Platelet Transfusion Requirements Research Articles

Allergic reaction due to platelet transfusion in antenatal patients: A case series with a review of literature

Abstract INTRODUCTION: Thrombocytopenia is a common condition in antenatal patients that may require platelet transfusion to prevent or treat bleeding. However, platelet transfusion can cause adverse reactions, such as allergic reactions, which can range from mild urticaria to severe anaphylaxis, signifying the diversity of immune responses. The present case series describes the clinical scenarios of such antenatal patients who experienced allergic reactions after platelet transfusion and explores the possible reasons, management, and prevention of such responses. METHODS: Data from medical records of three antenatal patients who received platelet transfusion at our hospital between July 2022 and June 2023 and developed allergic reactions within 24 h of transfusion was collected, which included their demographic characteristics, clinical features, laboratory tests, treatment, and outcomes. RESULTS: The three patients were aged 23, 25, and 28 years and presented with active labor. They had thrombocytopenia due to different causes: immune thrombocytopenia and postpartum hemorrhage. They received platelet transfusions for the prevention or treatment of bleeding. Within 24 h of transfusion, they developed allergic reactions characterized by urticaria, pruritus, angioedema, and hypotension. They were treated with antihistamines, corticosteroids, and fluids and the patients responded to the treatment. The factors that triggered the allergic reactions were not determined, but possible mechanisms include immunoglobulin E (IgE)-mediated hypersensitivity, non-IgE-mediated anaphylactoid reactions, plasma protein-mediated allergic reactions, ABO-incompatible platelet transfusion, and cytokine-mediated reactions. CONCLUSION: Allergic reactions are a potential complication of platelet transfusion in antenatal patients and can be life-threatening. Clinicians should be alert to the signs and symptoms of allergic reactions and be ready to treat them quickly. The origin of allergic reactions is often unclear, but premedication, leukoreduction, and washing of platelets may reduce the risk of such reactions.

Early Cold Stored Platelet Transfusion Following Traumatic Brain Injury: A Randomized Clinical Trial.

To determine the feasibility, efficacy, and safety of cold stored compared to room temperature platelet transfusion in patients with traumatic brain injury. Data demonstrating the safety and efficacy of cold stored platelet transfusion are lacking following traumatic brain injury. A phase 2, randomized, open label, clinical trial was performed at a single U.S. trauma center. Traumatic brain injured patients with positive brain imaging and a need for platelet transfusion received up to two apheresis units of cold stored platelets stored out to 14 days versus standard care room temperature platelet transfusion. The primary outcome was feasibility and the principal clinical outcome for efficacy and safety was the 6-month Glasgow Coma Scale-Extended score. The 6-month Glasgow Outcome Scale-Extended score distributions were not different across cold stored and room temperature platelet arms (OR-1.58, 95%CI 0.71 to 3.54, P=0.27). A lower rate of neurosurgical craniotomy/craniectomy was found for those receiving cold stored platelets (difference -14.4%, 95%CI -26.5% to -2.3%, P=0.03). Adverse event rates did not differ across groups. The storage age of the cold stored product was not associated with outcome differences. In brain injured patients requiring platelet transfusion, early cold stored platelet transfusion is feasible, and did not result in improved 6-month Glasgow Coma Scale-Extended scores. Early cold stored platelet transfusion was associated with a lower rate of neurosurgical operative intervention without an increase in adverse events. The storage age of the cold stored platelet product was not associated with outcome differences. Future phase 3 clinical trials are required to determine clinical outcome differences and safety attributable to cold stored platelet transfusion following traumatic brain injury.

Thrombopoietin Receptor Agonists for Thrombocytopenia in Pediatric Hematologic Malignancies.

Thrombopoietin receptor agonists (TPO-RAs) have demonstrated efficacy in treating clinically significant thrombocytopenia, including chemotherapy-induced thrombocytopenia in adults. However, data regarding their safety and efficacy in pediatric, adolescents, and young adult (AYA) patients with hematologic malignancies are limited. We retrospectively identified 15 pediatric and AYA patients aged 25 years or younger with hematologic malignancies treated with a TPO-RA at UCSF Benioff Children's Hospitals between 2015 and 2023. Platelet counts and transfusion requirements were compared before and after TPO-RA therapy. The median age at TPO-RA initiation was 16 years (range: 7-25 years). Nine patients (60%) had a history of bleeding or comorbidity that predisposed to severe bleeding risk. Eleven patients received romiplostim and four patients received eltrombopag. The median platelet count significantly increased from 24×109/L at baseline to 54×109/L after 3 weeks of any TPO-RA therapy (p = 0.029). Monthly platelet transfusion requirements significantly decreased from a median of 15 to two units after TPO-RA therapy (p = 0.007). Fourteen of the 15 patients (93%) achieved a sustained platelet count >50,000/µL within 8 weeks, with a median time to response of 3 weeks. No TPO-RA-related adverse events were observed. TPO-RAs were effective in managing refractory thrombocytopenia in pediatric and young adult patients being treated for hematologic malignancies, with a favorable safety profile, even among patients with multiple comorbidities. These findings warrant further investigation through prospective clinical trials to confirm efficacy and establish clinical guidelines for this population.

