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A 16-year-old female with no prior comorbidities came with chief complaints of fever and headache for 4 days associated with altered sensorium for the past 15 days in the form of decreased responsiveness. On general physical examination, Glasgow Coma Scale score was 6 with E1V1M4. Vitals were stable. Central nervous system examination revealed that bilateral pupils were sluggishly reactive to light. All extremities were rigid. A provisional diagnosis of viral encephalitis was made and was empirically started on acyclovir, doxycycline, ceftriaxone and vancomycin. Cerebrospinal fluid (CSF) showed lymphocytic pleocytosis, elevated proteins and normal glucose. CSF pan-neurotropic virus panel was negative. Serological testing for immunoglobulin M (IgM) against Japanese encephalitis virus positive. Magnetic resonance imaging of the brain showed symmetrical T2 hyperintense areas in the bilateral basal ganglia, thalamus and splenium of corpus callosum. The patient was started on oral levodopa plus carbidopa; sensorium became better and Parkinsonian features improved gradually.

A 19-year-old male from a rural background with no prior co-morbidities came with chief complaints of fever for 4 days, associated with chills, rigors, myalgias and high coloured urine. On general examination, pallor was present. Vitals and systemic examination were normal. Routine blood investigations were sent, and two sets of blood cultures were sent. The patient was empirically started on ceftriaxone. Complete urine examination showed >20 red blood cells (RBCs)/high-power field (HPF) with four to five RBC casts/HPF. Initial creatine phosphokinase was 3216 U/L. The patient's fever spikes continued, and antibiotic was changed to piperacillin-tazobactam after 48 h and then to meropenem. Fever spikes continued. The patient developed pancytopenia and acute respiratory distress syndrome. Final blood culture report grew Brucella melitensis. The patient was started on streptomycin and doxycycline. The patient improved clinically and discharged in clinically stable condition.

A 48-year-old male, farmer by occupation with no prior comorbidities came with chief complaints of fever since 5 months, associated with loss of weight and loss of appetite. On general examination, pallor and clubbing were present. Blood pressures were 120/20 mmHg in both upper limbs and 200/60 mmHg in both lower limbs. Cardiovascular examination, on auscultation there was pansystolic murmur in mitral area radiating to axilla, holodiastolic murmur in aortic area. All routine blood investigations were done. Echocardiogram was suggestive of infective endocarditis of aortic valve with multiple thickened vegetations over aortic valve. Blood cultures grew Enterococcus faecium in two sets. The patient was treated with antibiotics according to culture sensitivity report. Patient improved clinically and was discharged.

The severe phenotype of leukocyte adhesion deficiency is a rare, congenital disorder of leukocyte function that is usually fatal in the first few years of life. Allogeneic hematopoietic stem cell transplantation currently offers the only curative approach for this disease. We describe the first successful matched unrelated donor bone marrow transplant in an infant with leukocyte adhesion deficiency. [ABSTRACT FROM AUTHOR]

Renal malakoplakia is reported in an 11 year old girl with myelomeningocele and associated neuropathic bladder. She is the first reported child to have survived bilateral renal malakoplakia with full recovery of renal function after medical management. [ABSTRACT FROM AUTHOR]

