Beneficial Effect of Intravenous Dexamethasone in Children With Mild to Moderately Severe Acute Chest Syndrome Complicating Sickle Cell Disease

Blood ◽  
1998 ◽  
Vol 92 (9) ◽  
pp. 3082-3089 ◽  
Author(s):  
Juan Carlos Bernini ◽  
Zora R. Rogers ◽  
Eric S. Sandler ◽  
Joan S. Reisch ◽  
Charles T. Quinn ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Beneficial Effect ◽  
Hospital Stay ◽  
Sickle Cell ◽  
Linear Regression Analysis ◽  
Therapeutic Modality ◽  
Acute Chest Syndrome ◽  
High Dose ◽  
Blood Transfusions ◽  
Cell Disease

Abstract Acute chest syndrome (ACS) in patients with sickle cell disease (SCD) has historically been managed with oxygen, antibiotics, and blood transfusions. Recently high-dose corticosteroid therapy was shown to reduce the duration of hospitalization in children with SCD and vaso-occlusive crisis. Therefore, we chose to assess the use of glucocorticoids in ACS. We conducted a randomized, double-blind placebo-controlled trial to evaluate the efficacy and toxicity of intravenous dexamethasone (0.3 mg/kg every 12 hours × 4 doses) in children with SCD hospitalized with mild to moderately severe ACS. Forty-three evaluable episodes of ACS occurred in 38 children (median age, 6.7 years). Twenty-two patients received dexamethasone and 21 patients received placebo. There were no statistically significant differences in demographic, clinical, or laboratory characteristics between the two groups. Mean hospital stay was shorter in the dexamethasone-treated group (47 hours v 80 hours; P = .005). Dexamethasone therapy prevented clinical deterioration and reduced the need for blood transfusions (P < .001 and = .013, respectively). Mean duration of oxygen and analgesic therapy, number of opioid doses, and the duration of fever was also significantly reduced in the dexamethasone-treated patients. Of seven patients readmitted within 72 hours after discharge (six after dexamethasone; P = .095), only one had respiratory complications (P = 1.00). No side effects clearly related to dexamethasone were observed. In a stepwise multiple linear regression analysis, gender and previous episodes of ACS were the only variables that appeared to predict response to dexamethasone, as measured by lengh of hospital stay. Intravenous dexamethasone has a beneficial effect in children with SCD hospitalized with mild to moderately severe acute chest syndrome. Further study of this therapeutic modality is indicated. © 1998 by The American Society of Hematology.

Beneficial Effect of Intravenous Dexamethasone in Children With Mild to Moderately Severe Acute Chest Syndrome Complicating Sickle Cell Disease

Blood ◽  
1998 ◽  
Vol 92 (9) ◽  
pp. 3082-3089 ◽  
Author(s):  
Juan Carlos Bernini ◽  
Zora R. Rogers ◽  
Eric S. Sandler ◽  
Joan S. Reisch ◽  
Charles T. Quinn ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Beneficial Effect ◽  
Hospital Stay ◽  
Sickle Cell ◽  
Linear Regression Analysis ◽  
Therapeutic Modality ◽  
Acute Chest Syndrome ◽  
High Dose ◽  
Blood Transfusions ◽  
Cell Disease

