The Eveluation of Pulmonary Functions in Children with Sickle Cell Anemia.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 4628-4628
Author(s):  
Cem Kurt ◽  
Ilgen Sasmaz ◽  
Bulent Antmen ◽  
Yurdanur Kilinc ◽  
Sadi Kurdak ◽  
...  
Keyword(s):  
Patient Group ◽  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Exercise Test ◽  
Acute Chest Syndrome ◽  
Control Group ◽  
Healthy Children ◽  
Diffusion Test ◽  
Pulmonary Functions

Abstract Abstract 4628 Aim In this study we evaluated to pulmonary functions and determined relations of these findings with clinical parameters in children with sickle cell anemia (SCA) who were at follow up in our pediatric heamatology clinic. Materials and Methods 24 children with sickle cell anemia and 9 healthy children as control group include to the study. Complete blood count, hemoglobin electrophoresis and biochemical values were eveluated for both groups. At pulmonology department, the carbonmonoxide diffusion test performing for both groups. At the same day spirometric respiratory function evaluation and exercise test performed both groups at department of sports physiology. The data recieved are compared statistics. Results HbS, HbF, SGPT, ferritin, total bilirubine, direkt bilirubine and Fe++ values were high at patient group (p<0.05). Hemoglobin and hematocrit values were low at patient group according to control group as expected (p<0.05). The number of SCA patient who had one-three venoocclusive crises (VOC) were 14 (58.3%), patient who had three or more VOC were 7 (29.2%) and patient who had no VOC were 3 (12.5%). The number of patient who had acute chest syndrome (ACS) were 5 (20.9%) and 19 patients had no ACS (79.1%). Ýmpaired isole carbonmonoxide diffusion test was established at the 62.5% of the patient's. At patient group, spirometric FEV1 and MEF25 measurement were found lower than the control group (p<0.05). At exercise test VO2/HR rate were lower for patient group (p<0.05). VE/VO2 rate (p=0.023) and R (p=0.016) measurement were found higher. Conclusion Pulmonary gas transfer was found difficult in patients with SCA. Respiratory airways established obstructed in spirometric evaluation. Obstructive defficiensies have to be follow up. Oxygen pulse and respiratory exchange rates were determined low and more oxygen usage was observed for aerobic metabolic activity. With these results, ýt can be say that chronic inflamation process at lung due to oxygen radicals and hipoksemia in sickle cell patients, the aerobic respiratory load was increased. Disclosures: No relevant conflicts of interest to declare.

scholarly journals Study of transcranial Doppler ultrasound and endothelin-1 in children with sickle cell anemia: a single-center study

2021 ◽  
Vol 20 (3) ◽  
pp. 31-35
Author(s):  
S. Ragab ◽  
E. Badr ◽  
H. El-Kholy ◽  
M. El-Hawy
Keyword(s):  
High Risk ◽  
Patient Group ◽  
Sickle Cell ◽  
Serum Ferritin ◽  
Sickle Cell Anemia ◽  
Transcranial Doppler ◽  
Control Group ◽  
Healthy Children ◽  
Endothelin 1 ◽  
The Impact

