All-Trance Retinoid Acid (ATRA) Makes Refractory Immune Thrombocytopenia (RITP) Less Refractory: A Retrospective Study of Ritp Patients Treated with Combination Therapies Including ATRA

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 3338-3338
Author(s):  
Min Wang ◽  
Xiao-hui Zhang ◽  
Wei Han ◽  
Yu Wang ◽  
Dai-Hong Liu ◽  
...  
Keyword(s):  
Immune Thrombocytopenia ◽  
Conflicts Of Interest ◽  
Treatment Protocol ◽  
Peak Level ◽  
Autoimmune Disorder ◽  
The Other ◽  
Severe Bleeding ◽  
First Line ◽  
Phone Calls ◽  
Bleeding Episodes

Abstract Abstract 3338 Background: Refractory immune thrombocytopenia (RITP) is a severe bleeding disorder with a high mortality rate of 10% to 30%, which is resulted from the toxicities associated with therapies, as well as from life-threatening bleedings. Up to now, treatments for RITP patients are still limited and no consensus has been reached about the standard treatment protocol, therefore, it remains a great problem and challenge for hematologists to think out ways to treat RITP, that is to say, new therapies should be explored to maintain a relatively stable and safe platelet counts with minimum toxicities, so as to improve patients' quality of life and reduce mortalities related to severe bleedings and therapies. ITP is an autoimmune disorder ascribed not only to increased PLT destruction, but also to reduced PLT production, which appears to be related to impaired megakaryocytes (MKs) maturation. ATRA belongs to a class of retinoids, which exerts immunomodulatory and differentiation inducing capacities, and has been included in clinical trails about autoimmune diseases and has been successfully applied in clinic to cure APL by inducing the differentiation of cancer cells. Design and Methods: In this study, we retrospectively reviewed the medical charts of 35 RITP patients who took ATRA as part of the combination therapy from February 2008 to August 2012. We made phone calls for some other detailed medically relevant information not recorded, including previous ITP history, bleeding episodes and etc. All the patients fulfilled the primary ITP criteria as described. They had a median ITP duration of 29 months (range, 6–129 months), with a diagnosed RITP duration of 11months (range, 2–79 months). All the patients have received a median of 6 therapies (range, 3–8) prior to taking ATRA, including steroids, IVIG, CSA, azathioprine, danazol and etc. The median lowest PLT number in the course of their disease was 12×109/L (range, 1–27×109/L), and the median PLT count before starting take ATRA was 11×109/L (range, 1–23×109/L). Patients were treated with ATRA (10mg 3times/day), in combination with any one of methylprednisolone, danazol and CSA or nothing not randomly. When patients were severely thrombocytopenia or present with bleeding sings or symptoms, transfusion of PLT or injection of thrombopoietin were applied. Patients were monitored every 1 to 3 days at the first two weeks and every 1to 4 weeks afterwards for PLT counts, and were monitored every 1 month for transaminitis and other side effects. Results: Of the 35 patients, 25 responded to our treatments in a median of 8 weeks (range, 3–16 weeks), with PLT increased to greater than 30*109/L, and remained in remission in a median of 24 months, after which, 10 patients relapsed and 8 patients regained remission with the addition of other drugs. The other 15 patients remained in remission after 24 months during the maintenance therapy process and the following drugs decrement process. 5 patients had a relatively stable and safe PLT count (median 58×109/L, rang 38–225×109/L) after discontinuation of all the medications and the other 10 continued the therapy with a low dose for relapsing after the medicine discontinuation. The median peak PLT count after starting the ATRA therapy was 94×109/L (rang 57–225×109/L), and it is after a median of 3 month (range, 1.5–8 months) before the median PLT count reached to peak level. During the treatment process, no severe adverse events and bleedings happened. Patients refractory to previous conventional first-line therapies and combined therapies responded to the combination of ATRA with any one of methylprednisolone, danazol or CSA, which implies that ATRA has the potential of making RITP less refractory and even curing RITP. Given to the limited samples size and nonrandomization of our study, no prognostic factors were found in our study, including sex, age, ITP duration, the lowest PLT count, previous therapy numbers, previous bleeding episodes, the combined drug and etc. Conclusion: ATRA may have the potential to help RITP patients gain remission and maintain remission combined with one of the conventional first-line therapies, which requires to be verified by more large-sized, prospective, randomized, and controlled clinical trials, and the mechanism of ATRA in treating RITP also needs to be further investigated. Disclosures: No relevant conflicts of interest to declare.