Perioperative care of a patient with immune thrombocytopenia purpura undergoing tubo-ovarian abscess incision and drainage: Case report.

Tubo-ovarian abscess (TOA) is a serious health hazard for women, causing severe sepsis. Antimicrobial treatment is effective, but one-third of patients experience unfavorable outcomes. ITP, an autoimmune condition, can lead to bruising and bleeding. Diagnosing TOA in women of childbearing age is crucial, and combining emergency surgery with ITP patients can increase treatment costs and reduce quality of life. ITP can lead to severe complications, including postoperative hemorrhage, and may require platelet transfusions, glucocorticosteroids, and immunoglobulin. These treatments increase costs, decrease quality of life, and impact prognosis. Preventing ITP is crucial. Patients should be administered blood products based on platelet count and anemia or spontaneous bleeding tendencies. Perioperative blood management should aim for a target platelet level of 30 × 109/L and a hemoglobin concentration of 80 g/L before surgery. Post-surgery, perioperative care is crucial and vigilant for secondary bleeding. A tubo-ovarian abscess (TOA) is a frequently encountered inflammatory mass in therapeutic settings. TOA is a serious consequence of pelvic inflammatory disease (PID) that can lead to severe sepsis. In recent years, the incidence of TOA has increased, presenting a significant health hazard for women. To effectively target the diverse range of bacteria responsible for TOA, it is essential to use antimicrobial medicines that have a wide spectrum of activity. Nevertheless, the efficacy of antibiotic treatment stands at approximately 70%, while a significant proportion of patients, around one-third, experience unfavorable clinical outcomes necessitating drainage or surgical intervention. Immune thrombocytopenia (ITP) is an autoimmune condition characterized by a marked decrease in the quantity of platelets present in the bloodstream. ITP is characterized by thrombocytopenia, which leads to a heightened susceptibility to bruising and bleeding. The diagnosis of ITP and the prediction of treatment response continue to pose important and persistent issues in the field of hematology. The platelet count is commonly employed as a surrogate indicator of disease severity in patients with ITP and thus plays a crucial role in determining the necessity of treatment. A 25-year-old woman with a history of sexual activity underwent open abdominal exploration due to the sudden onset of lower abdominal pain. During the operation, a left TOA was discovered, and an incision and drainage were performed. Symptomatic treatments, such as anti-infectives and abdominal drainage, were administered. The culture of pus in the abdominal cavity suggested the presence of Escherichia coli. However, the patient presented with ITP and had a platelet count of less than 50 × 109/L before the operation. After the operation, the patient developed incisional and pelvic hematomas with signs of infection. As a result, the patient was discharged from the hospital after undergoing another laparotomy and receiving platelet transfusions and immunotherapy. Clinicians should be vigilant when diagnosing TOA in women of childbearing age, even in the absence of high-risk factors. A timely antibiotic or surgical intervention is necessary to preserve fertility and ensure quality of life. Combining emergency surgery with ITP patients poses a significant challenge for clinicians in terms of treatment. ITP can lead to serious complications, such as postoperative bleeding, which may require platelet transfusions, glucocorticoids, and immunoglobulin. This can increase the cost of treatment, reduce the quality of life, and seriously affect the prognosis. Therefore, preventing ITP is crucial. It is important to pay attention to the perioperative care of patients after surgery and be alert to the possibility of secondary hemorrhage.

Effect of platelet storage duration on platelet increment and clinical outcomes in critically ill patients – A randomised controlled trial

Background and objectivesPlatelet storage duration may influence transfusion effectiveness and patient outcomes.The present study aimed to evaluate the effect of platelet storage duration on platelet increment and clinical outcomes in patients admitted to the intensive care unit (ICU). Material and methodsThis prospective, open-label, randomized controlled trial, conducted at a single centre, enrolled ICU patients requiring platelet transfusion. Patients were randomly assigned to receive platelet concentrates aged ≤ 3 days (Group 1) or 4–5 days (Group 2). Platelet increments were assessed by Absolute Platelet Count Increment (ACI), Corrected Count Increment (CCI), and Percentage Platelet Recovery (PPR). Clinical outcomes including bleeding, infection rates, ICU stay, red cell transfusion requirements, and mortality were also monitored. ResultsPatients transfused fresher platelets (Group 1) had higher median ACI, CCI and PPR at 1 h compared to those transfused older platelets (Group 2) though the difference was not statistically significant. At 24 h, Group 1 patients had a median ACI of 28,000/µl compared to 14,000/µl in Group 2(p = 0.001). The median CCI was 16,800 in Group 1 versus 8,200 in Group 2(p = 0.001). Group 1 also had a higher median PPR of 45.7% compared to 23.6% in Group 2(p = 0.011).There was no significant difference in clinical outcomes such as bleeding, infection rates, ICU stay, or mortality between the groups. Multivariate analysis indicated that co-morbidities and higher APACHE-III score were associated with increased mortality. ConclusionTransfusion of fresher platelets resulted in higher increments and transfusion effectiveness but did not affect clinical outcomes or mortality. Trial registration detailsClinical Trials Registry of India (CTRI/2023/03/050676).