To assess the impact of our transcranial Doppler (TCD) screening program on the incidence of a first stroke in children with sickle cell anemia and to study the role of elevated serum endothelin-1 (an inflammatory mediator) in these children. Background: stroke is a major complication of sickle cell disease (SCD), even in very young children. About 11% of children with homozygous sickle cell anemia (SS) develop stroke by the end of the second decade of life. The underlying etiology in most cases is an ischemic stroke caused by large-vessel stenosis or occlusion. Transcranial Doppler (TCD) recommended as a routine screening test to identify children at high risk of developing a stroke, measures flow velocities within large intracranial arteries. TCD should be routinely performed in children between 2 and 16 years as this age group is at the highest risk of sickle cerebral vasculopathy. We carried out a prospective case-control study which included 2 groups: a patient group consisted of 30 children with sickle cell anemia and sickle thalassemia and a group of 30 healthy children of matched age and sex. Each group included 11 males (36.5%) and 19 females (63.5%); the age range was 2 to 17 years. Both groups underwent a thorough clinical examination and laboratory tests (CBC, liver and renal function, serum ferritin and endothelin-1). Additionally, TCD was performed in all children included in the patient group. According to the results of TCD, time-averaged mean of the maximum velocity (TAMMX) was < 170 cm/s (normal), 170–200 cm/s (conditional), ≥ 200 cm/s (high risk) in 20 (66.7%), 4 (13.3%) 6 (20%) patients, respectively. The level of endothelin-1 was significantly higher in the patients (57.1 ± 91.3) than in the controls (21.9 ± 14.8). Hemoglobin concentration was significantly lower in the patient group than in the control group, but the levels of reticulocytes, WBCs and serum ferritin were significantly higher in the patients than in the healthy controls. Serum Endotheline-1 level was higher in patients with sickle cell anemia than control group.

scholarly journals Up-Regulation of Mir-21 and Mir-130a and Serum Leptin Levels on Leg Ulcers Development in Sickle Cell Anemia

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 975-975
Author(s):  
Thais Helena Chaves Batista ◽  
Rodrigo Marcionilo Santana ◽  
Marcondes José de Vasconcelos Costa Sobreira ◽  
Gabriela da Silva Arcanjo ◽  
Diego Arruda Falcao ◽  
...  
Keyword(s):  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Leg Ulcer ◽  
Acute Chest Syndrome ◽  
Control Group ◽  
Blood Collection ◽  
Leg Ulcers ◽  
Serum Leptin ◽  
Ulcer Group ◽  
And Control

Introduction: Leg ulcers (LUs) are a cutaneous complication of sickle cell anemia (SCA), whose etiology is considered multifactorial. In the search for new candidates for modulators of SCA clinical events, recent evidence suggests the significant role of mechanisms related to post-transcriptional regulation, especially microRNAs (miRNAs). Thus, the analysis of miRNAs miR-21 and miR-130a differential expression in patients with SCA becomes an interesting approach, since both act in the regulation of several biological mechanisms related to the pathophysiology of LU, especially the tissue repair process. In addition, these miRNAs have already been related to the regulation of serum leptin levels, a strong angiogenic pleiotropic hormone that acts in the healing process of skin lesions. Therefore, the aim of the study was to investigate the influence of miR-21 and miR-130a and serum leptin levels on the development of LUs in SCA patients. Methods: After analyzing medical records, 60 SCA patients were selected. Patients who presented some of the main clinical manifestations that may have etiology due to the underlying disease (for example: osteonecrosis, stroke, priapism and acute chest syndrome) were not included. Patients with a history of LU were considered cases, and those who did not develop this complication (n=20), were considered control (median age: 26 years, range: 19-61, 50% males). The control group was called "HbSS-Control" and the case group was divided into two subgroups: Active leg ulcer group, composed of 19 patients with active LU at the time of blood collection (median age: 35 years, range: 24-56, 68% males), and healed leg ulcer group, composed of 21 patients with healed LU at the time of blood collection (median age: 34 years, range: 22-52, 43% males). In addition, it was analyzed a group of 10 donors with normal hemoglobin profile (median age: 25 years, range: 20-30, 50% males), identified as "HbAA-Control". Expression levels of miRNAs extracted from peripheral blood, using mirVanaTM PARIS Kit (Invitrogen™) were evaluated by RT-qPCR technique utilizing TaqMan® probes. Serum leptin levels of the patients were evaluated employing the ELISA method (Human Leptin ELISA Kit, Millipore®). Mann-Whitney and Kruskal-Wallis tests were applied to compare continuous variables. Results: Up-regulation of both miRNAs was observed in the active leg ulcer group in contrast to the healed leg ulcer (miR-21: P&lt;0.0001, Figure 1A, Fold change [FC]=14,2; miR-130a: P=0.0004, FC=18,8, Figure 1B) and Control-HbSS groups (miR-21: P&lt;0.0001, FC=34,4, Figure 1A; miR-130a: P=0.0006, FC=15,3, Figure 1B) and the HbAA-Control group (miR-21: P&lt;0.0001, FC=5,8, Figure 1C; miR-130a: P=0.0009, FC=10,9, Figure 1D). However, there was no significant difference between the healed leg ulcer, HbSS-Control and HbAA-Control groups (miR-21: P=0.1829, Figure 1E; miR-130a: P=0.3537; Figure 1F). Furthermore, the active leg ulcer group had lower serum leptin levels when compared to the healed leg ulcer and Control-HbSS groups (P=0.0058; Figure 2A). The levels of leptin in the healed leg ulcer group did not differ from the Control-HbSS group (P=0.5929; Figure 2B). Conclusion: Our results demonstrated an inverse relation between the miRNAs miR-21 and miR-130a expression with serum leptin levels, suggesting that the up-regulation of these miRNAS may be related to the chronicity and healing of LUs in individuals with SCA through decreased of serum leptin levels. Disclosures No relevant conflicts of interest to declare.