scholarly journals Hypovitaminosis D Influences the Clinical Presentation of Immune Thrombocytopenia in Children with Newly Diagnosed Disease

2019 ◽  
Vol 8 (11) ◽  
pp. 1861
Author(s):  
Petrovic ◽  
Benzon ◽  
Batinic ◽  
Culic ◽  
Roganovic ◽  
...  
Keyword(s):  
Immune Thrombocytopenia ◽  
Clinical Presentation ◽  
Coastal Region ◽  
Autoimmune Disorder ◽  
Hypovitaminosis D ◽  
Severe Bleeding ◽  
Newly Diagnosed ◽  
Randomized Controlled Studies ◽  
Bleeding Score ◽  
D Values

Immune thrombocytopenia (ITP) is an acquired autoimmune disorder characterized by isolated thrombocytopenia defined as platelet count in peripheral blood <100 × 109/L. Hypovitaminosis D is very common in children with autoimmune diseases. To analyze whether hypovitaminosis D is associated with the clinical presentation of ITP in children, medical records of 45 pediatric patients with newly diagnosed immune thrombocytopenia in the coastal region of Croatia were evaluated. The severity of bleeding was assessed using two bleeding scores. Children with lower 25-hydroxyvitamin D (25(OH)D) values had higher values of the skin-mucosa-organ-gradation (SMOG) bleeding score and respectively more severe bleeding on diagnosis of ITP. With further analysis of the main domains of that score, we found that patients with a lower 25(OH)D value had more severe bleeding in the skin and organs. When 25(OH)D and ITP Bleeding Scale (IBLS) score were analyzed, a negative correlation was found, but it was not significant. Our findings suggest that hypovitaminosis D influences the severity of the clinical presentation of ITP in children on initial diagnosis of the disease. Therefore, therapy with 25(OH)D could be a new potential option for treatment of ITP. To investigate the connection between 25(OH)D and the incidence and severity of ITP, further studies, especially randomized controlled studies, are needed.

Primary Immune Thrombocytopenia (ITP) Guidelines for Bone Marrow Aspirate Reduce the Diagnostic Rate of Myelodysplastic Syndromes

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 4974-4974
Author(s):  
Iolanda Vincelli ◽  
Esther Natalie Oliva ◽  
Francesca Ronco ◽  
Patrizia Cufari ◽  
Bianca Oliva ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Immune Thrombocytopenia ◽  
Bone Marrow Aspirate ◽  
Conflicts Of Interest ◽  
Autoimmune Disorder ◽  
Case Review ◽  
Initial Screening ◽  
Immune Mediated ◽  
Primary Immune Thrombocytopenia ◽  
Systemic Symptoms

Abstract Abstract 4974 Introduction: Primary immune thrombocytopenia (ITP) is an acquired immune-mediated disorder characterized by isolated thrombocytopenia, defined as a peripheral blood PLT count <100 Gi/L, and the absence of any obvious initiating and/or underlying cause of the thrombocytopenia (Rodeghiero et al, Blood, 2009). Guidelines consider bone marrow (BM) aspirate informative in patients with thrombocytopenia > 60 years of age, those with systemic symptoms or abnormal signs or in some cases where splenectomy is considered (Provan et al, Blood, 2009). Methods: We performed a retrospective chart analysis between January 2008 and June 2010 of all patients referred to our clinic for isolated thrombocytopenia but with a PLT count 100– 149 Gi/L. According to recent guidelines, these patients are not to be considered thrombocytopenic and do not require further investigation. The aim of the study was to evaluate the validity of omitting BM analysis in these cases. Results: Twenty-three cases (13 males/10 females) of mean age 58 ± SD 19 years were evaluated at our clinic for a PLT count below normal lab range values. At the time of evaluation none had bleeding symptoms. PLT counts ranged from 101 to 149 Gi/L, mean 123 Gi/L. Four patients had an enlarged spleen. After initial screening, 2 patients had a complex autoimmune disorder and 1 case had HCV hepatitis. The remaining 20 patients had a bone marrow (BM) aspirate performed: a diagnosis of myelodysplastic syndrome (MDS, WHO classification refractory thrombocytopenia) was obtained in 13 cases (65%) and BM biopsy was performed in 12, completed by cytogenetics in 9 cases (7 normal, 1 del20q, 1 –Y). Patients diagnosed with MDS were significantly older (66 ± SD 13 vs 47 ± SD 21 years, p = 0.017), but 4 cases (31%) were < 60 years of age (44, 49, 51 and 55 years of age, respectively). Conclusions: The most recent guidelines, which lower the PLT threshold to 100 Gi/L from 150 Gi/L for the investigation of causes of thrombocytopenia, reduce the diagnostic rate of MDS. In our retrospective review, 65% of patients would not have had an early diagnosis of MDS. Furthermore, BM aspirate should be considered irrespective of age, since one third of the patients in our case review had MDS with PLT > 100 Gi/L as a single cytopenia and age under 60. Disclosures: No relevant conflicts of interest to declare.