Safety and outcomes of an on-demand transfusion strategy versus prophylactic transfusion of platelets in patients with liver cirrhosis and severe thrombocytopenia undergoing high-risk procedures: A post-hoc analysis of two randomized controlled trials

BackgroundThere are limited studies assessing whether prophylactic platelet transfusions prior to high-risk procedures reduce the risk of bleeding in patients with liver cirrhosis. MethodsWe performed a post-hoc analysis of two prior randomized clinical trials (CTRI/2017/12/010822 and CTRI/2021/05/033464), which compared TEG-guided prophylactic platelet transfusion to standard of care (empirical prophylactic transfusion for all patients prior to the procedure) or on-demand transfusion (no prophylactic transfusions). We aimed to assess the risk of major procedure-related bleeding or mortality among patients who had received prophylactic platelet transfusions versus those who did not (on-demand transfusions). ResultsA total of 118 patients were included in the analysis, with baseline demographics well matched between groups. The leading etiologies of cirrhosis were cryptogenic (42, 35.6%) and autoimmune liver disease (30, 25.4%). The most common procedures performed were percutaneous liver biopsy (73, 61.8%), followed by transjugular intrahepatic portosystemic shunt (14,11.9%) and transarterial chemoembolization (14,11.9%) respectively. No episode of major bleeding or procedure-related mortality occurred in either group, though minor bleeding occurred in 5 patients. A significantly lower number of patients in the on-demand group required platelet transfusions compared to those receiving empirical transfusions as part of standard care. ConclusionProcedure-related bleeding rates were not significantly higher among patients with liver cirrhosis undergoing high-risk procedures without prophylactic platelet transfusions compared to those who received them. Larger randomized trials are required to validate these findings from our post-hoc analysis.

Clinical characteristics and outcomes of preterm versus term uterine rupture: a nationwide observational study.

To assess and compare the clinical aspects of uterine rupture by dividing the gestational age at uterine rupture occurrence into < 37-week (preterm) and ≥ 37-week (term) groups. This retrospective cohort study analyzed data from 187 acute-care hospitals across Japan and included patients who experienced uterine rupture. Data were sourced from the Diagnosis Procedure Combination inpatient database, spanning July 2010 to March 2022. The patients' characteristics, in-hospital procedures, and outcomes were compared between those with uterine rupture at < 37 and ≥ 37weeks of gestation. The main outcomes were hysterectomy, complications, proportion of blood transfusions, and postoperative length of stay. A total of 298 patients were identified, with 161 in the preterm group and 137 in the term group. Placenta accreta spectrum occurred more frequently in the preterm group than in the term group (18.0% vs. 6.6%, respectively; P = 0.003). Vacuum delivery (19.0% vs. 0.6%, P < 0.001) and uterine fundal pressure (2.9% vs. 0.0%, P = 0.004) were more likely to be applied in the term group. The maternal need for mechanical ventilation (26.3% vs. 12.4%, P = 0.003), the proportion of disseminated intravascular coagulation (40.1% vs. 25.5%, P = 0.009), and the requirement for platelet transfusions (32.8% vs. 15.5%, P < 0.001) were greater in the term. The postoperative hospital stays were also longer in the term group. This study shows that individual characteristics vary with the gestational age at uterine rupture, and maternal morbidity is notably higher in term compared to preterm ruptures.

Refractory Thrombocytopenia is the Earliest Diagnostic Criterion for Sinusoidal Obstruction Syndrome in Children.

Sinusoidal obstruction syndrome (SOS) is a life-threatening complication of hematopoietic stem cell transplantation (HSCT), whose diagnostic criteria changed over time to achieve a timelier diagnosis. Recently, pediatric-specific criteria presented by the European Society for Blood and Marrow Transplantation (pEBMT) incorporated transfusion-refractory thrombocytopenia (RT) as an early indicator of SOS in children. However, a comparison of all individual diagnostic parameters belonging to pEBMT and former SOS diagnostic criteria has never been performed. This retrospective study conducted at a pediatric tertiary care hospital analyzed all pediatric HSCT cases diagnosed with SOS among 170 children transplanted from 2009 to 2023. Eleven patients developed SOS during this period (incidence: 11/170, 6.5%). pEBMT, Seattle, and Baltimore criteria were retrospectively applied to the 11 cases and compared, showing that RT was the earliest fulfilled parameter (median onset: 6d post-HSCT). pEBMT and Seattle criteria identified 11/11 SOS cases, with pEBMT leading to an earlier diagnosis. RT typically manifested before diagnosis, with significantly higher platelet transfusion requirements before diagnosis than after. RT is the earliest satisfied criterion in pediatric SOS and typically occurs in the initial stages of the disease before diagnosis. Further research is needed to identify additional early indicators of pediatric SOS.

