Blinatumomab Prior to CAR-T Cell Therapy-A Treatment Option Worth Consideration for High Disease Burden.

The optimal bridging therapy before CAR-T cell infusion in pediatric relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r BCP-ALL) still remains an open question. The administration of blinatumomab prior to CAR-T therapy is controversial since a potential loss of CD19+ target cells may negatively impact the activation, persistence, and, as a consequence, the efficacy of subsequently used CAR-T cells. Here, we report a single-center experience in seven children with chemorefractory BCP-ALL treated with blinatumomab before CAR-T cell therapy either to reduce disease burden before apheresis (six patients) or as a bridging therapy (two patients). All patients responded to blinatumomab except one. At the time of CAR-T cell infusion, all patients were in cytological complete remission (CR). Four patients had low positive PCR-MRD, and the remaining three were MRD-negative. All patients remained in CR at day +28 after CAR-T infusion, and six out of seven patients were MRD-negative. With a median follow-up of 497 days, four patients remain in CR and MRD-negative. Three children relapsed with CD19 negative disease: two of them died, and one, who previously did not respond to blinatumomab, was successfully rescued by stem cell transplant. To conclude, blinatumomab can effectively lower disease burden with fewer side effects than standard chemotherapeutics. Therefore, it may be a valid option for patients with high-disease burden prior to CAR-T cell therapy without clear evidence of compromising efficacy; however, further investigations are necessary.

The Impact of High CMV Viral Load and Refractory CMV Infection on Pediatric HSCT Recipients with Underlying Non-Malignant Disorder.

Hematopoietic stem cell transplantation (HSCT) is a curative therapy for an increasing number of nonmalignant indications. Its use is restricted by severe transplant-related complications, including CMV infection; despite various prophylactic and therapeutic strategies, CMV reactivation has remarkable morbidity and mortality. The analysis included 94 children with nonmalignant disorder who underwent allogeneic HSCT in the Department of Pediatric Hematology, Oncology, and Bone Marrow Transplantation in Wroclaw during years 2016-2020. Twenty-seven (29%) children presented with CMV infection, including ten (10/27; 37%) with high level CMV viremia (10,000 copies/mL). Six patients experienced subsequent CMV reactivation. The first-line ganciclovir-based (GCV) treatment was insufficient in 40% (11/27) of children. Overall survival (OS) was significantly lower in children with high CMV viremia compared to those with low levels/no CMV [1yrOS High CMV = 0.80 (95% CI 0.41-0.95) vs. 1yrOS others = 0.96 (95% CI 0.89-0.99)]. Similarly, patients with resistant and recurrent infections had greater risk of death. CMV reactivation at any level relevantly prolonged the hospital stay. CMV reactivation with high viremia load and resistant/recurrent CMV infections lead to a significant decrease in OS in children with nonmalignant disorders treated with HSCT. Our data proves there is an urgent need to introduce an effective anti-CMV prophylaxis in this cohort of patients.

Successful Salvage Haploidentical Bone Marrow Transplantation in a Child With Hemophagocytic Lymphohistiocytosis, When the Previously Matched Unrelated Donor Tested Positive for SARS-CoV-2 on the Day of Stem Cells Collection.

The coronavirus disease 2019 pandemic has made us adjust our standards and cope with unpredictable circumstances affecting the whole world, including the medical field. A 2-year-old boy diagnosed with X-linked lymphoproliferative disease type 2 with concomitant positive polymerase chain reaction test for Epstein-Barr virus-DNA was admitted to our transplant ward. His treatment scheme had to be modified at the last moment because of a donor disqualification due to a positive polymerase chain reaction result for severe acute respiratory syndrome coronavirus 2 just before the apheresis. We decided to perform salvage haploidentical bone marrow transplant from the patient's mother because it was the only possible option. Now, in a 5-month observation period after the hematopoietic stem cell transplantation, our patient is in good general condition. His case convinced us to redirect our approach to transplant procedure preparation. Following the European Group of Blood and Marrow Transplantation recommendations, we use cryopreserved apheresis materials to ensure the availability of stem cell products before the start of a conditioning regimen.