Acute chest syndrome (ACS) in patients with sickle cell disease (SCD) has historically been managed with oxygen, antibiotics, and blood transfusions. Recently high-dose corticosteroid therapy was shown to reduce the duration of hospitalization in children with SCD and vaso-occlusive crisis. Therefore, we chose to assess the use of glucocorticoids in ACS. We conducted a randomized, double-blind placebo-controlled trial to evaluate the efficacy and toxicity of intravenous dexamethasone (0.3 mg/kg every 12 hours × 4 doses) in children with SCD hospitalized with mild to moderately severe ACS. Forty-three evaluable episodes of ACS occurred in 38 children (median age, 6.7 years). Twenty-two patients received dexamethasone and 21 patients received placebo. There were no statistically significant differences in demographic, clinical, or laboratory characteristics between the two groups. Mean hospital stay was shorter in the dexamethasone-treated group (47 hours v 80 hours; P = .005). Dexamethasone therapy prevented clinical deterioration and reduced the need for blood transfusions (P < .001 and = .013, respectively). Mean duration of oxygen and analgesic therapy, number of opioid doses, and the duration of fever was also significantly reduced in the dexamethasone-treated patients. Of seven patients readmitted within 72 hours after discharge (six after dexamethasone; P = .095), only one had respiratory complications (P = 1.00). No side effects clearly related to dexamethasone were observed. In a stepwise multiple linear regression analysis, gender and previous episodes of ACS were the only variables that appeared to predict response to dexamethasone, as measured by lengh of hospital stay. Intravenous dexamethasone has a beneficial effect in children with SCD hospitalized with mild to moderately severe acute chest syndrome. Further study of this therapeutic modality is indicated. © 1998 by The American Society of Hematology.

Red Blood Cell Exchange Transfusion, An Underutilized Effective and Reliable Therapeutic Modality in Sickle Cell Disease, Reduces Number of Admissions and Length of Hospital Stay

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 4822-4822
Author(s):  
Aref Agheli ◽  
Kirshma Khemani ◽  
Madhumati Kalavar ◽  
William Steier ◽  
Zili He
Keyword(s):  
Sickle Cell Disease ◽  
Hospital Stay ◽  
Sickle Cell ◽  
Blood Viscosity ◽  
Exchange Transfusion ◽  
Hemoglobin Level ◽  
Therapeutic Modality ◽  
Acute Chest Syndrome ◽  
Cell Disease ◽  
One Year

Abstract Background: The pathophysiology of sickle cell disease (SCD) is based on increased blood viscosity due to abnormal red blood cells (RBCs), which causes SCD complications, such as chronic hemolytic anemia, vaso-occlusive crisis with tissue hypoxemia, and organ dysfunction. Effective treatment of Sickle Cell Anemia is to reduce the blood concentration of Hemoglobin S (Hb S) RBCs. Exchange transfusion (ET) remains an effective but possibly underutilized therapy for the management of various acute and chronic complications of SCD such as acute chest syndrome, thromboembolic stroke, splenic and hepatic infarction, right upper quadrant syndrome, multi-organ failure syndrome, or in preparation for surgery by reducing HbS to less than 30%. RBC ET quickly replaces abnormal RBCs with normal RBCs, thus improving oxygen transport while reducing overall blood viscosity. Methodology: To determine the effectiveness of ET in SCD in reducing the total number of admissions and total in-hospital Length of Stay (LOS) in patients, admitted with any of acute complications of SCD, we retrospectively reviewed the medical records of 38 patients between June 15, 2007 and June 15, 2008. The eligibility criteria were age above 18 years old and admission to the hospital for any SCD complication. Nineteen patients had ET with Hb A containing RBCs, generally with an average packed RBC exchange volume of 70–80 ml/kg patient’s weight. Nineteen patients were treated with conventional managements. Three patients were excluded from the ET group because of prolonged LOS due to non-SCD- related complications. Four patients in the non-Exchange group signed against medical advice on the first day of admission and were excluded from analysis. Results: Sixteen (42.1%) patients were male and 22 (57.9%) patients were female. Their ages ranged from 19 to 67 years old, mean (SD) 30.2 (10.8). Of eligible patients, 19 (50%) patients received at least one therapeutic ET during the one year period of the study. In an independent-Samples T test analysis, the mean (SD) LOS were 7.5 (0.6) and 4.2 (0.6) days for the groups without ET and with ET respectively (95% CI = −5.2 to 1.5, p=.0011) (Figure 1). In this small studied group, this resulted average 3.3 days shorter in-hospital stay in ET group, could have saved 62 in-hospital days in the group who received conventional treatments. This number could have been easily much greater, since our hospital’s electronically stored data revealed that during year 2007, there had been 278 SCD admissions in all age groups. There was no mortality in the ET group, nor were any transfusion-related complications reported. In another analysis of one year follow up data, the number of admissions for the patients who never received ET ranged from 1 to 14, mean (SD) 1.7 (2.3) times in year 2007, while in patients who received at least one ET, the number of following admissions ranged from 0 to 2, mean (SD) 0.7 (1.3) times during the next year (95% CI = 0.16 to 1.7, p= .020). Hemoglobin level of patients in conventional treatment group on the day of discharge ranged from 6.5 to 10.7, mean (SD) 8.9 (1.9) and in ET group it ranged from 8.4 to 12.4, mean (SD) 10.2 (1.2) gr/dl (p= .045). Conclusion: Patients with SCD are frequently admitted to hospital for vaso-occlusive crisis and other complications. Exchange transfusion is a reliable, safe, and effective therapeutic modality in SCD patients, in particular during a catastrophic event. ET can significantly reduce the number of hospital admissions and in-hospital stay days in these patients. In addition, patients managed with ET have a better hemoglobin level on discharge. Figure Figure