Stroke in Sickle Cell Anemia After Excellent Response to Hydroxyurea.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 4622-4622
Author(s):  
Ubaldo Martinez ◽  
Samir K. Ballas
Keyword(s):  
Atrial Septal Defect ◽  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Septal Defect ◽  
Acute Chest Syndrome ◽  
Excellent Response ◽  
Blood Exchange ◽  
Atrial Shunt ◽  
The Right

Abstract Abstract 4622 Introduction Strokes occur in about 10% of children with sickle cell anemia (SS) less than 10 years old. These strokes are usually ischemic in nature. Stroke due to SS in adults is less common and is usually hemorrhagic in nature. We wish to report an unusual adult patient with SS and excellent response to HU who developed a stroke that was unrelated to SS. Case Report and Results A 35-year-old female with sickle cell anemia had mitral valve prolapse and migraine headaches presented 2 days after developing abrupt bilateral blurry vision, left facial numbness and weakness of her left leg. Her SS has been complicated by acute chest syndrome, bilateral hip avascular necrosis and frequent painful crises prior to hydroxyurea (HU) therapy. She was enrolled in the multicenter study of hydroxyurea (MSH) in SS and has been on 2500mg of HU per day for the past 13 years. She had an excellent response to HU with no recurrent acute chest syndrome and decreased need for blood transfusion. After starting HU, the frequency of crises requiring hospital admission decreased from 1 admission every 1 to 2 months to less than 1 admission per year except when hydroxyurea was discontinued for pregnancy. Her fetal hemoglobin increased from 6.1 % before HU to a maximum of 45%. Her MCV increased from 96 fl to a maximum of 132 fl and Hb from 8.0 g/dl to 9.8 g/dl Her exam was remarkable for left lower extremity weakness which was more pronounced proximally. All cranial nerves were intact and there was normal sensation bilaterally. CT scan of the brain showed three foci of hypodensity and MRI of the brain showed increased signal on T2, FLAIR and diffusion weighted images within the frontoparietal deep white matter consistent with infarction in the border zone of the middle cerebral artery (MCA)-anterior cerebral artery (ACA). MR angiography of the intracerebral and extracerebral vessels demonstrated focal narrowing of the right MCA at the trifurcation suggesting an embolic cause. Common causes of stroke were ruled out with routine studies. Her hemoglobin electrophoresis after admission but before blood exchange transfusion showed HbS of 55% and HbF of 45%. She underwent exchange transfusion 2 days after admission and was started on chronic blood exchange transfusions with the assumption that she had ischemic stroke due to SS. Initial transthoracic echocardiogram with contrast injection did not show an atrial shunt. Follow-up transesophageal echocardiogram after discharge showed a secundum atrial septal defect with a defect size of 1.4 cm. Right heart catheterization was performed and the pulmonary flow to systemic flow (Qp/Qs) was 1.7:1. An Amplatzer atrial septal defect (ASD) closure device was deployed with transesophageal echocardiographic guidance and a large thrombus was removed from the right atrium. At the patient's request exchange red cell transfusions were discontinued. The patient has continued treatment with hydroxyurea and aspirin. Conclusions Young patients with cryptogenic stroke have a much higher prevalence of atrial shunts and in particular patent foramen ovale than patients with other forms of stroke and therefore a cause-effect association is suggested. Young adults with stroke should be evaluated for common and reversible causes of stroke including paroxysmal emboli. Transesophageal echocardiography is the gold standard for diagnosing atrial shunts. Strokes in patients younger than age 55 are related to paroxysmal emboli and have a risk of recurrence of approximately 30% within one year. High risk features for stroke recurrence with an atrial shunt include hypercoagulable states, large opening and presence of an atrial septal aneurysm. Optimal management of patients with a stroke and an atrial shunt is unknown. Options include surgical closure, percutaneous device closure, anticoagulation and antiplatelet therapy. Patients with sickle cell disease and stroke should receive long term blood transfusions to reduce HbS below 30% if the stroke is felt to be related to sickle cell disease vasculopathy. The patient described with SS had a stroke and had an atrial septal defect that was repaired. The MRI/MRA findings are consistent with paroxysmal emboli. The patient is receiving treatment with hydroxyurea and aspirin having discontinued red cell exchange transfusions and at two years of follow-up has not had a recurrent stroke. Disclosures: No relevant conflicts of interest to declare.