Immune Thrombocytopenia Associated to Low-Dose Alemtuzumab Therapy in Chronic Lymphocytic Leukemia: A Single Retrospective Center Experience

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 4598-4598
Author(s):  
Gianluigi Reda ◽  
Francesco Maura ◽  
Giuseppe Gritti ◽  
Daniele Vincenti ◽  
Mariarita Sciumé ◽  
...  
Keyword(s):  
Chronic Lymphocytic Leukemia ◽  
Disease Progression ◽  
Cumulative Dose ◽  
Immune Thrombocytopenia ◽  
Low Dose ◽  
Conflicts Of Interest ◽  
Autoimmune Disorder ◽  
Intravenous Immunoglobulins ◽  
Lymphocytic Leukemia ◽  
Platelet Counts

Abstract Abstract 4598 Immune thrombocytopenia (ITP) is a common autoimmune complication in chronic lymphocytic leukemia (CLL), occurring in up to 5% of patients. The occurrence of alemtuzumab-associated ITP have been rarely reported in CLL and it has never been reported so far as a significant event complicating alemtuzumab treatment in clinical trials. Recently, a new distinctive form of secondary ITP occurring in 6 out of 215 patients with relapsing-remitting multiple sclerosis treated with alemtuzumab in a randomized, controlled phase II trial has been reported (Cuker et al, Blood 2011). We investigated the incidence of ITP in a cohort of 64 consecutive patients treated with low-dose alemtuzumab for relapsed/refractory CLL from 2003 to 2010. Subcutaneous alemtuzumab was administered at the dose of 10 mg three times a week, up to 18 weeks. ITP was documented in 12/64 patients: in 3 patients (4.7%) ITP developed before alemtuzumab treatment and no relapses of the autoimmune disorder were observed during the treatment; in 9 patients (14.8%, with an incidence of 5.7 events/100 patient-year) ITP developed after alemtuzumab treatment. Median time to ITP from initial alemtuzumab exposure was 12 months (range 1–42 months). Concomitant hemolytic anemia (Evans syndrome) was observed in one patient. At ITP onset, median platelet counts were 11×109/L (range 3–70) and anti-platelet antibodies (Capture P® Method, ImmucorGamma, Norcross GA, USA) were found in 7 of the 8 patients tested. No patients showed severe or life-threatening bleeding. Three of nine patients who developed ITP after alemtuzumab therapy, experienced CLL progression requiring chemo-immunotherapy after 3, 4 and 13 months from ITP onset, respectively. One patient achieved a partial remission of CLL with ITP resolution, while the other two died of disease progression. In the remaining six cases, ITP was not associated with disease progression and was treated with corticosteroids and/or intravenous immunoglobulins. Five patients achieved normal platelet counts, while one patient did not respond. Low-dose alemtuzumab (theoretical cumulative dose 540 mg, equal to half of the classic cumulative dose and one-third of the individual dose) is an effective, safe and well tolerated treatment for CLL, as reported by several recent studies (Cortelezzi et al, Leukemia 2009; Brit J Haematol 2011). In our cohort of CLL patients treated with alemtuzumab, the incidence of ITP was 14.8% that is almost three times higher than previously reported in CLL. These data may indicate a role of T-lymphocyte dysregulation induced by alemtuzumab in the pathogenesis of ITP. Our data also suggested the importance of monitoring platelet counts during follow-up in patients treated with low-dose alemtuzumab for both haematological and non-haematological diseases. Disclosures: No relevant conflicts of interest to declare.

