scholarly journals Geographic Remoteness Is Associated with Increased Blood Product Utilization and Higher Rates of Hospitalization and Deaths in Adults with Immune Thrombocytopenia

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 4061-4061
Author(s):  
Erika Wall ◽  
John Podstawka ◽  
Haowei Linda Sun
Keyword(s):  
Bone Marrow ◽  
Blood Cells ◽  
Immune Thrombocytopenia ◽  
Abdominal Imaging ◽  
Blood Products ◽  
Coagulation Parameters ◽  
Packed Red Blood Cells ◽  
Chronic Itp ◽  
Geographic Remoteness

Abstract INTRODUCTION Immune thrombocytopenia (ITP) is a hematological disease characterized by immune-mediated destruction of platelets. Prior to starting therapy for ITP it is critical to determine whether it is idiopathic or related to a secondary underlying condition as this informs treatment. There is significant use of blood products and components in patients with chronic ITP for management of thrombocytopenia and bleeding, including intravenous immune globulin (IVIg). Platelet transfusions are generally reserved for life-threatening bleeding or may be used in the preoperative setting in patients unresponsive to other therapies. The aims of this study are to identify gaps in process of care and to examine the impact of geographical remoteness on health service utilization and outcomes in adults with chronic ITP in Alberta. METHODS Adults who received rituximab, splenectomy, or thrombopoietin receptor agonists (TPO-RA) as second-line therapy for ITP during 2012-2019 in the province of Alberta, Canada were identified via the provincial special drug access database. Diagnostic workup including bone marrow biopsy results, abdominal imaging (ultrasound or CT scan), coagulation parameters, viral serologies for hepatitis, human immunodeficiency virus (HIV), serum protein electrophoresis (SPEP), and quantitative immunoglobulins were recorded and rates of completed tests were calculated. Utilization of IVIg, platelets, and packed red blood cells was assessed. Rates of hospitalization, mortality, and ITP-related deaths were calculated and compared according to geographic region. RESULTS Of the 204 patients identified for analysis 106 were female (52%). Most patients (123; 60%) lived within a major centre, whereas 21 (10%) lived over 250 km from a major centre. Review of diagnostic laboratory parameters revealed incomplete coagulation parameters in 117 patients (58%), and no coagulation parameters checked in 16%. Eighty-nine patients (44%) did not have quantitative immunoglobulins tested, and 57 (28%) did not have an SPEP performed. Fifty-three (26%) did not have any abdominal imaging performed to assess for splenomegaly or liver disease. Thirty-five (17%) did not have any viral serologies for hepatitis B, C, or HIV completed. Bone marrow aspirate and biopsy was performed in 110 patients (54%). Eighty-six (77%) of these biopsies yielded a normal result. Eight biopsies (7%) displayed a lymphoproliferative disorder or plasma cell disorder which was suspected or known prior to completing the test. There was significant geographic discrepancy in utilization of blood products and hospitalizations. During 527 patient years of follow up, 83 patients received a total of 343 doses of platelets. Eleven patients (13%) received platelet transfusions for inappropriate indications, and eight (9%) for unclear indications. One hundred twenty-seven patients received IVIg (mean 1290 g) with comparable usage across geographic regions. Compared to patients within 250 km from a major centre, those with geographic remoteness (>250 km from a major centre) utilized more platelets (mean 5.2 vs 1.2 doses; Figure 1) and packed red blood cells (mean 4.3 vs 1.2 units; Figure 2). Those with geographic remoteness also experienced a higher rate of ITP-related hospitalizations (mean 1.5 vs 1.1) and deaths (24% versus 9%). At a median follow-up of 3.42 years from ITP diagnosis, 27 patients (13%) were deceased. Fourteen of these deaths were ITP-related due to bleeding or infection (52%). There appears to be a gradient of rates of both all-cause and ITP-related deaths by distance from a major centre (Figures 3 and 4). DISCUSSION This study highlights gaps in quality of care in patients with chronic ITP in Alberta, Canada. A significant number of patients have an incomplete workup for ITP at the time of diagnosis with the most forgotten tests being coagulation studies, SPEP, quantitative immunoglobulins, viral serologies, and abdominal imaging. Additionally, we identified an unexpectedly high rate of bone marrow biopsies performed in our population. Most of these bone marrow examinations did not result in any change in management. Finally, this study identified that geographic remoteness is associated with increased health services utilization and ITP-related deaths. These data can be used to inform further quality improvement initiatives in chronic ITP and help address geographic inequities in healthcare outcomes. Figure 1 Figure 1. Disclosures Sun: Bayer: Consultancy; Novo Nordisk: Consultancy; Pfizer: Consultancy; Shire: Consultancy; Octapharma: Consultancy, Research Funding.