Arginine Therapy for Vaso-Occlusive Pain Episodes in Sickle Cell Disease.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 573-573 ◽  
Author(s):  
Claudia R. Morris ◽  
Michael Ansari ◽  
Lisa Lavrisha ◽  
Nancy Sweeters ◽  
Frans A Kuypers ◽  
...  
Keyword(s):  
Treatment Group ◽  
Sickle Cell Disease ◽  
Hospital Stay ◽  
Sickle Cell ◽  
Clinical Deterioration ◽  
Acute Chest Syndrome ◽  
No Production ◽  
Cell Disease ◽  
Unequal Variance ◽  
Significant Difference

Abstract Abstract 573 BACKGROUND: Vaso-occlusion is the principal factor in the morbidity of sickle cell disease (SCD). Vaso-occlusive painful episodes (VOE) are common, debilitating, and the leading cause of hospitalizations, emergency room visits, and are associated with an increased mortality rate. There is no effective therapy that targets the underlying mechanisms of VOE. Symptomatic relief with analgesics is the only availabel treatment. Nitric oxide (NO) is a potent vasodilator that contributes to a variety of vaso-occlusive events in SCD. We have found that an arginine deficiency and low NO bioavailability occurs during VOE in SCD. Since arginine is the obligate substrate for NO production, and an acute deficiency is associated with VOE, we hypothesized that arginine supplementation may be a safe and beneficial treatment for sickle cell pain. PATIENTS AND METHODS: Hospitalized SCD patients > 3 years diagnosed within 24 hours with VOE and without associated complications were eligible; written informed consent was obtained. A total of 56 patients completed randomization in this double-blinded, placebo controlled trial. A standardized treatment and monitoring program for VOE was followed. Average age was 13.9 ± 4 years (range 3.6-19 years), and 52% were female. Patients received intravenous (IV) or oral arginine (0.1 gram/kg TID, n=28) or placebo (n=28) for 5 days or until discharge from the hospital. Narcotic records for 2 patients (randomized to placebo arm) were incomplete and were not included in the narcotic use analysis. An intention to treat analysis was performed for narcotic use applying an unpaired t-test with Welch's correction to adjust for unequal variance. RESULTS: Age was equally distributed between treatment and placebo groups. 57% of the arginine treatment group and 46% of the placebo group were female. A significant reduction in narcotic use (defined as total morphine use over the course of the hospital stay in mg/kg) by 56% was observed in the treatment arm receiving IV or oral arginine compared to placebo (mean ± SEM: 1.8 ± 0.4 mg/kg; n=28 vs. 4.1 ± 0.8mg/kg; n=26, p=0.01). Average length of hospitalization was 4.5 ± 0.4 days, and there was no significant difference between the 2 groups (4.1 ± 0.3 vs. 4.8 ± 0.5 days, p = 0.27; arginine vs. placebo arm). Four episodes of acute chest syndrome (ACS) developed during the study, three in the treatment arm and one in the placebo arm. There was one patient who experienced clinical deterioration associated with ACS requiring emergent transfusion and a transfer to the pediatric intensive care unit (PICU) in the placebo arm. No clinical deterioration or PICU transfers occurred in the arginine arm. Five in the treatment arm received transfusion vs. four in the placebo arm. No drug-related adverse events were observed. No significant differences were observed between pre and post therapy liver or renal function, or hematological parameters in the arginine treatment group vs. placebo. Two patients admitted for pain management ultimately did not receive IV narcotics. Both had been randomized into the arginine-treatment arm and received arginine therapy per protocol throughout their hospital stay and required only oral narcotics and non-steroidal analgesia. Reduction in narcotic use in the treatment arm remained significant even when these 2 patients were excluded from the analysis (p=0.02). CONCLUSIONS: IV arginine therapy represents a novel nutritional intervention for the treatment of pain in hospitalized patients with SCD. Use of IV arginine should also be considered in the treatment of VOE in the emergency department setting prior to hospitalization, although further investigation is warranted. A reduction of narcotic use by over 50% observed in this study is remarkable, as this is the first successful intervention for sickle cell-related pain that targets the underlying mechanism of vaso-occlusion through a promising NO-based therapy. Arginine is a safe and inexpensive intervention with narcotic-sparing effects that should be considered as an adjunct to standard therapy for VOE requiring hospitalization. Disclosures: Off Label Use: Arginine for treatment of sickle cell vaso-occlusive pain episodes.