Inhibition of RHOA activity preserves the survival and hemostasis function of long-term cold-stored platelets

AbstractPatients with thrombocytopenia require platelet transfusion to prevent and stop hemorrhage. Cold storage of platelets results in complex molecular lesions, including changes in membrane microdomains that are recognized by host macrophages and hepatocyte counter-receptors, resulting in phagocytosis and clearance upon transfusion. For this reason, platelets are stored at room temperature, a method that confers increased risk of bacterial contamination. By applying signaling analysis and genetic and pharmacological approaches, we identified that cold-induced activation of RAS homolog family, member A (RHOA) GTPase causes the major hallmarks of platelet cold storage lesions. RHOA deficiency renders murine platelets insensitive to cold storage–induced damage, and pharmacological inhibition by a RHOA activation inhibitor, R-G04, can prevent the cold storage–induced lesions. RHOA inhibition prevents myosin activation and clathrin-independent formation and internalization of lipid rafts enriched in active glycosyltransferases as well as abnormal distribution of GPIbα. RHOA inhibition further prevents the metabolic reprogramming of cold storage–induced lesions and allows the maintenance of glycolytic flux and mitochondria-dependent respiration. Importantly, human platelets transfused in mice after cold storage, in the presence of R-G04 or its more potent enantiomer S-G04, can circulate in vivo at similar levels as room temperature–stored platelets while retaining their hemostatic activity in vivo, as assessed by bleeding time correction in aspirin-treated mice. Our studies provide a mechanism-based translational approach to prevent cold storage–induced damage, which is useful for human platelet transfusion in patients with thrombocytopenia.

Immature platelet fraction levels predict the development of prolonged thrombocytopenia after haematopoietic stem cell transplantation

Prolonged thrombocytopenia (PT) is a serious complication after haematopoietic stem cell transplantation (HSCT). PT has been suggested to be associated with an increased platelet transfusion requirement and poor outcomes after transplantation. Due to the complex mechanism of PT development, it is difficult to diagnose in the early post-transplant period. Our study aimed to identify an early predictive marker for PT after HSCT. Previous studies showed that the clinical utility of immature platelet fraction (IPF) predicts platelet recovery after chemotherapy and successful engraftment. However, the relationship between IPF and PT after HSCT remains unclear. Fifty-two patients with malignant haematological diseases who underwent HSCT were included in the study. We observed the kinetics of recovery of haematological parameters after transplantation and performed receiver operating characteristics (ROC) curve analysis using data from the 52 HSCT patients. The days to rise and peak of IPF, absolute IPF count (A-IPF) and highly fluorescent IPF (H-IPF) were almost synchronised in all patients, at day 10 and day 15, respectively. The begin to rise levels of IPF, H-IPF and A-IPF were all significantly lower in the PT group than in the good engraftment (GE) group (p=0.0016, p=0.0094, p=0.0086, respectively). The peak levels of IPF were significantly lower in the PT group than the GE group (p=0.0036). However, the peaks of H-IPF and A-IPF were not statistically significant between the two groups (p=0.3383, p=0.0887, respectively). The area under the ROC curve (AUC) of IPF rise was 0.739 (95% CI 0.583-0.896; p<0.05) and the cut-off value was 3.5%, while the AUC of IPF peak was 0.800 (95% CI 0.637-0.962; p<0.01) and the cut-off value was 8.0%. In conclusion, early low levels of IPF predict the development of PT after HSCT. These findings may help improve the management and treatment strategies for PT after HSCT.

Delayed cold-stored vs. room temperature stored platelet transfusions in bleeding adult cardiac surgery patients—a randomized multicentre pilot study (PLTS-1)