Results of high-altitude climate treatment of immune thrombocytopenia in children in the Kyrgyz Republic (five-year analysis)

2020 ◽  
Author(s):  
А.К. Эсенгелди
Keyword(s):  
High Altitude ◽  
Immune Thrombocytopenia ◽  
Disease Duration ◽  
Objective Response ◽  
Kyrgyz Republic ◽  
Chronic Itp ◽  
Effi Ciency ◽  
The Subject ◽  
Further Development

Введение: Вопрос о дальнейшем совершенствовании базисной терапии иммунной тромбоцитопении (ИТП) по-прежнему широко обсуждается специалистами и является предметом оживленных дискуссий. Цель исследования: оценить эффективность высокогорной климатотерапии при хронической ИТП у детей за 5-летний период наблюдения после ежегодного лечения на высокогорной базе. Материалы и методы: В исследование включено 22 ребенка с хронической ИТП в возрасте от 3 до 14 лет, средний возраст — 6,30 ± 1,02 года, длительность заболевания 2-8 лет. Для лечения детей поднимали на высокогорную базу. Продолжительность лечения в высокогорье составляла 40 дней. Результаты: Из 22 детей, получивших 5 курсов высокогорной климатотерапии, у двоих наблюдали полную ремиссию в виде увеличения тромбоцитов более 100×109/л без кровоточивости. Объективный ответ в виде двукратного увеличения числа тромбоцитов, но менее 100×109/л без кровоточивости установлен у 18 детей, отсутствие эффекта было зарегистрировано у 2 детей. Заключение: Повторные курсы высокогорной климатотерапии способствуют улучшению клинической картины, существенному повышению числа тромбоцитов в периферической крови и достижению ремиссии при ИТП у детей. Background: Further development of basic treatment of immune thrombocytopenia (ITP) is still widely discussed by specialists and is the subject of lively discussions. Objectives: to assess the effi ciency of high-altitude climatotherapy in children with chronic ITP for a 5-year follow-up period after annual treatment at high-altitude base. Patients/Methods: The study included 22 children with chronic ITP from 3 to 14 years, the average age was 6.30 ± 1.02 years, and disease duration was 3-8 years. Children were raised to high-altitude base. Treatment duration was 40 days. Results: 22 children received 5 courses of high-altitude climatotherapy. Complete remission was observed in 2 patients with increasing of platelets more than 100×109/L without bleeding. In 18 children an objective response was found in the form of twofold increasing of platelets number, but less than 100×109/L without bleeding; no effect was recorded in 2 children. Conclusions: Repeated courses of high-altitude climatotherapy improve clinical characteristics, significantly increase the number of platelets in the peripheral blood and contribute to remission in children with ITP.

Novel Management of Immune Thrombocytopenia in Gaucher Disease Patients

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 5006-5006
Author(s):  
Hanna Rosenbaum
Keyword(s):  
Bone Marrow ◽  
Platelet Count ◽  
Avascular Necrosis ◽  
Gaucher Disease ◽  
Immune Thrombocytopenia ◽  
High Dose ◽  
Type I ◽  
Platelet Counts ◽  
Skeletal Complications