scholarly journals Elevated End-Tidal Carbon Monoxide Concentration in Children with Sickle Cell Anemia

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 1390-1390
Author(s):  
Ashutosh Lal ◽  
Kristen Yen ◽  
Lasandra Patterson ◽  
Alisa Goldrich ◽  
Anne M Marsh ◽  
...  
Keyword(s):  
Carbon Monoxide ◽  
Young Children ◽  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Second Hand Smoke ◽  
Control Group ◽  
Exhaled Breath ◽  
Healthy Children ◽  
The Mean ◽  
End Tidal

Abstract Background: Carbon monoxide (CO) produced during oxygen-dependent cleavage of porphyrin ring of heme is excreted in exhaled breath. The catabolism of heme is increased when red blood cells are destroyed at an accelerated rate. Thus, quantifying CO in exhaled breath could serve as an indicator of hemolysis. However, the requirement for forced breath sample has limited the measurement of exhaled CO in young children. Objective: To assess end-tidal CO concentration (ETCOc) in children with sickle cell anemia (SCA). Design/Methods: ETCOc was measured using the CoSense ETCO Monitor (Capnia Inc. Palo Alto, CA). Children between 5-14 years with SCA (Hb SS) who were not on chronic transfusions were eligible. Healthy children served as age-matched controls. Children with exposure to second-hand smoke, acute respiratory infection or symptomatic asthma were excluded. End-tidal breath samples were collected by placing the tip of a nasal cannula 5 mm into the nares. Up to 3 measurements were taken for each subject and the highest ETCOc value was used for analysis. (ClinicalTrials.gov: NCT01848691) Results: The mean (range) age of 16 children with SCA and 16 controls was 9.7 years (5-14 years) and 9.9 years (5-14 years), respectively. The mean (± s.d.) ETCOc for SCA was 4.85 ± 2.24 ppm versus 0.96 ± 0.54 ppm for control group (p<0.001). The ETCOc in the control group ranged from 0.2 to 2.3 ppm, but was ≤1.2 ppm in 14/16, which is suggested as the upper limit of normal for healthy children. In the SCA group, the ETCOc range was 1.8 to 9.7 ppm, with values ≥2.4 ppm in 15/16 subjects. A threshold ETCOc value of >2.1 ppm provided both sensitivity and specificity equal to 93.8% (69.8-99.8%) for distinguishing SCA from healthy children. Children with SCA who had higher absolute reticulocyte count also demonstrated higher ETCOc (r=0.62, p=0.011). Patients with severe anemia (hemoglobin <8 g/dL) had a higher mean ETCOc (5.43 ppm) than the rest (4.40 ppm) but the difference was not significant. ETCOc level tended to increase with age in SCA (r=0.45, p=0.08). Conclusions: Carbon monoxide in exhaled breath can be measured in young children in the clinic using a portable monitor. ETCOc may be a valuable tool for non-invasive monitoring of the severity of hemolysis in SCA. The mean ETCOc was 5-fold higher in SCA compared with controls, with little overlap seen between the groups. This suggests a potential use for ETCOc as a point-of-care screening test for SCA in children. Figure 1 Figure 1. Figure 2 Figure 2. Disclosures Lal: Capnia, Inc: Research Funding. Yen:Capnia, Inc. : Employment. Bhatnagar:Capnia, Inc: Employment.