BackgroundPlatelets stored at 1–6 °C are hypothesized to be more hemostatically active than standard room temperature platelets (RTP) stored at 20–24 °C. Recent studies suggest converting RTP to cold-stored platelets (Delayed Cold-Stored Platelets, DCSP) may be an important way of extending platelet lifespan and increasing platelet supply while also activating and priming platelets for the treatment of acute bleeding. However, there is little clinical trial data supporting the efficacy and safety of DCSP compared to standard RTP.MethodsThis protocol details the design of a multicentre, two-arm, parallel-group, randomized, active-control, blinded, internal pilot trial to be conducted at two cardiac surgery centers in Canada. The study will randomize 50 adult (≥ 18 years old) patients undergoing at least moderately complex cardiac surgery with cardiopulmonary bypass and requiring platelet transfusion to receive either RTP as per standard of care (control group) or DCSP (intervention group). Patients randomized to the intervention group will receive ABO-identical, buffy-coat, pathogen-reduced, platelets in platelet additive solution maintained at 22 °C for up to 4 days then placed at 4 °C for a minimum of 24 h, with expiration at 14 days after collection. The duration of the intervention is from the termination of cardiopulmonary bypass to 24 h after, with a maximum of two doses of DCSP. Thereafter, all patients will receive RTP. The aim of this pilot is to assess the feasibility of a future RCT comparing the hemostatic effectiveness of DCSP to RTP (defined as the total number of allogeneic blood products transfused within 24 h after CPB) as well as safety. Specifically, the feasibility objectives of this pilot study are to determine (1) recruitment of ≥ 15% eligible patients per center per month); (2) appropriate platelet product available for ≥ 90% of patients randomized to the cold-stored platelet group; (3) Adherence to randomization assignment (> 90% of patients administered assigned product).DiscussionDCSP represents a promising logistical solution to address platelet supply shortages and a potentially more efficacious option for the management of active bleeding. No prospective clinical studies on this topic have been conducted. This proposed internal pilot study will assess the feasibility of a larger definitive study.Trial registrationNCT 06147531 (clinicaltrials.gov).

A retrospective comparison of abbreviated course “7+7” vs standard hypomethylating agent plus venetoclax doublets in older/unfit patients with newly diagnosed acute myeloid leukemia.

6507 Background: Hypomethylating agent (HMA) plus venetoclax (VEN) regimens are standard of care in older/chemotherapy ineligible patients with acute myeloid leukemia (AML). While the VEN label recommends continuous 28-day cycles, shortened VEN durations may mitigate cytopenias and improve tolerability. It is unknown how shorter VEN cycles compare to standard HMA+VEN in terms of efficacy/safety. Methods: We performed a retrospective comparison of patients with newly diagnosed (ND) AML treated with azacitidine (AZA) x 7 days plus VEN x 7 days (“7+7” regimen) at 7 French centers (n=82, Willekens, ASH 2022) vs patients treated with standard dose HMA+VEN (s-HMA/VEN) at a US center (n=173), generally consisting of 21-28 days of VEN. We compared composite complete remission rate (CRc, consisting of CR + CRi), overall survival (OS), event-free survival (EFS), and myelosuppression between regimens. Results: Baseline characteristics are shown in the table. The s-HMA-VEN group consisted of 10-day decitabine (DAC) in 59% with the remaining patients receiving 5-day DAC or 7-day AZA. The CRc rate was similar; 72% with “7+7” vs 71% with s-HMA/VEN (p=0.89). The CR rate was 57% with “7+7” vs 55% with s-HMA/VEN (p=0.72). Median cycles to first response was 1 in both groups, however 42% of responders on “7+7” required more than 1 cycle for first response whereas almost all responders on s-HMA-VEN (99%) had a first response after cycle 1. Median cycles to best response was 2 with “7+7” vs 1 with s-HMA-VEN (p=0.02). 4-week mortality was similar (2% with “7+7”, 6% with s-HMA-VEN; p=0.24). 8-week mortality was higher with s-HMA-VEN (17%) compared to “7+7” (6%) (p=0.02). The median OS was 11.2 months (2-year 28%) with “7+7” vs 10.1 months (2-year 33%) with s-HMA/VEN (p=0.93). Outcomes stratified by molecular groups will be updated at the meeting. Fewer patients required platelet transfusions during cycle 1 with “7+7” compared to s-HMA/VEN (62% vs 77%, p=0.01). The cycle 1 rates of neutropenic fever and red cell transfusion requirements were similar. Conclusions: Acknowledging the limitations of a retrospective comparison across multiple centers, we did not observe a signal for a difference in efficacy between "7+7" vs standard VEN-based HMA doublets in ND-AML. The “7+7” regimen was associated with lower platelet transfusion requirements and lower 8-week mortality. [Table: see text]

Prediction Model for Severe Thrombocytopenia Induced by Gemcitabine Plus Cisplatin Combination Therapy in Patients with Urothelial Cancer.

Chemotherapy-induced thrombocytopenia is often a use-limiting adverse reaction to gemcitabine and cisplatin (GC) combination chemotherapy, reducing therapeutic intensity, and, in some cases, requiring platelet transfusion. A retrospective cohort study was conducted on patients with urothelial cancer at the initiation of GC combination therapy and the objective was to develop a prediction model for the incidence of severe thrombocytopenia using machine learning. We performed receiver operating characteristic analysis to determine the cut-off values of the associated factors. Multivariate analyses were conducted to identify risk factors associated with the occurrence of severe thrombocytopenia. The prediction model was constructed from an ensemble model and gradient-boosted decision trees to estimate the risk of an outcome using the risk factors associated with the occurrence of severe thrombocytopenia. Of 186 patients included in this study, 46 (25%) experienced severe thrombocytopenia induced by GC therapy. Multivariate analyses revealed that platelet count ≤ 21.4 (×104/µL) [odds ratio 7.19, p<0.01], hemoglobin ≤ 12.1 (g/dL) [odds ratio 2.41, p=0.03], lymphocyte count ≤ 1.458 (×103/µL) [odds ratio 2.47, p=0.02], and dose of gemcitabine ≥ 775.245 (mg/m2) [odds ratio 4.00, p<0.01] were risk factors of severe thrombocytopenia. The performance of the prediction model using these associated factors was high (area under the curve 0.76, accuracy 0.82, precision 0.68, recall 0.50, and F-measure 0.58). Platelet count, hemoglobin level, lymphocyte count, and gemcitabine dose contributed to the development of a novel prediction model to identify the incidence of GC-induced severe thrombocytopenia.