Abstract Type I Gaucher disease (GD) the is characterized by hepatosplenomegaly, pancytopenia and skeletal complications due to the accumulation of glucocerebroside in macrophages. Thrombocytopenia is usually related to hypersplenism and infiltration of bone marrow by lipid-laden macrophages namely Gaucher cells. Enzyme replacement therapy (ERT) restores the hemoglobin and platelet count in GD patients. In GD ERT treated patients, manifesting persistent low platelet counts, immune thrombocytopenia (ITP) should be considered.Treatment of GD with concomitant ITP is a challenge. Splenectomy may worsen bone manifestations in GD patients and is controversial. Steroids should be used with caution because of possible induction of osteopenia and joints avascular necrosis. Thrombopoietin receptor analogues (TPO-RA) are therapeutic option in GD patients with ITP. Beneficial use of TPO-RA is reported in 2 cases. Patient 1: 39 YO male with new onset of purpura and low platelet count failed treatment with 1 mg/kg of Prednisone. Bone marrow biopsy (BM) showed Gaucher cells infiltration, numerous atypical megakaryocytes, normal erythropoiesis and myelopoiesis with no fibrosis. Low level of ß-glucocerebrosidase activity with compound heterozygosity for 84GG /R495H mutations, established the diagnosis of Type I GD. Low C4 and detection of IgG platelet antibodies added to the diagnosis of concomitant immune thrombocytopenia. ERT with taliglucerase alfa (ElelysoTM) 60 Units/kg/month was given with Prednisone for six weeks. Occurrence of retinal bleeding and purpura, with decrease of platelet count necessitated addition of high-dose IVIG with no response regarding platelet counts. Splenectomy was not considered due to known bony complication risk in splenectomised GD patients. Rituximab was given to prevent wet purpura recurrence with short response regarding platelet count. Romiplostim was initiated raising platelet count from 29,000/µL to 60,000/µL after 3 wks. and to 90,000/µL after 8 wks. enabling corticosteroids withdrawal. Same dose Romiplostim is maintained for the last 30 months with platelet counts of 90,000 - 110,000/µL with no bleeding events. Repeated BMB showed no increase in collagen fibrosis. Patient 2: 63 YO female patient diagnosed with Gaucher at age 33 with a history of purpura, ecchymosis, and occasional vaginal bleeding episodes. At age 53 the platelet count dropped to < 20,000/µL with presence of Anti Platelets Ab (IgG). BMB revealed megakaryocytic hyperplasia with atypical forms, focal infiltration by Gaucher cells and no fibrosis. Combined therapy by ERT (Imiglucerase® followed by Velaglucerase Alfa®), Prednisone (1mg/kg/d for 2 months) and one course of IVIG yielded no increase in platelet count. The patient refused Rituximab®. Romiplostim was initiated increasing platelet count to100,000/µL maintained throughout a year of follow up. Repeated BMB showed slight increase of fibrosis and marked hyperplasia of atypical megakaryocytes. Discussion: Thrombocytopenia is often present in GD and may be severe in approximately 15% of the patients. Persistent cytopenias may be caused by other underlying pathologies such as autoimmune disorders and are important to be recognized and addressed. Before ERT era GD patients with hypersplenism and severe cytopenia were splenectomised. Risks of splenectomy include serious bacterial infection and vascular complications limiting its use in chronic refractory ITP. Splenectomy is avoided in Gaucher patients, due to risk of exacerbating skeletal complications (bone infarcts, avascular necrosis). Stable bone marrow results regarding fibrosis in our patients are consistent with data from a recent 2-year follow-up of 100 ITP patients receiving Romiplostim treatment with no evidence of BM fibrosis. Conclusion: In patients with type I Gaucher disease and concomitant ITP, adjunctive treatment with Romiplostim was successful in maintaining haemostatic platelet counts with no adverse effects. Traditional treatment regimens of corticosteroids and splenectomy should be used with caution or avoided in GD patients due to possible aggravation of Gaucher skeletal disease and the risk of osteopenia and avascular necrosis resulting in increased morbidity in this cohort of patients. Use of TPO-RA should be considered in GD patients with ITP. Disclosures Off Label Use: Romiplostim in gaucher patients.

scholarly journals The Transition from Childhood to Adulthood in Chronic Immune Thrombocytopenia Patients: Clinical Management and the Role of Splenectomy and Thrombopoietin Receptor Agonists in a Single Center Experience

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 4987-4987
Author(s):  
Monica Carpenedo ◽  
Marco Spinelli ◽  
Sara Pezzatti ◽  
Momcilo Jancovic ◽  
Rossella Renso ◽  
...  
Keyword(s):  
Chronic Disease ◽  
Pharmacological Treatment ◽  
Immune Thrombocytopenia ◽  
Single Center ◽  
Thrombopoietin Receptor Agonists ◽  
Chronic Itp ◽  
Thrombopoietin Receptor ◽  
Limited Compliance ◽  
Grade 3