scholarly journals Thrombopoietin Receptor Agonist Use, Efficacy and Toxicity in Children with Immune Thrombocytopenia: Data from an Italian Multicenter Survey

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 4997-4997
Author(s):  
Paola Giordano ◽  
Giuseppe Lassandro ◽  
Marco Spinelli ◽  
Momcilo Jancovic ◽  
Paola Saracco ◽  
...  
Keyword(s):  
Platelet Count ◽  
Immune Thrombocytopenia ◽  
Conflicts Of Interest ◽  
Immunosuppressive Agents ◽  
Real Life ◽  
Complete Response ◽  
Clinical Use ◽  
First Line ◽  
Thrombopoietin Receptor ◽  
Multicenter Survey

Abstract Background: Immune Thrombocytopenia (ITP) is one of the most common conditions encoutered by the pediatric hematologist. Current first-line therapy includes: observation without drug therapy, corticosteroids and intravenous immune globulin. A minority of patients are refractory to first-line approaches. Second-line treatment options are: immunosuppressive agents and thrombopoietin receptor agonists (TPO-RA). Eltrombopag and Romiplostin are TPO-RA licensed for clinical use. Eltrombopag is, actually, the only TPO-RA approved in Italy (since two years ago) for children, over one year old, with a chronic and/or refractory ITP. Real life data of Eltrombopag are limited. Methods: We performed an Italian multicenter retrospective survey to study the clinical on-label use of TPO-RA, focus on Eltrombopag, in pediatric ITP. Our aims were, primarily, to bring out the prevalence of the use in clinical practice and secondarily to collect data on efficacy and toxicity. Results: We enrolled 69 pediatric ITP subjects from 15 Italian treatment centers (TC). 4 patients received Romiplostin as TPO-RA and were excluded by the analysis. 36/65 patients weer female (55%). Median age at ITP diagnosis: 6 years + 6 months (min 1 y + 2 m; max 16 y + 7 m). Median age at first Eltrombopag assumption: 11 years + 5 months (min 2 y + 0 m; max 17 y + 8 m). Accounting in 344 the total number of chronic ITP subjects treated by TC in the same observation period (July 2016-June 2018), we observed an Eltrombopag clinical use prevalence of 0.19 (95% CI 0.15 to 0.26). We underlined a "no response" to Eltrombopag (platelet count persistently less than 30000 per microliter) in 16/65 (25%); a "partial response" (platelet count between 30000 and 100000 per microliter) in 14/65 (21%) and a "complete response" (platelet count persistently up than 100000 per microliter) in 35/65 (54%). The overall response (partial or complete) was described in 49/65 (75%) children. During the follow up was seen in 16/49 (33%) subjects with initial response a platelet rise that waned to no response. There was no evidence of significant adverse events (clinicians are obliged, to monthly surveillance, by Italian drug agency for hypertransaminasemia and peripheral smear cell abnormalities). Conclusions: Our results demonstrate that Eltrombopag is a therapeutic option quite considered by Italian clinicians. Moreover, according with the percentages of clinical trials, Eltrombopag is safe and effective to rise platelet count. Further studies need to emphasize how factors favor a complete response and to know the incidence of long-term adverse effects. A prospective study designed and driven by Italian Association of Pediatric Hematology Oncology (AIEOP) Coagulation Disorders Working Group is, already, in progress. Disclosures No relevant conflicts of interest to declare.