Necrotising enterocolitis in a middle-income country: Early onset and risk factors for mortality.

There is limited literature available about necrotising enterocolitis (NEC) in low- and middle-income countries. This study sought to determine the proportion, pattern and risk factors for mortality among very low birth weight (VLBW) neonates with NEC in a middle-income setting. A retrospective observational cohort study was conducted on all infants with birth weights less than 1501 g admitted from 2018 to 2020 at Groote Schuur Hospital, Cape Town, South Africa. Data were obtained from the Vermont Oxford Network and hospital folders. A total of 104/1582 (6.6%) neonates were diagnosed with NEC with a median onset of 8 days of life. The mortality rate was 39.0%, compared to the all-cause mortality rate of 18.7% for VLBW neonates. Thirty-two (32.0%) neonates with NEC were transferred for surgery of whom 10 (31.3%) died. Small for gestational age (p = 0.13), NEC stage 2B or above (p = 0,002), a positive blood culture (p = 0.018), a raised C-reactive protein (p = 0.013), hyponatraemia (p = 0.004), anaemia requiring blood transfusion (p = 0.003) and thrombocytopenia requiring platelet transfusion (p = 0.033) were associated with mortality. A positive blood culture was found in 37.0% with a predominance of Klebsiella pneumoniae isolates. NEC has an early onset and a high mortality rate in our setting.

Comparison of etoposide combined with G-CSF and cyclophosphamide combined with G-CSF in the mobilization of autologous peripheral blood stem cells in patients with multiple myeloma

Objective: The effect and safety of etoposide combined with G-CSF were compared with those of cyclophosphamide combined with G-CSF in autologous peripheral blood mobilization in patients with multiple myeloma (MM) . Methods: Patients with MM who received autologous peripheral blood stem cell mobilization and collection in the Department of Hematology, Beijing Chaoyang Hospital Affiliated to Capital Medical University from January 1, 2020 to July 31, 2023 were included. A total of 134 patients were screened by propensity score matching technology according to a 1∶1 ratio. A total of 67 cases were each treated with ETO combined with G-CSF mobilization scheme (ETO group) and CTX combined with G-CSF mobilization scheme (CTX group). Their clinical data were retrospectively analyzed. Results: ①Collection results: the ETO and CTX groups [2 (1-3) d vs 2 (1-5) d; P<0.001] and CD34(+) cells [7.62×10(6) (2.26×10(6)-37.20×10(6)) /kg vs 2.73×10(6) (0.53×10(6)-9.85×10(6)) /kg; P<0.001] were collected. The success rate of collection was 100.0% (67/67) versus 76.1% (51/67) (P<0.001). Excellent rate of collection was 82.1% (55/67) versus 20.9% (14/67; P<0.001). Two patients in the ETO group switched protocols after 1 day of collection, and 11 patients in the CTX group switched protocols after 1-2 days of collection. ②Adverse reactions: granular deficiency with fever (21.5%[14/65] vs. 10.7%[6/56]; P=0.110), requiring platelet transfusion [10.7% (7/65) vs 1.8% (1/56) ; P=0.047]. ③Until the end of follow-up, 63 cases in the ETO group and 54 cases in the CTX group have undergone autologous transplantation. The median number of CD34(+) cells infused in the two groups was 4.62×10(6) (2.14×10(6)-19.89×10(6)) /kg versus 2.62×10(6) (1.12×10(6)-5.31×10(6)) /kg (P<0.001), neutrophil implantation time was 11 (9-14) d versus 11 (10-14) d (P=0.049), and platelet implantation time was 11 (0-19) d vs. 12 (0-34) d (P=0.035). One case in the CTX group experienced delayed platelet implantation. Conclusion: The mobilization scheme of etoposide combined with G-CSF requires relatively platelet transfusion, but the collection days are shortened. The collection success rate, excellent rate, and the number of CD34(+) cells obtained are high, and the neutrophil and platelet engraftment is accelerated after transplantation.