New methods of differentiation between primary and secondary hypertension in a pediatric population: A single-center experience.

BACKGROUND: Kidney diseases are the main causative factors of secondary hypertension (HTN) in children. Although primary HTN is less common in the pediatric population, its increasing prevalence, especially among teenagers, makes early diagnosis an emerging issue. OBJECTIVES: To analyze the potential differences between primary HTN and HTN secondary to renal diseases, in order to tailor diagnostic procedures to pediatric patients with suspicion of HTN. MATERIAL AND METHODS: A retrospective evaluation was performed of medical records of 168 children (aged from 1 month to 18 years) diagnosed with arterial HTN in the Pediatric Nephrology Department of Wroclaw Medical University (Poland). The comparative analysis concerned demographics, causes of HTN, clinical picture, laboratory tests, and parameters of ambulatory blood pressure monitoring (ABPM). RESULTS: Out of 168 children, 47% were diagnosed with primary HTN and 53% with secondary renal HTN. The patients with primary HTN were significantly older than those with HTN secondary to renal disease. Among the children with primary HTN, 26% were overweight and 42% were obese; among those with renal HTN, the proportions were 16% and 19%, respectively. The patients with primary HTN had significantly higher body mass index (BMI) percentiles and z-scores, and tended toward higher pulse pressure (PP) values. In the group with secondary HTN, ABPM parameters of diastolic blood pressure (DBP) and total cholesterol were significantly elevated. The BMI z-scores correlated positively with PP in the whole group. CONCLUSIONS: As expected, HTN secondary to renal disease prevails in younger children, but primary HTN has become an emerging issue in teenagers. The diagnostics of HTN secondary to kidney disease have revealed risk factors worsening the prognosis, including higher values of cholesterol or of parameters connected with DBP. Primary HTN risk factors include obesity and a tendency towards higher PP values.

Social participation of patients with multiple sclerosis.

BACKGROUND: The effect multiple sclerosis (MS) has on the social functioning and integration of patients has been recently considered as an important factor of the disease. OBJECTIVES: To assess social participation of MS patients with regard to demographic and disease-related variables. MATERIAL AND METHODS: The study comprised 201 MS patients: 140 women, 61 men, aged 24-69 years. The World Health Organization Disability Assessment Schedule (WHODAS 2.0) was applied to assess the aspects of social functioning and the Beck Depression Inventory (BDI) was applied to evaluate the level of depression. Disease duration, degree of disability in Expanded Disability Status Scale (EDSS), most disabling symptoms and type of treatment were determined. WHODAS 2.0 scores (total and within particular domains) and their relationships with age, gender, disease-related variables and level of depression were analyzed. RESULTS: The results of WHODAS 2.0 for 27.4% of patients exceeded the 90th percentile compared to the population norms (with the highest scores for "getting around" and "participation in society" domains). The results of BDI and WHODAS 2.0 were strongly correlated (p < 0.001; ß = 0.73) and mobility impairment was related to both of them (p < 0.001; ß = -0.12 and 0.25, respectively). Other disabling symptoms were associated with scores in domains "understanding and communicating", "getting around" and "participation in society". CONCLUSIONS: Social participation of the MS patients is affected by the impact of disease and associated with depression. Particular symptoms of neurological deficit (motor and visual impairment, fatigue) influence social functioning more than general disease-related variables.

Early Complications in Patients Undergoing Elective Open Surgery for Infrarenal Aortic Aneurysms.