Prognostic Significance of Early Vaso-Occlusive Complications in Children Who Have Sickle Cell Anemia.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 3176-3176
Author(s):  
Charles T. Quinn ◽  
Elizabeth P. Shull ◽  
Naveed Ahmad ◽  
Zora R. Rogers ◽  
George R. Buchanan
Keyword(s):  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Strong Predictor ◽  
Prognostic Significance ◽  
Adverse Outcomes ◽  
Acute Chest Syndrome ◽  
Disease Modifying Therapy ◽  
Pain Crisis ◽  
Disease Modifying

Abstract Sickle cell anemia (SS) is a phenotypically variable disease whose course is difficult to predict. The Cooperative Study of Sickle Cell Disease (CSSCD) found that dactylitis in the 1st year of life predicted adverse outcomes in later childhood. We aimed to determine whether early vaso-occlusive complications, including dactylitis, were prognostic in the Dallas Newborn Cohort. We studied all cohort members with SS or sickle-β0-thalassemia who were &lt;1 yr of age at their first clinic visit, ≥5 yrs of age at last follow-up, and who had complete records. We defined 3 potential “early” (occurring in the first 3 yrs of life) predictors: any hospitalization for (1) pain crisis (non-dactylitis), (2) dactylitis, or (3) acute chest syndrome (ACS). We studied the associations of these predictors with the following “late” (occurring on or after the 3rd birthday) outcomes: death of any cause; overt stroke; use of hydroxyurea (HU), chronic transfusion (CT), or stem cell transplantation (SCT); and mean number of hospitalizations for late pain crisis and ACS. Late pain and ACS episodes were enumerated for each patient between the 3rd birthday and the last clinical encounter or the start date of a disease-modifying therapy (HU, CT, or SCT), whichever was first. Mean number of pain and ACS events was analyzed for the late follow-up period in total and in 2-yr intervals. Outcomes up to age 20 were included. Two-sided Fisher exact and t-tests were used appropriately. There were 264 subjects (256 SS; 54.9% male). Mean age at first visit was 4.1±2.3 mos (±S.D.) and mean follow-up was 12.1±4.3 yrs. The following early hospitalizations occurred: 53 subjects (20.1%) had pain crisis; 16 (6.1%) had dactylitis, and 85 (32.9%) had ACS. There were 5 deaths and 30 overt strokes. Sixty-six subjects were treated with HU (37), CT (40), and/or SCT (1). We found that subjects who had early pain, dactylitis, or ACS (compared with those who did not) were not more likely to die (1.7 vs. 2.1%; P&gt;0.99) or have a stroke (12.2 vs. 10.3%; P=0.69). However, the use of a disease-modifying therapy was more common among subjects who had early pain (37.7 vs. 19.9%; P=0.01) and ACS (37.6 vs. 16.2%; P&lt;0.001), but not dactylitis (18.8 vs. 23.6%; P&gt;0.99). This prediction held only for HU use when the treatments (HU, CT, or SCT) were analyzed separately. Subjects who experienced early pain or ACS had on average a 2.2-fold (P=0.02) or 2.1-fold (P=0.01), respectively, higher number of late pain crises between ages 3 and 11, but not beyond (all P&gt;0.05). Dactylitis did not predict a higher number of late painful events at any age (all P&gt;0.05). Likewise, neither early pain nor dactylitis was associated with a higher number of hospitalizations for late ACS (all P&gt;0.05). However, subjects who had early ACS had a 1.7 to 3.6-fold higher mean number of ACS events throughout all late age groups (all P&lt;0.05). In summary, early hospitalization for pain, dactylitis, or ACS did not predict death or stroke. Early pain and ACS were associated with use of HU in later childhood, but not CT or SCT. Early pain and ACS predicted an increased number of hospitalizations for pain until age 11, but not beyond. Early ACS was a strong predictor of recurrent ACS throughout childhood. Notably, we found that hospitalization for dactylitis had no particular prognostic significance, unlike the CSSCD. In conclusion, the prognostic significance of early vaso-occlusive complications is limited.