Effect of Recombinant Human Thrombopoietin on Platelet Reconstitution after Autologous Peripheral Blood Stem Cell Transplantation in Patients with Multiple Myeloma

To analyze the effect of recombinant human thrombopoietin (rhTPO) on platelet (PLT) reconstitution after autologous peripheral blood stem cell transplantation (APBSCT) in patients with multiple myeloma (MM). The clinical data of 147 MM patients who were diagnosed in the First Affiliated Hospital of Soochow University and received APBSCT as the first-line therapy were retrospectively analyzed. According to whether rhTPO was used during APBSCT, the patients were divided into rhTPO group (80 cases) and control group (67 cases). The time of PLT engraftment, blood product infusion requirements, the proportion of patients with PLT recovery to≥50×109/L and≥100×109/L at +14 days and +100 days after transplantation, and adverse reactions including the incidence of bleeding were compared between the two groups. There were no significant differences between the two groups in sex, age, M protein type, PLT count at the initial diagnosis, median duration of induction therapy before APBSCT, and number of CD34+ cells reinfused (all P >0.05). The median time of PLT engraftment in the rhTPO group was 10 (6-14) days, which was shorter than 11 (8-23) days in the control group (P < 0.001). The median PLT transfusion requirement in the rhTPO group during APBSCT was 15(0-50)U, which was less than 20 (0-80)U in the control group (P =0.001). At +14 days after transplantation, the proportions of patients with PLT≥50×109/L in the rhTPO group and the control group were 66.3% and 52.2%, while the proportions of patients with PLT≥100×109/L were 23.8% and 11.9%, respectively, with no significant differences (all P >0.05). At +100 days after transplantation, the proportion of patients with PLT≥50×109/L in rhTPO group and control group was 96.3% and 89.6%, respectively (P >0.05), but the proportion of patients with PLT≥100×109/L in rhTPO group was higher than that in control group (75.0% vs 55.2%, P =0.012). There was no difference in the overall incidence of bleeding events in different locations during period of low PLT level of patients between the two groups. In rhTPO group, the rhTPO administration was well tolerated, and the incidences of abnormal liver and kidney function and infection were similar to those in the control group. When MM patients undergo first-line APBSCT, subcutaneous injection of rhTPO can shorten the time of platelet engraftment, reduce the transfusion volume of blood products, and be well tolerated, moreover, more patients have achieve a high level of PLT recovery after transplantation, which is very important for ensuring the safety of APBSCT and maintenance therapy.

Acute pulmonary injury in hematology patients supported with pathogen-reduced and conventional platelet components

AbstractPatients treated with antineoplastic therapy often develop thrombocytopenia requiring platelet transfusion, which has potential to exacerbate pulmonary injury. This study tested the hypothesis that amotosalen-UVA pathogen–reduced platelet components (PRPCs) do not potentiate pulmonary dysfunction compared with conventional platelet components (CPCs). A prospective, multicenter, open-label, sequential cohort study evaluated the incidence of treatment-emergent assisted mechanical ventilation initiated for pulmonary dysfunction (TEAMV-PD). The first cohort received CPC. After the CPC cohort, each site enrolled a second cohort transfused with PRPC. Other outcomes included clinically significant pulmonary adverse events (CSPAE) and the incidence of treatment-emergent acute respiratory distress syndrome (TEARDS) diagnosed by blinded expert adjudication. The incidence of TEAMV-PD in all patients (1068 PRPC and 1223 CPC) was less for PRPC (1.7 %) than CPC (3.1%) with a treatment difference of –1.5% (95% confidence interval [CI], –2.7 to –0.2). In patients requiring ≥2 PCs, the incidence of TEAMV-PD was reduced for PRPC recipients compared with CPC recipients (treatment difference, –2.4%; 95% CI, –4.2 to –0.6). CSPAE increased with increasing PC exposure but were not significantly different between the cohorts. For patients receiving ≥2 platelet transfusions, TEARDS occurred in 1.3% PRPC and 2.6% CPC recipients (P = .086). Bayesian analysis demonstrated PRPC may be superior in reducing TEAMV-PD and TEARDS for platelet transfusion recipients compared with CPC recipients, with 99.2% and 88.8% probability, respectively. In this study, PRPC compared with CPC demonstrated high probability of reduced severe pulmonary injury requiring assisted mechanical ventilation in patients with hematology disorders dependent on platelet transfusion. This trial was registered at www.ClinicalTrials.gov as #NCT02549222.

The Prevalence and Associated Risk Factors for Neonatal Thrombocytopenia Among Newborns Admitted to the Neonatal Intensive Care Unit.