Abstract Introduction. Children diagnosed with Immune thrombocytopenia (ITP) will develop a chronic disease (plt < 100 x 109/L lasting >12 months since the onset) in 20-30% of cases. The transition from ITP started in childhood to adulthood has been scarcely studied and no specific studies have been published Aims.To present the results of a single center retrospective survey on ITP pts diagnosed in childhood who were sent in an adult setting to continue the management Methods. Charts of ITP pts diagnosed in childhood (1-17 yrs) in our Pediatric Dept who developed a chronic disease, need at least one line of therapy and were sent to our adult ITP office from Jan 2013 to Feb 2018, were retrospectively reviewed. Demographic and clinical data were collected and outcome was assessed based on established guidelines Results. Our Pediatric Dept accounts for a mean of 37 newly ITP diagnosis/year in children aged 1-17 yrs, with 32.8% of pts developing a chronic disease. During the selected observation time, 60 pts was expected to become > 18 yrs old with a chronic ITP. Overall 28 pts (14 female) pts were sent to our Adult ITP office at pediatrician's discretion (46.6% of expected). Table 1 summarizes demographic and clinical characteristics of pts. Median age at ITP diagnosis was 9.5 yrs (12 moths to 17 yrs). Median age of pts coming to the adult ITP office was 21 yrs (18 to 37 yrs) and the reason they were sent for was the need to continue a pharmacological treatment in 8 cases (eltrombopag-Elt-), bleeding in 3 cases, pregnancy in 4 cases, plt count < 50 x 109/L in 13 cases. The median n of lines of treatment already received in childhood was 1 (1 to 4). 16 pts have received only steroid since diagnosis (on demand treatment for bleedings), 8 pts were treated with thrombopoietin receptor agonists (TPO-RA) and 1 also with rituximab. One patient, 8 yrs old, was splenectomized because of grade 3 multiple bleedings, with complete response (CR). He was sent to adult ITP office at 37 yrs when ITP relapsed, with grade 3 bleeding. Overall the median follow up from diagnosis, after the transition to adult ITP office is 18 yrs (4-39 yrs), the median n of lines at data cut-off was 2.5 (1-5) and the overall outcome is summarized in Fig 1. 11 patients were treated with TPO-RA (8 pts received Elt since childhood, 2 pts started Elt and 1 Rom in adulthood), ORR was 81.8% (CR=7). Since the transition 4 patients switched the TPO-RA because response to the first TPO-RA was suboptimal or minor side effect (headache) was reported or limited compliance was suspected. Splenectomy was offered to all patients treated with TPO-RA to avoid chronic pharmacological treatment, and to other selected patients with a low plt count and a history of bleeding. 8/11 patients accepted splenectomy. Median time since diagnosis to splenectomy was 8 yrs (1 to 18 yrs) and in all pts a stable CR (plt > 100 x 109/L) was achieved, with a median follow up after surgery of 36.5 months (10-48 months). Surgery was performed also in the previous splenectomized pt (29 yrs later) because an accessory spleen was detected, and a CR was achieved (follow up 40 months). 2 patients stopped TPO-RA on a personal decision, refused splenectomy or other treatments: their plt count is < 30 x 109/L without bleeding. 15 pts were on active follow up without therapy (median plt count 58 x 109/L) and without any bleeding. One patient is waiting for splenectomy, taking prednison. The 4 pregnant patients were treated with steroids and IVIG, had natural labour without adverse events and their babies had a normal plt count. After pregnancy one pt was treated with Elt for 1 year, then she refused treatment and splenectomy. All 4 women returned to a number of plts similar to pre-gestational count. In 1 pt a MYH9 related-disorder was confirm at 21 yrs. Overall a CR was achieved during TPO-RA treatment in 9/11 pts and 9/28 (32%) achieved a stable CR after splenectomy Conclusions. The transition from childhood to adulthood in chronic ITP pts leads clinicians to challenges related to growing age, especially in female pts approaching the fertility and pregnancy specific needs. However only a minority of children with ITP developed a chronic disease which required a prolonged treatment. TPO-RA seems to be effective and well tolerated but chronic administration has limited compliance. Splenectomy, even if performed in adulthood after many yrs since diagnosis, allows to achieve a stable CR sparing young adults from chronic pharmacological treatment. Disclosures Gambacorti-Passerini: Pfizer: Consultancy, Honoraria, Research Funding; BMS: Consultancy.

