Background
Despite the improvements in supportive care for allogeneic‐hematopoietic cell transplantation (allo‐HCT) recipients, infectious complications and infection‐related mortality (IRM) continue ...to be a major issue for transplantation centers.
Methods
We herein report the infectious complications and IRM of 107 and 89 patients that underwent haploidentical (haplo‐HCT) or HLA‐identical HCT at a tertiary referral center during 2013‐2020. Patients in the haplo‐HCT group received post‐transplant cyclophosphamide (PT‐Cy), and all received reduced‐intensity conditioning regimens.
Results
More haplo‐HCT recipients presented severe infections in the pre‐engraftment period (22.4% vs. 6.7%, p = 0.003). Viral (14.9% vs. 4.5%, p = 0.016) and fungal (12.1% vs. 1.1%, p = 0.003) etiologies were more common in this period in this group. The 100‐day and 2‐year cumulative incidence of IRM was 15% and 21% for the haplo‐HCT and 5.6% and 17% for the HLA‐identical group; no significant differences were observed between the groups. Fungal pathogens mainly contributed to IRM (33.3%). Infections were the most common cause of mortality (40/81, 49.4%). There were significant differences in donor/recipient CMV serostatus between transplant groups (0.002).
Conclusions
No differences in IRM were observed based on allo‐HCT type, with more haplo‐HCT patients suffering from severe infections in the pre‐engraftment period. Studies to assess future prevention, diagnostic, and treatment strategies to reduce IRM are warranted.
Background and Objective.
Hematopoietic stem cell transplantation (HSCT) in developing countries is cost‐limited. Our primary goal was to determine the cost structure for the HSCT program model ...developed over the last decade at our public university hospital and to assess its clinical outcomes.
Materials and Methods.
Adults and children receiving an allogeneic hematopoietic stem cell transplant from January 2010 to February 2011 at our hematology regional reference center were included. Laboratory tests, medical procedures, chemotherapy drugs, other drugs, and hospitalization costs were scrutinized to calculate the total cost for each patient and the median cost for the procedure. Data regarding clinical evolution were incorporated into the analysis. Physician fees are not charged at the institution and therefore were not included.
Results.
Fifty patients were evaluated over a 1‐year period. The total estimated cost for an allogeneic HSCT was $12,504. The two most expensive diseases to allograft were non‐Hodgkin lymphoma ($11,760 ± $2,236) for the malignant group and thalassemia ($12,915 ± $5,170) for the nonmalignant group. Acute lymphoblastic leukemia ($11,053 ± 2,817) and acute myeloblastic leukemia ($10,251 ± $1,538) were the most frequent indications for HSCT, with 11 cases each. Median out‐of‐pocket expenses were $1,605, and 1‐year follow‐up costs amounted to $1,640, adding up to a total cost of $15,749 for the first year. The most expensive components were drugs and laboratory tests.
Conclusion.
Applying the cost structure described, HSCT is an affordable option for hematological patients living in a developing country.
The costs of laboratory tests, medical procedures, chemotherapy drugs, other drugs, and hospitalization of patients receiving an allogeneic hematopoietic stem cell transplantation (HSCT) were scrutinized to calculate the total cost for each patient and the median cost for the procedure and to assess its clinical outcomes. The results show that HSCT is an affordable option for hematological patients living in a developing country.
Immune thrombocytopenia (ITP) results from platelet destruction and production suppression. Eltrombopag belongs to a new class of thrombopoietin-mimetic drugs that raise platelet counts in ITP ...patients. We performed a single-arm study to assess the response to a single course of dexamethasone (40 mg by mouth, days 1-4) in combination with eltrombopag (50 mg, days 5-32) in 12 adults with newly diagnosed ITP in an outpatient setting. Median follow-up was 12.5 months. After therapy (day 33), 100% of patients achieved at least ≥30 × 109/L platelets. Four patients relapsed. Complete response at 6 months (platelets ≥100 × 109/L) was achieved in 50% of patients and response at 6 months (platelets ≥30 <100 × 109/L) was achieved in another 25%; relapse-free survival was 66.7% at 12 months (median response duration of 8.3 months). In conclusion, eltrombopag/dexamethasone is a feasible frontline therapy for ITP. This trial is registered at www.clinicaltrials.gov as NCT01652599.
•Eltrombopag/dexamethasone is a safe and effective combination for treating newly diagnosed ITP patients.•This treatment may prove useful in achieving lasting responses without additional immunosuppression in some patients.
Objective
There is a paucity of the studies of adolescents with acute lymphoblastic leukemia (ALL). This is more noticeable in low‐ and middle‐income countries. The international 5‐year event‐free ...survival (EFS) and overall survival (OS) for this age group is around 80%, with pediatric‐inspired protocols offering better results.
Methods
A retrospective analysis of adolescents aged 16–20 diagnosed with ALL during the period 2004–2015 treated with a high‐risk pediatric protocol at an academic center from a middle‐income country was performed. Five‐year OS and EFS were estimated by the Kaplan–Meier analysis. Hazard ratios of relapse and death were estimated by the Cox regression model.