scholarly journals Comparison of Clinical Evolution of Children with Sickle Cell Disease before and after Treatment with Hydroxyurea at Saint Damien Hospital, Tabarre-Haiti,2013-2018

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 2306-2306
Author(s):  
Nora St Victor Dély ◽  
Ofelia A. Alvarez ◽  
Vanessa J Dor ◽  
Emmeline Lerebours
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Clinical Course ◽  
Acute Chest Syndrome ◽  
Blood Transfusions ◽  
Cell Disease ◽  
Clinical Evolution ◽  
Before And After ◽  
The Mean ◽  
Painful Crisis

INTRODUCTION Sickle cell disease (SCD), an autosomal recessive hemoglobinopathy, is associated with a high morbidity and mortality rate, especially in low income countries. In Africa, 5% of deaths among children under five are attributable to SCD [59th World Health Assembly, WHO 2006]. This chronic disease greatly alters the quality of life of affected children. However, according to several published studies, SCD clinical course can be improved with the administration of hydroxyurea, an antimetabolite drug. [Nkashama, Pan African Medical Journal,2015] Saint-Damien, a pediatric hospital in Haiti, has a current cohort of 1248 sickle cell children. Forty of them (3 %) benefit from hydroxyurea administration since November 2015. In this hospital, data on how hydroxyurea modifies SCD clinical course are lacking, despite the advantage of this drug described in literature [Charache,New England Journal of Medicine,1995]. This study aims to compare the evolution of children treated at Saint Damien Hospital, before and after receiving hydroxyurea. METHODS A retrospective analytic study was conducted from November 2013 to June 2018 in the Sickle Cell Clinic at Saint-Damien Hospital. We included 40 children aged 2 to 15 years old treated with hydroxyurea. All of them benefit of the same treatment protocol: Initial dose of 10 mg per kg per day increase to maintenance dose of 25 mg per kg per day. Any child whose treatment has been permanently discontinued regardless of the cause was excluded. Epidemiological and clinical data were collected using Excel 2010. We compared children clinical evolution two years before and two years after hydroxyurea administration using these parameters: frequency and duration of hospitalizations, hospitalization frequency for specific complications (pain crisis, stroke and acute chest syndrome), and frequency of blood transfusions. We calculated frequencies, ratios and means using Epi Info. We realized statistical analysis to compare quantitative variables with a p value significant when less than 5%. RESULTS Gender ratio was 1:1. The mean age of children at enrollment on hydroxyurea was 8 years. Thirty-eight children of 40 (95 %) experienced at least one hospitalization before receiving the drug, compared with 17 (42.5%) after, p=0.025. The mean duration of hospitalization was 9 days before and 6 days after, p=0.0319. The average number of hospitalizations per child was decreased by 30 %. Seventy percent of children were hospitalized at least once due to painful crisis 2 years before receiving hydroxyurea, compare to 22.5 % after. Thirty-one children (77.5%) were transfused at least once before receiving the drug and 9 (22.5%) after receiving it. There was no cases of acute chest syndrome or stroke reported after hydroxyurea, unlike before the introduction of the drug. (Table 1) CONCLUSION The percentage of hospitalized children and the average length of hospitalization stay decreased significantly with hydroxyurea intake; as well as the frequency of painful crisis and blood transfusions. Hydroxyurea acts directly on the two main causes of hospitalization in the sickle cell, reducing the morbidity related to this pathology; and demonstrating the direct benefit of this drug at Saint Damien Hospital. Since our cohort is young, we have not been able to follow his evolution over a longer period of time. We plan to continue to observe this cohort. But these first results already allow us to recommend a broader use of hydroxyurea for pediatric patients with SCD in Haiti. Disclosures Alvarez: Forma Therapeutics: Consultancy; Novartis: Consultancy.