UK Helicopter Emergency Medical Services’ use of circulatory access devices, blood product transfusion and fluid warmers – a cross-sectional survey

Trauma ◽  
2018 ◽  
Vol 22 (1) ◽  
pp. 45-50
Author(s):  
Jonathan Morris ◽  
Simon Hughes
Keyword(s):  
Red Blood Cells ◽  
Blood Cells ◽  
Blood Product ◽  
Central Venous Access ◽  
Venous Access ◽  
Blood Product Transfusion ◽  
Blood Products ◽  
Central Venous ◽  
Packed Red Blood Cells ◽  
Warming Devices

Introduction The pre-hospital environment provides significant challenges to clinicians who wish to rapidly administer warmed blood products and fluids to patients with haemorrhagic shock. Large-bore circulatory access is required with the use of devices that will successfully warm cold blood with minimal impact on flow rates. Until now, no information has been available that defines UK Helicopter Emergency Medical Services’ (HEMS) use of circulatory access and fluid warming devices, nor the recent adoption of pre-hospital blood product transfusion. Methods A survey was sent to all 22 UK HEMS asking which circulatory access devices crews have available, whether blood products are being transfused and if fluid warming devices are used as part of their resuscitations. Results All services responded. All UK HEMS use peripheral intravenous cannulae and intraosseous access. In addition, seven use central venous catheters and three use large-bore peripheral access (the Arrow Rapid Infusion Catheter®). Three services use landmark technique alone to gain central venous access, whereas four use a combination of landmark and ultrasound-guided techniques. Different sites for central venous access are used: subclavian (seven services), internal jugular (four) and femoral (four). Fourteen services carry pre-hospital blood products of which six transfuse packed red blood cells; four transfuse packed red blood cells and fresh frozen plasma; four transfuse packed red blood cells and lyophilised plasma. Eight services carry no pre-hospital blood products. Seventeen HEMS use fluid warmers; 13 use the Belmont® buddy lite™ and four use the QinFlow Warrior. Conclusion The use of a variety of policies and range of equipment has evolved across UK HEMS, demonstrating a lack of consensus on best practice. This is the first study to record a complete picture of current UK HEMS practice with regard to the use of circulatory access devices, fluid warmers and blood product administration.

Can Anti-Thyroid Antibodies Influence the Outcome of Primary Chronic Immune Thrombocytopenia in Children?

2020 ◽  
Vol 20 (3) ◽  
pp. 351-355 ◽  
Author(s):  
Paola Giordano ◽  
Maurizio Delvecchio ◽  
Giuseppe Lassandro ◽  
Federica Valente ◽  
Valentina Palladino ◽  
...  
Keyword(s):  
Autoimmune Thyroiditis ◽  
Immune Thrombocytopenia ◽  
Pediatric Patients ◽  
Pediatric Population ◽  
Clinical Signs ◽  
Thyroid Antibodies ◽  
Chronic Itp ◽  
Immune Mediated

Background: Immune thrombocytopenia (ITP) is an acquired immune mediated disorder characterized by isolated thrombocytopenia. Pediatric ITP patients can develop autoantibodies such as anti-thyroglobulin (TG) and anti-thyroperoxidase (TPO), even in the absence of clinical signs of autoimmune disease. Objective: The purpose of this article is to provide a review about: 1) the prevalence of positivity of anti-thyroid antibodies (TPO and TG) in pediatric patients with chronic ITP; 2) the role of autoimmune thyroiditis on the outcome of chronic ITP. Method: The authors individually completed a review of the literature for this article. Retrospective and prospective clinical studies with pediatric cohorts were considered. Results: From the analysis of data, we found 4 papers which included studies only on pediatric population, and which corresponded to selected criteria. Pediatric ITP patients have been shown to have a statistically significant prevalence of anti-thyroid antibodies over healthy controls (11.6-36% versus 1.2-1.3%). No correlation has been found between the platelet count and the prevalence of positive anti-thyroid antibodies at any time of the follow up. Conclusion: The results of our bibliographic research demonstrated that: a) pediatric patients with chronic ITP tend to have a statistically significant prevalence of anti-thyroid antibodies positivity respect to general pediatric population; b) there are no clear data about the role of autoimmune thyroiditis as prognostic factor for chronic course of ITP in pediatric age.

scholarly journals Influence of Prescribed Blood Products on the Incidence of Deep Vein Thrombosis and Pulmonary Embolism in Surgical Patients

2017 ◽  
Vol 23 (8) ◽  
pp. 938-942
Author(s):  
Alenka Premuš Marušič ◽  
Igor Locatelli ◽  
Aleš Mrhar ◽  
Martin Caprnda ◽  
Ludovit Gaspar ◽  
...  
Keyword(s):  
Deep Vein Thrombosis ◽  
Red Blood Cells ◽  
Blood Cells ◽  
Surgical Patients ◽  
Blood Products ◽  
Control Groups ◽  
Packed Red Blood Cells ◽  
Vein Thrombosis ◽  
And Control ◽  
Deep Vein

