levels pretransplant (72.9%) and posttransplant (26.4%). Results demonstrate sustained compliance over a 2-year period with monitoring and supplementation of vitamin D pre- and peritransplant. Aggressive vitamin D repletion posttransplant decreased the incidence of vitamin D deficiency in HSCT patients.
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Protective environment for hematopoietic cell transplant (HSCT) recipients: The Infectious Diseases Working Party EBMT analysis of global recommendations on health-care facilities
Styczynski, J., Tridello, G., Donnelly, J. P., Iacobelli, S., Hoek, J., Mikulska, M., Aljurf, M., Gil, L., Cesaro, S.
Bone marrow transplantation. 2018
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Editor's Choice
Abstract
International guidelines on protective environment for HSCT recipients proposed a set of 10 global recommendations in 2009 on protective environment (GRPE) concerning hospital room design and ventilation. The EBMT Infectious Diseases Working Party undertook a survey on the status on protective environment for HSCT recipients with the aim of surveying current practices and their agreement with GRPE recommendations. The questionnaire consisted of 37 questions divided into 5 sections about filtration, air changes, maintenance, and the protective environment in rooms and the surrounding unit. Overall, 177 centres (response rate 33%) from 36 countries responded, indicating that 99.4% of patient rooms were equipped with HEPA filters, but only 48.6% of the centre's staff were aware of, and could confirm, regular replacement of filters based on manufacturers' recommendations. Well-sealed rooms were used in terms of windows (70.6%), ceilings (35%), and plumbing pipes (51.4%). The sensor monitors in the patient room used to determine when the HEPA filters require changing were installed only in 18.1% of centres. Only 1 centre fulfilled all 10 GRPE recommendations, while 62 centres fulfilled the 3 level "A" recommendations. In conclusion, HEPA-filtered rooms are available in almost all centres, while fewer centres fulfilled other requirements. Knowledge on the details and maintenance of protective environments in the HSCT setting was inadequate, reflecting a lack of communication between the health personnel involved, hospital infection control and the hospital maintenance services.
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Randomized controlled trial of individualized treatment summary and survivorship care plans for hematopoietic cell transplantation survivors
Majhail, N. S., Murphy, E., Laud, P., Preussler, J. M., Denzen, E. M., Abetti, B., Adams, A., Besser, R., Burns, L. J., Cerny, J., et al
Haematologica. 2018
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Editor's Choice
Abstract
Survivorship care plans may facilitate long-term care for cancer survivors, but their effectiveness has not been established in hematopoietic cell transplantation recipients. We evaluated the impact of individualized survivorship care plans on patient-reported outcomes among transplant survivors. Adult (≥18 years at transplant) survivors who were 1-5 years post-transplantation, proficient in English, and without relapse or secondary cancers were eligible for this multicenter randomized trial. Care plans were developed based on risk-factors and treatment exposures using patient data routinely submitted by transplant centers to the Center for International Blood and Marrow Transplant Research and published guidelines for long-term follow-up of transplant survivors. Phone surveys assessing patient-reported outcomes were conducted at baseline and 6-months. Primary endpoint was confidence in survivorship information, and secondary endpoints included cancer and treatment distress, knowledge of transplant exposures, health care utilization and health-related quality of life. Of 495 patients enrolled, 458 completed a baseline survey and were randomized (care plan=231, standard care=227); 200 (87%) and 199 (88%) completed the 6-month assessments, respectively. Patient characteristics were balanced in the two arms. Participants on care plan arm reported significantly lower distress scores at 6-months and an increase in the Mental Component Summary quality of life score assessed by the SF12 instrument. No effect was observed on the endpoint of confidence in survivorship information or other secondary outcomes. Provision of individualized survivorship care plans generated using registry data was associated with reduced distress and improved mental domain of quality of life among 1-5 year hematopoietic cell transplantation survivors. (clinicaltrials.gov NCT02200133).
PICO Summary
Population
495 adult survivors of haematopoietic stem cell transplantation 1-5 years post-transplant.
Intervention
Individualised survivorship care plans (N= 231)
Comparison
Standard Care (N=227)
Outcome
Participants on care plan arm reported significantly lower distress scores at 6-months and an increase in the Mental Component Summary quality of life score. No effect was observed on the endpoint of confidence in survivorship information or other secondary outcomes.