Pattern of Care and Association of Response with Rituxan Among Primary and Secondary Immune Thrombocytopenia Patients

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 4920-4920 ◽  
Author(s):  
Ajaz Bulbul ◽  
Sadaf Rashad ◽  
Denice Tsao-Wei ◽  
Susan Groshen ◽  
Matney Huber ◽  
...  
Keyword(s):  
Immune Thrombocytopenia ◽  
Conflicts Of Interest ◽  
Line Treatment ◽  
Platelet Response ◽  
First Line ◽  
Pattern Of Care ◽  
Exact Test ◽  
Age Of Diagnosis ◽  
Eventual Loss ◽  
Line Setting

Abstract Background: Immune thrombocytopenia (ITP) is a heterogeneous disorder of immune dysregulation of T and autoreactive B cells leading to immune mediated destruction of platelets due to eventual loss of immune tolerance against platelet epitopes. Whether intensification regimens like Rituximab improve sustained platelet response (SR) across different lines is unclear. Methods: A retrospective review of three hundred sixty-two patients of thrombocytopenia seen between Jan 1990 to June 2016 across three rural community practices across southeastern NM was performed. Eighty-eight patients with chronic liver disease and or drug related thrombocytopenia were excluded from the statistical analysis. Fisher's exact test was used to test the association of treatment and response in both primary and secondary ITP. International working group (IWG-2009) classification was used to assess response criteria. (SR) was defined as a platelet count of >50,000 per cubic millimeter six months after treatment. Results: Two hundred thirty-three patients (64%) had primary ITP 41 (11%) had secondary ITP. Median age of diagnosis was 61 and median platelet on diagnosis was 90 (0-148). Of the 274 patients included in this analysis, 143 ITP patients (52%) were followed by observation alone and 131 (48%) received treatment. Sixty-six (24%) patients received second line treatment and further 41 (15%) patients received third line treatment. Complete remission (CR) was achieved in 68% in first line. Sixty-one percent patients had a CR with Prednisone in first line as compared to 84% with dexamethasone (p=0.15). Forty-seven (17%) of all ITP patients received Rituximab (across multiple lines of treatment). Twenty percent of patients who achieved CR/PR in first line had received IVIg. Rituximab was used in 9%,39% and 34% of first, second and third lines of treatment respectively. Across all lines of treatment Rituximab was not associated with a higher CR (p=0.47) or SR (p=0.35) (Table 1). Fifteen percent of patients had toxicity requiring interruption of Rituximab including 1 patient with sepsis. Hyperglycemia, insomnia were the three most common toxicities seen in 19%, 11% patients overall (Table 2). Conclusion: Rituximab use across all lines of treatment was not associated with a higher CR or SR rate in our dataset. The higher than expected Response rates may be due to inclusion of other intensification treatments like IVIg. The use of Rituximab however is limited in real world rural practices especially in the first line setting possibly partly due to insurance denials and cost. Its use seems more common in subsequent lines of treatment. Other ways to intensifying therapy in first line should be evaluated in prospective clinical trials like use of IVIg with or without the use of Rituximab and whether upfront intensive treatment avoids earlier recurrence need to be studied. Table 1 Table 1. Disclosures No relevant conflicts of interest to declare.

scholarly journals Eltrombopag in immune thrombocytopenia: efficacy review and update on drug safety

2018 ◽  
Vol 9 (6) ◽  
pp. 263-285 ◽  
Author(s):  
Jose Ramon Gonzalez-Porras ◽  
Jose Maria Bastida
Keyword(s):  
Risk Factors ◽  
Clinical Trials ◽  
Clinical Practice ◽  
Immune Thrombocytopenia ◽  
Autoimmune Disorder ◽  
Efficacy And Safety ◽  
First Line ◽  
Platelet Production ◽  
Thrombopoietin Receptor ◽  
Current Guidelines

Immune thrombocytopenia (ITP) is an autoimmune disorder that induces a decrease in the number of circulating platelets due to spleen destruction and inability of megakaryocytes to restore normal counts. Immunosuppressive therapy with glucocorticoid drugs constitutes the first line of treatment. However, lack of response to these agents is not uncommon, and the management of refractory patients is a matter of controversy. In fact, day-to-day clinical practice shows that, in spite of the current guidelines, splenectomy, which is currently considered a suitable second-choice therapy, is being replaced by treatment with thrombopoietin receptor agonists. These boost platelet production by megakaryocytes. The use of one of these, namely eltrombopag, has been permitted for ITP patients refractory to first-line drugs or splenectomy, for the last 10 years. This review summarizes the experience reported using eltrombopag in ITP, paying attention to efficacy and safety. Results from clinical trials will be discussed, and studies performed in the course of daily clinical practice will also be reviewed, as these are useful to assess the potential of the drug in real-world settings. The management of adverse events and the use of eltrombopag in particular situations will also be covered. The experience reported so far permits us to suggest that eltrombopag efficiently induces recovery of platelet counts. Furthermore, recent papers have demonstrated that a sustained response after discontinuation, initially thought to be problematic, may be possible in a nonnegligible number of cases. The safety profile is satisfactory, although patients presenting with thromboembolism risk factors should be treated with caution until the eltrombopag-associated prothrombotic risk is fully established. In summary, although larger studies are still needed to clarify some issues, eltrombopag may be a useful alternative tool for ITP patients refractory to conventional medical management or splenectomy.