Granulocyte colony-stimulating factor (G-CSF) used after hematopoietic stem cell transplantation (HSCT) can enhance neutrophil recovery in patients rendered neutropenic by the preparative regimen. G-CSF is contraindicated in patients with sickle cell disease (SCD), because life-threatening complications can ensue in the presence of sickle vasculopathy. The safety profile of G-CSF after HSCT for SCD has not been described, however. We report clinical outcomes in the first 100 days post-HSCT in 62 patients supported with G-CSF until neutrophil recovery on a clinical trial of reduced- intensity conditioning HSCT for SCD. The patients received G-CSF for a median of 9 days (range, 5 to 33 days) post-transplantation from the best available stem cell source. Preparation for transplantation included a target hemoglobin S level of ≤45%. Neutrophil engraftment (absolute neutrophil count >0.5 × 10 3 /mL) was achieved at a median of 13 days (range, 10 to 34 days), and platelet engraftment (>50 × 10 3 /mL) was achieved at a median of 19 days (range, 12 to 71 days). The median duration of inpatient hospitalization following stem cell infusion (day 0) was 21.5 days (range, 11 to 33 days). No patient developed SCD-related complications following G-CSF use. The most common organ toxicities encountered between G-CSF initiation (on day +7) and day +100 were anorexia (n = 14), hypertension (n = 11), and electrolyte imbalance requiring correction (n = 9). Central nervous system-related events were noted in 5 patients, all of whom had preexisting cerebral vasculopathy/moyamoya disease, attributed to reversible posterior leukoencephalopathy syndrome in the presence of calcineurin inhibitor therapy and hypertension. We conclude that G-CSF does not adversely impact SCD HSCT recipients and can be safely used post-transplantation to enhance neutrophil recovery., (Copyright © 2021 The American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc. All rights reserved.)

Hematopoietic cell transplantation (HCT) is a highly complex procedure that requires a dedicated multidisciplinary team to optimize its safety. In addition, institutions may have different needs regarding indications based on regional disease prevalence or may have an interest in developing specialized services. Yet, structured recommendations are not commonly available. Here, the Transplant Center and Recipient Issues Standing Committee for the Worldwide Network for Blood and Marrow Transplantation (WBMT) organized a structured review of all pertinent elements to establish a transplant program. First, we solicited components from committee members and grouped them in domains (infrastructure, staff, cell processing laboratory, blood banking, laboratory, radiology, pharmacy, HLA testing, ancillary services and quality). Subsequently, reviewers scored all elements on a 7-point scale, from an absolute requirement (score of 1) to not required (score of 7). An independent group of five experienced transplant physicians reviewed the rankings. Minimum requirements to establish any HCT program were identified among elements with mean score of ≤2.0, and specific elements for allogeneic and autologous HCT were identified. Mean scores >2.0-4.0 were classified as preferred recommendation, and mean scores of >4.0 to ≤7.0 were considered ideal recommendations for advanced and complex types of transplantation. This structured set of recommendations guides the prioritization of minimum requirements to establish a transplant program and to set the path for expansion and further development., Competing Interests: Declaration of Competing Interest None., (Copyright © 2019 The Authors. Published by Elsevier Ltd.. All rights reserved.)

We examined the risk of subsequent neoplasms (SNs) and late mortality in children and adolescents undergoing allogeneic hematopoietic cell transplantation (HCT) for nonmalignant diseases (NMDs). We included 6028 patients (median age, 6 years; interquartile range, 1-11; range, <1 to 20) from the Center for International Blood and Marrow Transplant Research (1995-2012) registry. Standardized mortality ratios (SMRs) in 2-year survivors and standardized incidence ratios (SIRs) were calculated to compare mortality and SN rates with expected rates in the general population. Median follow-up of survivors was 7.8 years. Diagnoses included severe aplastic anemia (SAA; 24%), Fanconi anemia (FA; 10%), other marrow failure (6%), hemoglobinopathy (15%), immunodeficiency (23%), and metabolic/leukodystrophy syndrome (22%). Ten-year survival was 93% (95% confidence interval [95% CI], 92% to 94%; SMR, 4.2; 95% CI, 3.7-4.8). Seventy-one patients developed SNs (1.2%). Incidence was highest in FA (5.5%), SAA (1.1%), and other marrow failure syndromes (1.7%); for other NMDs, incidence was <1%. Hematologic (27%), oropharyngeal (25%), and skin cancers (13%) were most common. Leukemia risk was highest in the first 5 years posttransplantation; oropharyngeal, skin, liver, and thyroid tumors primarily occurred after 5 years. Despite a low number of SNs, patients had an 11-fold increased SN risk (SIR, 11; 95% CI, 8.9-13.9) compared with the general population. We report excellent long-term survival and low SN incidence in an international cohort of children undergoing HCT for NMDs. The risk of SN development was highest in patients with FA and marrow failure syndromes, highlighting the need for long-term posttransplantation surveillance in this population.