Deep vein thrombosis (DVT) and pulmonary embolisms (PEs) are common complications after surgical procedures. The influence of prescribed blood products on the occurrence of DVT and PE was evaluated in postsurgical patients in this retrospective case–control study. The records of 286 surgical patients were analyzed: DVT (n = 52), PE (n = 92), and a control group (n = 142). The amounts of prescribed blood, blood products, and vitamin K were reviewed, together with appropriate prescribing of low-molecular-weight heparins. The influence of prescribed blood products on the occurrence of DVT or PE was analyzed using multinomial logistic regression. We demonstrated a significant difference between the test and control groups ( P < .05) in relation to receiving packed red blood cells. Treatment with red blood cells was associated with an increased risk of PE but not DVT. Patients who developed PE after surgery were hospitalized for longer (median 10 days) than patients with DVT (median 6 days). There was no difference between the test and control groups concerning treatment with fresh frozen plasma. Inadequate thromboprophylaxis significantly increased the likelihood of DVT. There is a connection between receiving packed red blood cells and occurrence of postoperative PE in surgical patients. Thus, patients receiving red blood cells should be monitored more closely after surgery, as they are more likely to develop PE postoperatively.

scholarly journals Immunoglobulin Levels As Predictors of the Development of Chronic Disease in Pediatric Immune Thrombocytopenia

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 388-388
Author(s):  
Amanda Bell Grimes ◽  
Taylor Olmsted Kim ◽  
Jenny M. Despotovic ◽  
Susan Kirk
Keyword(s):  
Chronic Disease ◽  
Autoimmune Disease ◽  
Immune Thrombocytopenia ◽  
Spontaneous Resolution ◽  
Systemic Autoimmune Disease ◽  
Chronic Itp ◽  
Age Of Diagnosis ◽  
Acute Itp ◽  
Immunoglobulin Levels

Background: Immune thrombocytopenia (ITP) is the most common acquired bleeding disorder in children. Although ~75% of these patients will go on to have spontaneous resolution of disease, up to 25% will develop chronic ITP, with significant and prolonged bleeding symptomatology and impaired quality of life. At this time, there is no definitive method to predict the development of chronic disease at the time of ITP diagnosis. Aims: We aim to identify clinical biomarkers predictive of the development of chronic ITP at the time of diagnosis among pediatric ITP patients. Methods: The clinical records of 280 pediatric ITP patients enrolled in a biological banking study at a large pediatric tertiary care referral center from July 1, 2015 to July 1, 2019 were reviewed in accordance with IRB-approved protocols. ITP diagnosis and chronicity of disease (vs. spontaneous resolution within 1 year of diagnosis) was confirmed by a pediatric hematologist, as defined by current international expert committee standards (Neunert et al, Blood, 2011;117(16):4190-207; Provan et al, Blood, 2010;115(2):168-186). Patients with non-classical ITP were excluded (1 child with drug-induced ITP in the setting of acyclovir therapy, and 1 neonate with passive ITP in the setting of maternal lupus); and patients who were lost to follow-up or had ongoing disease of &lt;1 year duration were subsequently excluded. Patient characteristics including age, gender, ethnicity, presence of concurrent autoimmune disease, and time to resolution were collected. Pertinent biomarkers including immunoglobulin levels (IgG, IgA, and IgM), anti-nuclear antibody (ANA), C-reactive protein (CRP), and immature platelet fraction (IPF) which were obtained at the time of diagnosis were documented, if if obtained prior to the administration of any ITP-directed therapy, and within 2 weeks of diagnosis. Chi-squared test or Fisher's exact test was utilized to compare nonparametric categorical data. Mann-Whitney U test was used to compare nonparametric continuous data. A multivariate backwards logistic regression model was conducted to determine independent associations with the development of chronic ITP. Statistical analyses were performed using SPSS Statistics 26 (IBM, Armonk, New York). A Bonferroni correction was applied to correct for multiple comparisons. A p value &lt; 0.05 was defined as statistically significant. Results: We identified 251 pediatric ITP patients with sufficient length of follow-up to define acute (&lt;1 year) vs. chronic (&gt;1 year) disease within our 4-year study period. This included 132 patients with acute ITP (53%) and 119 patients who went on to develop chronic ITP (47%). Mean age of diagnosis among those with acute ITP was 5.5 years, while mean age of diagnosis among those with chronic ITP was 7.7 years. Within the entire cohort, 126 (50%) were male, and 124 (49%) were of Hispanic ethnicity. Fifty-eight had ITP attributable to systemic autoimmune disease - 17% within the Acute ITP group, and 30% within the Chronic ITP group. Among all patients (n=251), 188 with evaluable biomarkers (immunoglobulin levels, ANA, CRP, or IPF) at the time of diagnosis were identified. These included 174 patients with Ig levels at diagnosis, 43 patients with ANA titers at diagnosis, 25 patients with CRP levels at diagnosis, and 78 patients with IPF at diagnosis. By univariate analysis, we found that abnormalities in immunoglobulin levels at diagnosis were significantly associated with the development of chronic ITP. Low IgG levels (p = 0.015) were more prevalent in chronic (10.3%) vs. acute ITP (1.9%). High IgG levels (p = 0.015) were more prevalent in chronic (6.7%) vs. acute ITP (1.9%). High IgM levels (p = 0.03) were more prevalent in chronic (26.5%) vs. acute ITP (13.3%). Of note, the presence of Evans syndrome (p = 0.0005) and other systemic autoimmune disease (p = 0.011) were also significantly associated with the development of chronic ITP. Subsequent multivariate analysis identified low IgG level at diagnosis to be independently predictive of chronic ITP (p = 0.043, with an odds ratio of 5.7). Conclusion: Although further study is needed within a larger international pediatric ITP cohort, the findings from this institutional study indicate that biomarkers obtained in routine clinical care at the time of ITP diagnosis, specifically immunoglobulin levels, can be utilized to help predict development of chronic disease among pediatric ITP patients. Disclosures Despotovic: Amgen: Research Funding; Dova: Honoraria; Novartis: Research Funding.