scholarly journals Long-Term Prophylaxis in Patients with Von Willebrand Disease and Recurrent Bleeding in Italy

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 5056-5056 ◽  
Author(s):  
Piercarla Schinco ◽  
Cultrera Dorina ◽  
Federica Valeri ◽  
Alessandra Borchiellini ◽  
Cristina Teruzzi ◽  
...  
Keyword(s):  
Conflicts Of Interest ◽  
Von Willebrand Disease ◽  
Recurrent Bleeding ◽  
Severe Bleeding ◽  
Bleeding Tendency ◽  
Transfusion Requirements ◽  
Bleeding Episodes ◽  
Type 3 ◽  
Von Willebrand

Abstract INTRODUCTION: Von Willebrand disease (VWD) is a congenital bleeding disorder caused by Von Willebrand factor (VWF) deficiency or abnormalities. Apart from administration of desmopressin in mild-moderate cases, replacement therapy with VWF/Factor VIII (FVIII) concentrates is the therapy of choice for short-term prophylaxis (STP) during surgery or clinical interventions and it is the conventional approach for Long-Term Prophylaxis (LTP) in patients with severe bleeding tendency. However, LTP with FVIII/VWF concentrates is not always effective and in some cases. Repeated infusions of FVIII/VWF concentrates may increase the risk of thromboembolic events due to FVIII:C overload. The aim of this analysis was to evaluate the effectiveness of LTP with a VWF concentrate almost devoid of FVIII:C ( vWF conc.) vs VWF/FVIII concentrate in VWD patients with a heavy bleeding phenotype. METHODS: A retrospective analysis on four VWD patients [type 3 (n= 1), type 2M (n= 1) and type 1 (n= 2)] was carried out in two Italian Hemophilia Centers. These Centers have clinical experience with VWD patients with severe bleeding tendency, on LTP, who switched from VWF/FVIII concentrates to vWF conc. in order to obtain a better control of the bleedings. The patients were included in the analysis if they fulfilled the following criteria: periodical bleeding episodes and previous prophylaxis with VWF/FVIII concentrates. The patients had been previously treated with VWF/FVIII concentrates (prophylaxis: 35-50 IU/Kg/3 times per week; bleeding episodes: 30-50 IU/Kg/day as long as needed), before starting prophylaxis with vWF conc. (prophylaxis: 30-50 IU/Kg/2 times per week and in one case 30 IU/Kg/3 times per week; bleeding episodes: 30-50 IU/Kg/day as long as needed). Data on number of bleeding episodes and hospital admittances, hemoglobin, FVIII:C levels, and number of transfusions were collected. Data after 1 year’s prophylaxis with vWF conc. were compared with those after 1 year’s prophylaxis with VWF/FVIII concentrates. RESULTS: The number of all bleeding episodes decreased by 96% (mean 14 to 0,5 episodes) and all patients showed a consistent reduction of their own bleeding frequency. Hemoglobin and FVIII:C levels respectively increased by a mean of 2,8 gr/dl (from 8,05 gr/dl to 10,85 gr/dl) and 40% (from 17% to 57%) after LTP with vWF conc. The patient transfusion requirements (number of packed red blood cell concentrate-PRBC) dropped from a mean of 3.5 to zero, and the number of ordinary hospitalizations and day hospital admittances decreased by 100% after LTP with vWF conc. (mean of 15 to 0). CONCLUSIONS: Our analysis suggests that LTP with a VWF concentrate almost devoid of FVIII is effective and well tolerated highly beneficial for patients both in terms of reduction of bleeding episodes and reduction of number of days spent in hospital with an obvious improvement of the quality of life. A collection of clinical data from a larger population of patients is required to confirm and support these results. Disclosures No relevant conflicts of interest to declare.