OBJECTIVE: To assess the type and frequency of early postoperative complications in patients after open surgical infrarenal aneurysms repair and determination of their risk factors. STUDY DESIGN: An observational study Place and Duration of Study: The 4th Military Teaching Hospital in Wroclaw, Poland, from January 2012 to December 2016. METHODOLOGY: Analyses of medical records of 205 patients treated for infrarenal aneurysm with open surgery. Patients with clinical and radiological features of aneurysm rupture were excluded. Complications and early deaths (<30 days) were recorded. RESULTS: Men represented 170 (83%) of patients, the average age was 67.9 ± 6.68 years. Aneurysm diameter <60 mm occurred in 107 (52.2%) of patients, 60-80 mm in 76 (37.1%), and >80 mm in 22 (10.7%). Aortic prostheses (tube grafts) were implanted in 132 (64.4%) of patients and aorto-bifemoral (bifurcated) grafts in 73 (35.6%). The most common postoperative complications were: reoperation necessity (n=23; 11.2%), respiratory failure (n=21; 10.2%), respiratory tract infection (n=14; 6.8%), circulatory failure (n=14; 6.8%), lower limb ischemia (n=13; 6.3%), kidney failure (n=12; 5.9%), death (n=9; 4.4%), and surgical wound infection (n=9; 4.4%). Statistical analysis indicated intraoperative blood loss, duration of surgery, a larger aneurysm diameter, and diabetes as risk factors for early complications. CONCLUSION: Complications were the same as reported from other centres. There is a need to shorten surgery duration and minimise blood loss, as these modifiable factors were the predictors of early complications.

Oncologists and Breaking Bad News-From the Informed Patients' Point of View. The Evaluation of the SPIKES Protocol Implementation.

The way that bad news is disclosed to a cancer patient has a crucial impact on physician-patient cooperation and trust. Consensus-based guidelines provide widely accepted tools for disclosing unfavorable information. In oncology, the most popular one is called the SPIKES protocol. A 17-question survey was administered to a group of 226 patients with cancer (mean age 59.6 years) in order to determine a level of SPIKES implementation during first cancer disclosure. In our assessment, the patients felt that the highest compliance with the SPIKES protocol was with Setting up (70.6%), Knowledge (72.8%), and Emotions (75.3%). The lowest was with the Perception (27.7%), Invitation (30.4%), and Strategy & Summary (56.9%) parts. There could be improvement with each aspect of the protocol, but especially in Perception, Invitation, and Strategy & Summary. The latter is really important and must be done better. Older patients felt the doctors' language was more comprehensible (r = 0.17; p = 0.011). Patients' satisfaction of their knowledge about the disease and follow-up, regarded as an endpoint, was insufficient. Privacy was important in improving results (p < 0.01). In practice, the SPIKES protocol is implemented in a satisfactory standard, but it can be improved in each area, especially in Perception, Invitation, and Summary. It is suggested that more training should be done in undergraduate and graduate medical education and the effectiveness of the disclosure continue to be evaluated and improved.

Comparison of the Incidence of Complications and Secondary Surgical Interventions Necessary in Patients with Chronic Lower Limb Ischemia Treated by Both Open and Endovascular Surgeries.

BACKGROUND: Peripheral arterial disease (PAD) affects 3%-10% of the population before the age of 70 years and 15%-20% after that age.The aim of the study was to compare the incidence of complications and secondary interventions in patients who underwent each type of treatment. METHODS: We analyzed 734 medical records of the Department of Surgery at the 4th Military Teaching Hospital in Wroclaw, In total, 394 were operated on with open surgery; an endarterectomy (59.39%), a vascular prosthesis implantation (31.01%), or both of these techniques (6.6%), and 340 patients had angioplasty with (50.59%) or without stenting (49.41%). RESULTS: There were no statistically significant differences in the incidence of corresponding complications. The exception was the infection of the wound; significantly fewer were reported in the case of endovascular procedures (p = 0.0087). There were 12 occasions (3.53%) during endovascular surgeries when intraoperative conversion or re-operation using the open method occurred. In the case of open surgery, the mean hospital stay was 7.77 days (median: 8, mode: 8), while for endovascular management it was equal to 4.68 days (median: 4, mode: 3), p <0.0001. CONCLUSION: The endovascular method results in a similar re-operation rate and number of complications as open surgery.