Hematopoietic cell transplantation (HCT) is a highly complex procedure that requires a dedicated multidisciplinary team to optimize safety. In addition, institutions may have different needs regarding indications based on regional disease prevalence or may have an interest in developing specialized services. Structured recommendations are not commonly available, however. The Transplant Center and Recipient Issues Standing Committee of the Worldwide Network for Blood and Marrow Transplantation (WBMT) organized a structured review of all pertinent elements for establishing a transplantation program. First, we solicited components from committee members and grouped them into domains (infrastructure, staff, cell processing laboratory, blood banking, laboratory, radiology, pharmacy, HLA testing, ancillary services, and quality). Subsequently, reviewers scored each element on a 7-point scale, ranging from an absolute requirement (score of 1) to not required (score of 7). An independent group of 5 experienced transplantation physicians reviewed the rankings. The minimum requirements for establishing any HCT program were identified among elements with mean score of ≤2.0, and specific elements for allogeneic and autologous HCT were identified. Mean scores of >2.0 to 4.0 were classified as preferred recommendation, and mean scores of >4.0 to ≤ 7.0 were considered ideal recommendations for advanced and complex types of transplantation. This structured set of recommendations guides the prioritization of minimum requirements to establish a transplantation program and set the stage for expansion and further development., (Copyright © 2019 American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc. All rights reserved.)

Survivorship Care Plans (SCPs) 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 SCPs 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. SCPs 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 at 6 months. The primary end point was confidence in survivorship information, and secondary end points 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. Patients' characteristics were similar in the two arms. Participants on the 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 Short Form 12 (SF-12) instrument. No effect was observed on the end point of confidence in survivorship information or other secondary outcomes. Provision of individualized SCPs 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. Trial registered at clinicaltrials.gov 02200133 ., (Copyright© 2019 Ferrata Storti Foundation.)

Stroke and cognitive decline are hallmarks of sickle cell disease (SCD). The natural history of SCD predicts progressive loss of 1 IQ point per year attributable to disease-related pathology. Hematopoietic cell transplantation (HCT) is curative by reverting to donor-derived erythropoiesis, but evidence that HCT can positively influence disease-induced cognitive decline is lacking. The Sickle Cell Unrelated Transplant Trial prospectively evaluated cognition and brain magnetic resonance imaging (MRI) findings at 2 years after reduced-intensity conditioning followed by unrelated donor HCT. Thirteen study participants completed pre-HCT and post-HCT assessments of intelligence. The mean age of participants was 12.5 ± 3.3 years (range, 6.7 to 17.4 years). Eleven of the 13 recipients completed imaging studies at baseline and post-HCT. Seven had overt stroke pre-HCT, and 1 had an elevated transcranial Doppler velocity with abnormal MRI. The mean Full-Scale IQ was stable: 90.9 ± 13 at baseline and 91.2 ± 13 post-HCT. The mean Performance IQ was 89.9 ± 13 at baseline versus 90.9 ± 13 post-HCT, and mean Verbal IQ was 93.4 ± 13 at baseline versus 93.2 ± 13 post-HCT, respectively. Six recipients had stable MRI; 2 showed resolution of all areas of infarction. Three had additional infarcts post-HCT noted at the 2-year time point. This is the first report describing stabilization of IQ and central nervous system outcomes after unrelated donor HCT despite previous central nervous system morbidity and post-HCT posterior reversible encephalopathy syndrome. These preliminary results post-HCT suggest that HCT may stabilize the cognitive decline of SCD and should continue to be followed over the long term., (Copyright © 2019 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.)