scholarly journals Laboratory involvement in the diagnosis and monitoring of paroxysmal nocturnal hemoglobinuria: a case report

2021 ◽  
Vol 106 (106(812)) ◽  
pp. 89-92
Author(s):  
M.J. Ruíz-Márquez ◽  
J. Luis-Navarro
Keyword(s):  
Bone Marrow ◽  
Blood Cells ◽  
Paroxysmal Nocturnal Hemoglobinuria ◽  
Specific Treatment ◽  
Variable Number ◽  
Variable Degree ◽  
Effective Management ◽  
Hematopoietic Stem ◽  
Thrombotic Risk

Paroxysmal nocturnal hemoglobinuria is a rare non malignant clonal disease, caused by an acquired somatic mutation in a variable number of hematopoietic stem cells, whose consequence is an abnormal sensitivity of blood cells to complement-mediated lysis. It manifests as intravascular hemolytic anemia, a variable degree of bone marrow insufficiency and high thrombotic risk. The effective management of the disease is based on an adequate diagnosis and clinical and laboratory follow-up. Flow cytometry is the method of choice for diagnosis and monitoring of the patient. The only curative treatment is hematopoietic stem cell transplantation. Eculizumab is the first specific treatment approved for this disease. We present the case of a patient diagnosed with paroxysmal nocturnal hemoglobinuria, wo has recently started treatment with eculizumab. We show the clinical and analytical evolution during the first months of treatment as an assessment of is benefit.

scholarly journals Hyperthyroidism and Immune Thrombocytopenia (ITP) Overlap Presenting as Hypoxic Ischemic Encephalopathy in a 38-Year-Old Filipino Female: A Case Report

2021 ◽  
Vol 5 (Supplement_1) ◽  
pp. A934-A935
Author(s):  
Marion Bagaloyos Sarigumba ◽  
Monica Therese B Cating-Cabral
Keyword(s):  
Bone Marrow ◽  
Red Blood Cells ◽  
Blood Cells ◽  
Asian Population ◽  
Sudden Onset ◽  
Hypoxic Ischemic Encephalopathy ◽  
Laboratory Evaluation ◽  
Packed Red Blood Cells ◽  
Parenchymal Disease ◽  
Ischemic Encephalopathy