scholarly journals Trends of Splenectomy in Pediatric Patients with Immune Thrombocytopenia before and after the ASH 2011 Evidence-Based Practice Guidelines

Blood ◽  
2017 ◽  
Vol 130 (Suppl_1) ◽  
pp. 676-676
Author(s):  
Neel S Bhatt ◽  
Parth Bhatt ◽  
Keyur Donda ◽  
Fredrick Dapaah-Siakwan ◽  
Sunil Kumar Batalahally ◽  
...  
Keyword(s):  
Practice Guidelines ◽  
Immune Thrombocytopenia ◽  
Pediatric Patients ◽  
Conflicts Of Interest ◽  
Large Population ◽  
Multivariable Analysis ◽  
Significant Risk ◽  
Severe Bleeding ◽  
Discharge Data ◽  
Total Splenectomy

Abstract Background: Immune thrombocytopenia (ITP) is an autoimmune disorder which affects 1 in 10,000 children. Thrombocytopenia occurs largely due to rapid destruction of antibody sensitized platelets in the reticuloendothelial system. While spontaneous resolution is commonly seen in acute ITP, treatment of chronic or persistent ITP is controversial and could pose as management challenges for clinicians. Splenectomy increases platelet survival by removing a common site of platelet destruction and has a 70-80% response rate, however, published literature is conflicting regarding its ideal timing. Furthermore, it is an invasive procedure with significant risk of infections, venous thromboembolism. In 2011, the American Society of Hematology (ASH) published revised clinical practice guidelines for the management of ITP (Neunert C et al. Blood 2011). Splenectomy was recommended in patients with severe symptomatic ITP with significant or persistent bleeding refractory to intravenous immunoglobulin (IVIG), corticosteroids, and anti-D therapy. It is unclear if the rates of splenectomy have changed since the publication of these guidelines. Using a large population-based estimate, we aimed to study the trends of splenectomy in pediatric patients with ITP, and the factors predicting the procedure in this population. Methods: We used discharge data from the National (Nationwide) Inpatient Sample (NIS), Healthcare Cost and Utilization Project (HCUP), Agency for Healthcare Research and Quality (AHRQ). Pediatric patients (age ≤ 18 years) hospitalized with ITP from 2005-2014 were identified using International Classification of Diseases, Ninth Revision, Clinical Modification (ICD9-CM) code 287.31. Cases of total splenectomy were identified using ICD9-CM procedure code '41.50'. To calculate estimated trends of splenectomy, Cochran-Armitage trend test was used. Chi-square test, Mann-Whitney U test, survey logistic regression and hierarchal regression were used for analyses. Weights provided by NIS were used to calculate national estimates. A p-value &lt; 0.05 was considered significant. All analyses were performed using SAS 9.4 (SAS Institute Inc., Cary, NC, USA). Results: The NIS data included 62 million weighted pediatric hospitalizations between 2005 and 2014 (unweighted admissions n=13 million). After excluding patient transfers, a total of 40,998 weighted hospitalizations with ITP occurred during this time-period. Total splenectomy was performed in 1,009 patients. Splenectomy rate showed a decline prior to revised guidelines: 3.3% (2005-06) to 2.3% (2011-12). It continued to decline further after the publication: 1.6% (2013-14), p &lt;0.0001. Similar trend was noted for patients &lt; 5 years of age: 10.6% (2005-06), 5.2% (2011-12), and 3.5% (2013-14), p &lt;0.001. None of the other patient ages (5-10, 11-15, and 16-18), gender, or hospital regions showed consistent splenectomy trend in either direction. Multivariable analysis showed one year increase in patient age significantly increased adjusted odds of having splenectomy (odds ratio, OR 1.18, 95% confidence interval, CI 1.14-1.22, p &lt;0.0001). Having an intracranial bleed also significantly predicted splenectomy (OR 5.16, 95% CI 1.96- 13.61, p=0.0009). Splenectomy was more commonly done in patients from Midwest (OR 2.83, 95% CI 1.19-6.74, p= 0.02) and South region (OR 1.89, 95% CI 1.02-3.51, p=0.04) compared to Northeast region. Mortality rate was similar between patients who received splenectomy and who did not. Conclusion: Overall, splenectomy rates showed consistently downward trend in pediatric patients with ITP between 2005 and 2014. Reduction in splenectomy rates in children &lt;5 years of age could be due to increasing awareness for sepsis secondary to asplenia. Presence of severe bleeding (e.g. intracranial bleeding) in patients with ITP predicted splenectomy, which is consistent with ASH recommendations. Declining splenectomy rates in last 3 years since ASH guidelines release could mean that more patients receive medical management initially as recommended by ASH. However, due to possible under-coding for IVIG, corticosteroids, and anti-D and lack of longitudinal data, we were unable to study the association between prior treatment and splenectomy. Future studies should revisit the role of splenectomy in ITP with advent of novel therapeutic options such as thrombopoietin receptor agonists. Disclosures No relevant conflicts of interest to declare.