The Use of Chronic Transfusions in Sickle Cell Disease for Non-Stroke Related Indications

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 4934-4934 ◽  
Author(s):  
Lindsay Mize ◽  
Shelly Burgett ◽  
Julia Xu ◽  
Jennifer Rothman ◽  
Nirmish Shah
Keyword(s):  
Sickle Cell Disease ◽  
Acute Care ◽  
Sickle Cell ◽  
Emergency Department Visits ◽  
Acute Chest Syndrome ◽  
Blood Transfusions ◽  
Recurrent Pain ◽  
Cell Disease ◽  
Pain Crisis

Abstract Introduction: Sickle cell disease (SCD) is a chronic disease that can cause significant complications including acute chest syndrome, recurrent pain and stroke. Current guidelines for the use of chronic transfusions include primary and secondary prevention of stroke. Although there is currently limited support for the routine use of transfusions for acute vaso-occlusive crisis (VOC), there has been increasing use of chronic transfusions as an alternative treatment for recurrent VOC. Moreover, there is evidence that patients on chronic transfusions have less VOC. We sought to review the outcomes of patients at our institution placed on chronic transfusions for non-stroke related indications. Methods: We performed a retrospective cohort study to summarize clinical and nonclinical features of sickle cell patients on transfusions for non-stroke related complications. Demographic, clinical, and laboratory information were summarized. Acute care events per month were calculated for both the year prior and up to one year following initiation of chronic transfusions. Acute care events were defined as emergency department visits or hospitalization. Results: Of the 378 patients with SCD treated in the pediatric specialty clinic, there were 21 patients being either chronically transfused or exchange transfused. Six (20%) of these patients were initiated on chronic blood transfusions (CBT) for recurrent pain crisis (median age = 12, range 8 to 17). One of these patients also had suspected hepatic sequestration. All patients were type SS and had been treated with hydroxyurea (HU) for an average length of 6.5 years (range 1 to 12 years) at a mean dose of 25 mg/kg (SD 4) prior to initiation of CBT. All patients continued on HU during chronic blood transfusions. Patients were on chronic transfusions for a median of 11 months (range 3 to 58 months) with mean %S while on transfusions of 39.6% (SD 10). Patients were transfused on average every 5 weeks (range 4 to 6 weeks). Following initiation of transfusions, 50% were started on chelation based on criteria of having a ferritin >1000 ng/mL. Mean peak ferritin was not significantly increased in the year following the start of CBT (513 ng/mL ± 343 vs. 1260 ± 934, p=0.13). There was one new alloantibodies (anti-Jk) reported following initiation of CBT, which developed within 3 months. Acute care visits per month were significantly higher in the year prior as compared to after initiation of chronic blood transfusions (1.04 ± 0.45 vs. 0.28 ± 0.22, respectively; p=0.006) (Figure 1). Discussion: We found that patients started on chronic transfusions for pain crisis had a non-significant increase in peak ferritin and a significant reduction in acute care visits. Prior to CBT, all patients had been initiated on hydroxurea (mean dose of 25mg/kg) in an attempt to treat recurrent VOC. However, following therapy for an average of 6.5 years, patients were placed on CBT to prevent further acute care visits and reduce morbidity. All patients were continued on HU while on CBT with no dose adjustment or effort to titrate to maximum tolerated dose. While on CBT, patients had a mean %S of 40%, which is higher than the recommended goal of 30% for stroke related indications. Importantly, despite the higher mean %S, there was a drastic and significant decrease in acute care visits. It should be noted that although only three patients (50%) of patients were placed on chelation, the remaining three had been on transfusions for less than or equal to 6 months and likely to require chelation with continued therapy. The expected elevated ferritin highlights the difficulty in long-term treatment with chronic transfusion and risk for eventual iron overload. The balance between the clinical benefit and potential long-term complications leads to individual assessment of the risks and benefits prior to initiation of chronic transfusions for VOC. These results advocate for the use of prospective studies to evaluate the role for chronic transfusions for non-stroke related indications in SCD. Figure 1 Figure 1. Disclosures Shah: Novartis: Speakers Bureau.