Results
Five‐year EFS and OS for 57 adolescents were 23.3% and 48.9%, respectively. From the 41 patients who achieved complete remission, 24 (58.5%) relapsed. Bone marrow and central nervous system were the most frequent sites of relapse. Hazard ratio of treatment failure and death for patients with organomegaly at diagnosis was 2.026 and 2.970, respectively. Treatment‐related toxicity developed in 31 (54.4%) patients and febrile neutropenia was the most frequent in 14 (24.6%) cases. Twelve patients (21.1%) had poor adherence to treatment.
Conclusions
High relapse rate and low 5‐year EFS compared with international standards, was documented. Use of intensified pediatric regimens, adherence to proven effective medications, improved supportive care, and prevention of abandonment are necessary to improve survival rates in these patients.
Single-center prospective cohort diagnostic accuracy study. Our study aimed to evaluate the accuracy and reproducibility of Thoracic Ultrasound (TUS) in detecting pulmonary pathology in ...immunosuppressed patients. We conducted a single-center prospective study. Consecutive patients with febrile neutropenia who underwent CT (Computerized Tomography) underwent TUS evaluation within 24h of CT. Both studies were performed by an expert who was blinded to the clinical information and results of the alternative imaging modalities. 34 patients met the inclusion criteria. The median age was 39.9 years (±17 standard deviation). TUS as a diagnostic test had a sensitivity of 92.9% and specificity of 83.3%, negative predictive value of 71.4%, and positive predictive value of 96.3%. Substantial between-method agreement was demonstrated with a kappa of 0.71 (P = .001) between the TUS and chest CT findings. We obtained a kappa of 1 (P = .001) for the final diagnosis of Pleural Effusion (PE). We concluded that TUS is a promising screening test for immunocompromised individuals. The results showed good diagnostic performance of TUS compared to CT for the detection of pulmonary findings highly suggestive of pathology with high accuracy and reproducibility.
•Patient navigation is a process that helps patients overcome barriers to transplantation.•Collaboration with nonprofit patient support organizations is key.•Telemedicine is a useful tool for patient ...navigation in low- and middle-income countries.
Hematopoietic cell transplantation (HCT) is a promising treatment for hematological diseases, yet access barriers like cost and limited transplant centers persist. Telemedicine-based patient navigation (PN) has emerged as a solution. This study presents a cost-free PN telemedicine clinic (TC) in collaboration with the National Marrow Donor Program. Aim: to assess its feasibility and impac on HCT access determined by the cumulative incidence of transplantation.
In this single-center cohort study, patients of all ages and diagnoses referred for HCT participated. Two transplant physician-navigators established patient relationships via video calls, collecting medical history, offering HCT education and recommending pretransplant tests. The analysis involved descriptive statistics and intent-to-transplant survival assessment.
One hundred and three patients were included of whom n = 78 were referred for allogeneic HCT (alloHCT), with a median age of 28 years. The median time from initial contact to the first consult was 5 days. The cumulative incidence of transplantation was 50% at 6 months and 61% at 12 months, with varying outcomes based on HCT type. Notably, 49 patients were not transplanted, primarily due to refractory disease, progression or relapse (57.1%). Autologous HCT candidates and physician referrals were correlated with higher transplant success compared to alloHCT candidates and patients who were not referred by a physician.
Our pretransplant TC was feasible, facilitating access to HCT. Disease relapse posed a significant barrier. Enhancing timely physician referrals should be a focus for future efforts.
Immune thrombocytopenia (ITP) is an autoimmune disease that results from antibody-mediated platelet destruction and impaired platelet production. Novel therapies have emerged in the last decade, but ...15–20% of patients will relapse or fail and require further therapy. We performed a prospective, single-arm intervention study on seven patients with chronic, persistent, or refractory ITP from the Hospital Universitario “Dr. José E González”, in Monterrey, Mexico between 2015 and 2019. Eligible patients received oral oseltamivir 75 mg twice daily for 5 days and were followed up for six months. Most patients received a median of three distinct therapies (range 2–6). Four patients (57.1%) received combined therapy. The median time for any response was 55.5 days (range = 14–150). All patients responded at some point in time (ORR = 100%, six had a proportion of loss of response PR, and one achieved CR). Six months after oseltamivir administration, three patients (42.9%) maintained a response, and one patient had a CR (14.3%). Oseltamivir was well tolerated with a good overall response rate and was useful for treating chronic ITP. We observed an initial increase in the number of platelets; however, this response was not maintained.
Hematopoietic stem-cell transplantation (HSCT) is usually performed in well-equipped units inside a hospital. The cost of this in-hospital transplant is usually very high; therefore, this procedure ...is more difficult to perform in low- and middle-income countries. Autologous outpatient HSCT is now a common procedure; however, outpatient allogeneic transplants are more complicated. Only a few centers in the world have incorporated outpatient HSCT. This transplant requires special adaptation, like a day hospital, careful selection of patients, oral medications, and the patient must live relatively close to the hospital. The results until now suggest that this outpatient transplant is factible and similar to inpatient HSCT. The objective was to review and describe the different methods and results following an outpatient allogeneic-HSCT strategy.
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