Background Thrombocytopenia is the most prevalent hematological condition in neonates that develops in the neonatal intensive care unit (NICU). This set of illnesses is caused by either decreased platelet production due to placental insufficiency, increased platelet breakdown (consumption), or a combination of the two causes. Based on platelet count, it is defined as mild, moderate, or severe thrombocytopenia, with early and late onset. Purpose The purpose of this study is to determine the prevalence of thrombocytopenia and the factors that contribute to it in newborns hospitalized in the neonatal critical care unit at the Maternity and Children Hospital in Al Ahsa, Saudi Arabia. Methods This descriptive retrospective cross-sectional study was carried out at the NICU of the Maternity and Children Hospital in Al Ahsa, Saudi Arabia, over the span of one year (August 2022 to August 2023) among hospitalized neonates with thrombocytopenia. Thrombocytopenia is defined as a platelet count of 150,000 or less. These patients were monitored until they recovered or died. Results The inclusion criteria were met by a total of 242 newborns with thrombocytopenia. Half of the neonates (57%) were full-term, with Apgar scores greater than 5 at the first (84%) and fifth (93%) minutes, respectively. The great majority of individuals (84%) experienced early-onset thrombocytopenia of mild severity (62%) and were asymptomatic (93%). The majority of the cases resolved spontaneously, with only 21%requiring platelet transfusion. There was a significant relationship discovered between gestational age and the severity of thrombocytopenia, with very preterm infants having moderate to severe thrombocytopenia, as well as birth weight (p=0.001). Furthermore, neonates with severe thrombocytopenia had a considerably higher mortality rate (p=0.001). Conclusion The mortality and morbidity of newborns with perinatal risk for neonatal thrombocytopenia can be reduced with timely detection of the cause and development of thrombocytopenia, as well as adequate and early care.

Clinical Signs and Treatment of New-Onset Bone Marrow Failure Associated SARS-CoV-2 Infection in Children: A Single Institution Prospective Cohort Study.

Viral infections can cause direct and indirect damage to hematopoietic stem cells. The objectives of this study were to identify the frequency and severity of aplastic anemia in children infected with severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) as well as recognize the response to treatment. 13 children with newly diagnosed severe aplastic anemia were enrolled in this prospective clinical trial. Blood samples were obtained from all patients to detect SARS-CoV-2 antibodies, and nasopharyngeal swabs were collected for reverse-transcription Polymerase Chain Reaction to detect SARS-CoV-2 viruses. According to the laboratory results, patients were classified as having SARS-CoV-2 positive antibodies and SARS-CoV-2 negative antibodies. Both groups received combined cyclosporine (CsA) + Eltrombopag (E-PAG). The hematological response, either complete response (CR) or partial response (PR), no response (NR), and overall response (OR) rates of combined E-PAG + CsA treatment after 6 months were evaluated. Four children were recognized to have aplastic anemia and SARS-CoV-2 positive antibodies. Two patients fulfilled the hematological criteria for CR and no longer required transfusion of packed red blood cells (PRBCs) or platelets, and one had PR and was still PRBC transfusion-dependent but no longer required platelet transfusion. The remaining patient showed NR, and he had died before reaching the top of the HSCT waiting list. Moreover, six patients in the SARS-CoV-2 negative antibodies group had CR, while three patients had PR. The difference in ANC, Hg, and platelet counts between both groups was not significant. The SARS-CoV-2 virus is added to several viral infections known to be implicated in the pathogenesis of aplastic anemia. Studies are needed to establish a definitive association and determine whether the response of bone marrow failure to standard therapy differs from that of idiopathic cases.

Etoposide combined with cytarabine and pegfilgrastim for poorly mobilizing patients with multiple myeloma and lymphoma: A prospective multicentre study.

A chemotherapy-based mobilization regimen in patients who mobilize poorly, based on etoposide, cytarabine and pegfilgrastim (EAP), has recently been introduced. The aim of this prospective study was to investigate the efficacy and safety of the EAP regimen in patients with poorly mobilizing multiple myeloma (MM) or lymphoma. This single-arm clinical trial was performed at eight public hospitals in China and was registered as a clinical trial (NCT05510089). The inclusion criteria were; (1) diagnosis of MM or lymphoma, (2) defined as a 'poor mobilizer' and (3) aged 18-75 years. The EAP regimen consisted of etoposide 75 mg/m2/day on days 1-2, cytarabine 300 mg/m2 every 12 h on days 1-2 and pegfilgrastim 6 mg on day 6. The primary endpoint of the study was the ratio of patients achieving adequate mobilization (≥2.0 × 106 CD34+ cells/kg). From 1 September 2022 to 15 August 2023, a total of 58 patients were enrolled, 53 (91.4%) achieved adequate mobilization, while 41 (70.7%) achieved optimal mobilization with a median number of cumulative collected CD34+ cells was 9.2 (range 2.1-92.7) × 106/kg and the median number of apheresis per patient of 1.2. The median time from administration of the EAP regimen to the first apheresis was 12 days. Approximately 8.6% of patients required plerixa for rescue, which was successful. Twelve (20.7%) of the 58 patients suffered grade 2-3 infections, while 25 (43.1%) required platelet transfusions. The duration of neutrophil and platelet engraftment was 11 days. In conclusion, these results suggest that the EAP mobilization regimen might be a promising option for poorly mobilizing patients with MM or lymphoma.