Asthma Is Associated with Acute Chest Syndrome, but Not an Increase Rate of Hospitalization for Pain among Children with Sickle Cell Anemia: A Retrospective Cohort Study.

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 3406-3406
Author(s):  
Francoise Bernaudin ◽  
Robert C. Strunk ◽  
Annie Kamdem ◽  
Cecile Arnaud ◽  
Ping An ◽  
...  
Keyword(s):  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Therapeutic Intervention ◽  
Retrospective Cohort ◽  
Inhaled Corticosteroids ◽  
Medical Center ◽  
Acute Chest Syndrome ◽  
Thoracic Pain ◽  
Transfusion Therapy

Abstract Introduction In this study, we sought to validate the previous findings that asthma increases the incidence of acute chest syndrome (ACS) and pain in children with sickle cell anemia (SCA). Methods A retrospective cohort was comprised of children with SCA evaluated for at least six months from a single medical center. Asthma was defined as being present when the first wheezing episode was heard by a physician after the age of 2 years or after 3 episodes of bronchiolitis before the age of 2 years. ACS was defined as a recent abnormal X-ray of the lungs associated with fever, respiratory signs or thoracic pain. A painful episode was defined as pain that resulted in hospitalization. Therapeutic intervention (hydroxyurea, blood transfusion therapy or transplant) was uniformly applied to all children with three or more episodes of pain that required hospitalization within a 12 month period. Patient years were accumulated from birth until death, lost to follow up, last visit to the center or a therapeutic intervention, whichever came first. Results A total of 297 children with SCA were evaluable for a doctor diagnosis of asthma for a total of 1,805 patient-years. The mean length of follow-up was 6.1 patient-years. A doctor diagnosis of asthma was present in 8.4% (25 of 297). Among the children with asthma 75% (19 of 25) were consistently prescribed a beta 2 agonist or inhaled corticosteroids. After adjustment for the effect of age, asthma was significantly associated with ACS event (p = 0.03) but pain was not (>0.05). Conclusion Among children with SCA, asthma is associated with an increased incidence of ACS, but not pain that required hositalization. The absence of an association between asthma and pain may be related to uniform therapeutic intervention for children with repetitive painful episodes that require hospitalizations coupled with active treatment for asthma in most of the children.

Hydroxyurea Treatment of Young Children with Sickle Cell Anemia: Safety and Efficacy of Continued Treatment – the BABY HUG Follow-up Study

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 7-7 ◽  
Author(s):  
Zora R. Rogers ◽  
Billie Fish ◽  
Zhaoyu Luo ◽  
Rathi V. Iyer ◽  
Courtney D. Thornburg ◽  
...  
Keyword(s):  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Hospital Admissions ◽  
Controlled Trial ◽  
Laboratory Data ◽  
Acute Chest Syndrome ◽  
Follow Up Study ◽  
Hydroxyurea Treatment ◽  
The Impact