Blood transfusions for treating acute chest syndrome in people with sickle cell disease

2009 ◽  
Author(s):  
Dunia Alhashimi ◽  
Fatima Alhashimi ◽  
Saeed Dastgiri ◽  
Zbys Fedorowicz ◽  
Mona Nasser
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Acute Chest Syndrome ◽  
Blood Transfusions ◽  
Cell Disease

The Outcome Of Preoperative Transfusion Guideline On Sickle Cell Disease Patients At King Fahd Hospital-Jeddah (Ksa)

2020 ◽  
Vol 154 (Supplement_1) ◽  
pp. S111-S111
Author(s):  
S M Felemban
Keyword(s):  
Postoperative Complications ◽  
Sickle Cell Disease ◽  
Hospital Stay ◽  
Sickle Cell ◽  
Exchange Transfusion ◽  
Length Of Hospital Stay ◽  
Acute Chest Syndrome ◽  
Cell Disease ◽  
Postoperative Fever ◽  
Significant Difference

Abstract Introduction/Objective We have developed a local hospital transfusion guidelines to reduce and prevent the perioperative and the postoperative complications. This study was conducted to evaluate the outcome of practice for preoperative transfusion therapy in SCD patients in our institution. Methods A retrospective review of SCD patients undergoing surgery at King Fahd Hospital, Jeddah, Saudi Arabia was conducted between April 2005 and May 2010. The medical records were reviewed to define the perioperative risks and the postoperative complications in relation to the type of transfusion modality selected. Results The medical record of 75 sickle cell disease patients in whom the hematologists were consulted for their preoperative assessment. Preoperatively, 25.3 % had complete exchange transfusion (CETX), 17.3 % had partial exchange transfusion (PETX), 26.7 % had simple top up transfusion (STX) and 30.7 % had no transfusion (NTX). The postoperative complications were 20% vaso-occlusive crises (VOC), 2.7% acute chest syndrome (ACS), and 16% had fever. The mean duration for the hospital stay was 7.36 with SD of 5.83. There was no significant difference in the outcome between all types of transfusion modalities, the P value was (p 0.245), (p 0.282), (p 0.133), (p 0.220) for postoperative fever, VOC, ACS, and the length of hospital stay, respectively. However, The correlation was highly significant between the post-transfusion haemoglobin (Hb) level and the occurrence of postoperative fever (p 0.01) and VOC (p 0.03). Interestingly, SCD patients who received hydroxyurea were observed to have less postoperative complication like fever, and the result was highly significant (P&lt;0.01), while those who received prophylactic heparin in the postoperative period were found to have a reduced length of hospital stay (p&lt;0.01) and vaso-occlusive crises (p &lt;0.01). Conclusion The guidelines for preoperative transfusion in SCD patients was effective in reducing the postoperative morbidity and mortality. However, the selection of the optimum regimen for different surgical types in sickle cell disease patients and the surgical situations where preoperative transfusion is needed were all established in this guidelines.

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