scholarly journals Effects of Ramadan Fasting on Patients with Immune Thrombocytopenia (ITP) Receiving Eltrombopag

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 8-9
Author(s):  
Mohamed A Yassin ◽  
Aya Alasmar ◽  
Prem Chandra ◽  
Omer Ismail ◽  
Abdulqadir Jeprel Nashwan ◽  
...  
Keyword(s):  
Health Care Providers ◽  
Immune Thrombocytopenia ◽  
Conflicts Of Interest ◽  
Empty Stomach ◽  
Oral Drug ◽  
Minor Bleeding ◽  
Bleeding Tendency ◽  
Care Providers ◽  
Significant Drop ◽  
Bleeding Episodes

Introduction: The thrombopoietin receptor agonists (TPO-RA) such as eltrombopag (ELT) have, in recent years, changed immune thrombocytopenia (ITP) treatment, showing that there is both increased platelet destruction and suboptimal platelet production in ITP. ELT is an oral drug and studies have shown that absorption can be affected by meals. Thus, for optimum absorption, patients are counseled to take the ELT on empty stomach at least 1 hour before or 2 hours after a meal and at least 2 hours before and 4 hours after calcium containing meals. However, in the Muslim world, the holy month of Ramadan, where Muslims have to fast since dawn till sunset the scheduling of drug consumption can be affected. Objective: The aim of this study is to evaluate the effect of fasting on patients with ITP receiving Eltrombopag. Methods: A mixed design study was performed in which we retrospectively analyzed and interviewed all Muslim patients (n=29) who are 18 years and older in Qatar and fasted Ramadan while receiving Eltrombopag treatment between the years of 2015 and 2019 recording clinical and biological parameters and using a standardized patient form. Fasting status was confirmed by a telephone call to each patient. Patients' responses were retrospectively evaluated before, during and after Ramadan. A significant drop in platelets was defined by either drop in platelets to &lt; 30 000 /L or drop to ≥ half from baseline. Bleeding tendency was also evaluated as either no bleeding, minor cutaneuos and mucosal bleeding or life threatening bleeding involving major organs (i.e. central nervous system, gastrointestinal or genitourinary). Results: Twenty-nine patients were identified who matched our inclusion criteria for this study. Majority of which were Arabs (~83%) whereas the rest were of the sub-continent of India. Platelet responses were statistically significantly impacted (P-Valua: 0.005) by the fasting of the holy month of Ramadan, the mean platelet count before Ramadan was estimated at 87.78±67.75 while during Ramadan it dropped to 46.57±36.50. Wheras when compared Platelet beofr and after Ramadan showed insignificant difference (116.4±68.2 vs 132.9±91.3; P=0.389). Only 10% (3 patients) of the patients fasting Ramadan experienced bleeding episodes. 1 of which experienced minor bleeding (ecchymosis) and 2 had major bleeding episodes during Ramadan (CNS and GU bleeds). Conclusion: ELT is generally well tolerated and effectively achieves target platelet counts in adult ITP patients. However, the absorption of ELT is highly effected by food consumption and thus it is critical to adhere to the instruction given on how ELT should be consumed on an empty stomach. As has been shown due to the mis-scheduling of ELT consumption during the holy month of Ramadan. Health care providers must pay more attention to this period and provide a better tailored plan for patients in order to prevent platelet drop and pouts of bleeding that patients might experience Disclosures No relevant conflicts of interest to declare.

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