Abstract Abstract 7 BABY HUG [Clinical Trials #NCT00006400], an NIH-NICHD sponsored randomized placebo-controlled trial showed that hydroxyurea (HU) administered to 9–18 month old children with sickle cell anemia (SCA) provides substantial clinical benefit. Benefits include a decrease in pain crises, acute chest syndrome events, need for transfusion and hospital admission; hematologic improvement include higher total and fetal hemoglobin concentration, larger red cell size, and lower WBC counts with toxicity limited to transient reduction in absolute neutrophil count (ANC) [Lancet 2011; 377:1663–72]. The parent or guardian of all 176 children who completed at least 18 months of randomized treatment were offered participation in an initial observational BABY HUG Follow-Up Study and 163 (93%) consented to participate. Clinical and laboratory data were collected every 6 months by structured abstraction of the medical record regarding use of clinically prescribed HU (dose escalation recommended), blood counts, clinical imaging, and sickle cell-related events. At the time of enrollment the family did not know their child's randomized study treatment assignment; 133 (82%) initially chose clinical prescription of open-label HU. Acceptance of HU has remained high through 36 months of follow-up; during each 6 month data collection period 68–75% of participants reported having taken HU. Only 2 patients have left the study (due to relocation) and more than 93% of expected data have been collected. Preliminary analyses as of May 2011, including 417 patient years (pt-yrs) of follow up, demonstrate that in comparison to participants not taking HU, children who continue to take HU have statistically lower rates of pain crises requiring emergency department (ED) visits, episodic transfusions, and hospital admissions for any reason, including acute chest syndrome or febrile illness (see table). The substantial decrease in acute chest syndrome episodes is similar to the effect demonstrated with HU use in the randomized BABY HUG trial in younger infants and consistent with published trials detailing the benefit of HU therapy in older children and adults. The decrease in the rate of admission for febrile events in HU-treated patients is also comparable to that in the randomized trial, but the reason for this benefit is uncertain. There was no difference in hospitalization rates for painful events including dactylitis. Two patients in the non-HU group had a stroke. There were no differences between groups in the frequency of a palpable spleen or rate of acute splenic sequestration crises. Through 36 months of follow up children taking HU had persistently higher hemoglobin and MCV, and lower WBC and ANC than those not taking HU. Results of these analyses including growth and development assessments will enhance our understanding of the impact of HU use in children with SCA starting at a very young age. The accruing data from the BABY HUG Follow-Up Study demonstrate a continuation of the substantial benefits of early HU therapy with no discernable additional toxicities. Ongoing follow up of this cohort is essential to fully define these benefits as children grow, and to observe for late toxicity.Event Rate per 100 pt-yrsHUNo HUp valueED visit for Pain Crisis28.853.60.004Episodic Transfusion18.334.00.010Hospital Admission (any cause)74.9133.20.001Acute Chest Syndrome (admission)9.522.30.0001Febrile Illness (admission)30.764.3<.001Pain Crisis (admission)18.630.40.102 Disclosures: Off Label Use: Hydroxyurea is not indicated for treatment of children with sickle cell disease. Use of this medication was for clinical indications and not mandated by this observational study.

scholarly journals Evaluation of Oxidative Stress Biomarkers in Patients with Henoch-Schönlein Purpura

Folia Medica ◽  
2021 ◽  
Vol 63 (6) ◽  
pp. 928-931
Author(s):  
Kadir Soylemez ◽  
Fatih Temiz ◽  
Tahir Dalkiran ◽  
Yasar Kandur ◽  
Ergul Belge Kurutas ◽  
...  
Keyword(s):  
Oxidative Stress ◽  
Patient Group ◽  
Statistical Significance ◽  
Control Group ◽  
Healthy Children ◽  
Oxidative Stress Biomarkers ◽  
Systemic Involvement ◽  
The Mean