Abstract Background: The coexistence of these two conditions can mimic severe sepsis in critically ill patients which may leave severe thyrotoxicosis unrecognized. Its higher incidence in Asian population suggests a possible genetic propensity. Clinical Case: A 38-year-old Filipino female sought consult due to changes in behavior. Two months prior, patient had persistent heavy menstrual bleeding but refused work-up. There was progressive weakness and pallor in the interim but no consult was done until she had an aggressive behavior hitting everyone she sees. Patient has Graves disease, maintained on methimazole 15 mg/day and propranolol 30 mg/day. Patient was advised to undergo radioactive iodine therapy but was lost to follow-up for 2 years. Likewise, no medication adjustment nor monitoring of biochemical parameters was done during the same period. On admission, patient was disoriented, tachycardic and febrile. She was generally pale with petechiae on the extremities and blood clots on her lips. Internal examination of the introitus revealed fresh blood with clots. Pregnancy test was negative. Initial laboratory evaluation revealed severe anemia (3.3 g/dl) and thrombocytopenia (3000/uL). Peripheral smear showed mild anisocytosis of red blood cells with no blasts nor atypical lymphocytes and platelet clumps seen. Patient received 1 unit platelet apherese and 2 units packed red blood cells (PRBC) and was started on broad spectrum antibiotic. Neurologic evaluation was negative for craniopathies. Baseline cranial CT scan was not done due to absence of lateralizing signs and was managed as a case of hypoxic ischemic encephalopathy from severe blood loss. Patient was biochemically hyperthyroid with TSH 0.065 uIU/ml, FT4 2.67 ng/dl and 10-fold elevated anti-TPO. Methimazole was then resumed at 20 mg/day and propranolol at 30 mg/day. Neck ultrasound showed an enlarged thyroid with diffuse parenchymal disease. Furthermore, patient had negative Coombs test, normal fibrinogen and procalcitonin. Patient was weakly positive for ANA at 1:160 dilution but negative for other markers in the lupus panel. Consistent with ITP, patient was started on IV hydrocortisone. In the interim, patient had less vaginal bleeding but with persistent bicytopenia which required multiple platelet and PRBC transfusions. A bone marrow biopsy was contemplated. However, on the 7th hospital day, patient had sudden onset of right sided paresthesia with dysarthria and hematomas on extremities. MRI of the brain revealed a large intraparenchymal hemorrhage and patient eventually succumbed to it. Conclusion: Bone marrow dysfunction secondary to the hyperthyroid state may explain the lack of response to standard therapy of ITP. There is short platelet lifespan, which is rather metabolic in nature, from increased reticuloendothelial phagocytic activity by upregulation of Fc receptor activity in patients with hyperthyroidism.

Romiplostim for Primary Immune Thrombocytopenia (ITP) in Routine Clinical Practice: Results from a Multicentre Observational Study in Germany

2021 ◽  
Author(s):  
Marcel Reiser ◽  
Klaus M. Josten ◽  
Hermann Dietzfelbinger ◽  
Anouchka Seesaghur ◽  
Markus Schill ◽  
...  
Keyword(s):  
Clinical Practice ◽  
Observational Study ◽  
Immune Thrombocytopenia ◽  
Routine Clinical Practice ◽  
Newly Diagnosed ◽  
Platelet Response ◽  
Chronic Itp ◽  
Primary Immune Thrombocytopenia ◽  
Post Hoc

Introduction: The effectiveness and safety of romiplostim were evaluated by immune thrombocytopenia (ITP) phase (newly diagnosed/persistent/chronic) at romiplostim initiation. Methods: Post hoc analysis of a prospective, German, multicentre, observational study in adults with ITP who received ≥1 dose of romiplostim. Follow-up data were collected for ≤2 years. Outcomes included overall platelet response (≥1 platelet count ≥50 × 109/L at 2–24 weeks after romiplostim initiation) or durable platelet response (≥75% of measurements ≥50 x 109/L at 14–24 weeks), and adverse drug reactions (ADRs), evaluated by ITP phase. Results: Data from 96 patients were analysed (newly diagnosed, n=18; persistent, n=25; chronic, n=53). During the 2–24-week follow-up, overall platelet response was achieved in 100% (95% confidence interval [CI]: 81.5–100), 100% (86.3–100), and 96.2% (87.0–99.5) of patients with newly diagnosed, persistent, or chronic ITP, respectively; platelet responses were durable in 88.2% (63.6–98.5), 65.0% (40.8–84.6), and 69.4% (54.6–81.7) of patients. During the 2-year follow-up, ADRs occurred in 24.0–35.8% of patients across phases. Two patients with chronic ITP experienced bone marrow ADRs; no thrombotic ADRs occurred. Conclusion: Romiplostim was effective and well tolerated in patients with newly diagnosed, persistent, or chronic ITP in routine clinical practice.

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