Introduction: Henoch-Sch&ouml;nlein Purpura (HSP) is a systemic vasculitic syndrome characterized by non-thrombocytopenic purpura, arthritis/arthralgia, abdominal pain, and glomerulonephritis. The pathogenesis of HSP has not been clearly identified. Oxidative damage has a role in the pathogenesis of most cases. Aim: This study aimed to evaluate changes of oxidative stress by studying parameters like superoxide dismutase (SOD), catalase (CAT), and malondialdehyde (MDA) in an attempt to identify the role of oxidative stress in HSP from another perspective. Materials and methods: This study enrolled 23 pediatric patients (ten girls and thirteen boys) diagnosed with HSP who were under follow-up at Sutcu Imam University School of Medicine Department of Pediatrics between 2014 and 2016 and twenty healthy children as the control group. The parents of all subjects gave informed consent to participate in the study. In the HSP group, the beginning season of the illness and the systemic involvement during follow-up were determined. Blood specimens were obtained at presentation before any treatment was started. SOD, CAT activities, and MDA values in erythrocyte and plasma samples were compared between the patient group and the healthy children. Results: Twenty-three patients with HSP (13 males, 10 females) and 20 healthy children participated in this study. The mean age of the HSP cases was 8.21&plusmn;3.78 years (range 2-16 years) and of the controls was 8.6&plusmn;4.2 (range 3-14 years). The mean MDA value was 2.95&plusmn;0.71 nmol/ml in the patient group and 2.67&plusmn;0.66 nmol/ml in the control group (p=0.787). The mean level of the CAT enzyme was 1.32&plusmn;0.35 U/g Hb in the patient group and 7.8&plusmn;1.74 U/g Hb in the control group (p=0.001). The mean levels of the SOD enzyme were 3.06&plusmn;0.85 U/g Hb in the patient group and 0.97&plusmn;0.36 U/g Hb in the control group (p=0.001). Conclusions: Although high MDA levels support the role of lipid peroxidation in the pathogenesis of HSP, statistical significance was not reached owing to a limited number of our patients. The reduced CAT enzyme activity is consistent with the findings of previous reports. This finding supports the notion that oxidative stress can play a role in the pathogenesis of HSP. Keypoints: Our findings support the notion that oxidative stress can play a role in the pathogenesis of HSP.

The Value of Procalcitonin and Lipopolysaccharide Binding Protein to Differentiate the Fever In the Patients with Sickle Cell Anemia

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 4818-4818
Author(s):  
Selma Unal ◽  
Ali Ertug Arslankoylu ◽  
Necdet Kuyucu ◽  
Gönül Aslan ◽  
Semra Erdogan
Keyword(s):  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Binding Protein ◽  
Control Group ◽  
Healthy Children ◽  
Lipopolysaccharide Binding Protein ◽  
Wbc Count ◽  
Serum Crp ◽  
And Control ◽  
Lipopolysaccharide Binding

Abstract Abstract 4818 Objective: Differentional diagnosis of fever is very important in patients with sickle cell anemia (SCA) in order to prevent inappropriate antibiotic use, drug resistance and to shorten the hospitalization period. A reliable marker to be used in the differentional diagnosis of fever in these patients has not been defined yet. We aimed to evaluate the values of C Rective Protein (CRP), procalcitonin (PCT), and lipopolysaccharide binding protein (LBP) levels in the differentional diagnosis of fever in patients with SCA. Material and Methods: 86 children with SCA (40 males and 36 females, mean age of 9.6 ± 3.84, range: 1–18 years), (Group 1) and 49 healthy children as a control group (mean age: 8.8 ± 3.91, range: 1–18 years) (Group 2) were included in this study. Patients who had admitted to the emergency department for concurrently vasoocclusive crisis and fever (axillary temperature ≥38C°) were classified as Group 1A and who had vasoocclusive crisis but no fever were classified as Group 1B and the patients without fever and vasoocclusive crisis were classified as Group 1C. A detailed history was taken from every child, and a full physical examination was performed. The patients who had taken antibiotics in last one week were excluded. The type of vasoocclusive crisis were recorded in Group 1A and 1B patients. In Group 1A patients, the fever was evaluated with appropriate laboratory tests (WBC count, periferic blood smear, serum CRP level, urinary test, chest radiography, blood and urinary culture). No infection focus was identified in patient and conrol groups. The WBC count, serum CRP, PCT and LBP levels were evaluated in all the patient and control groups. Results: The median CRP level of all patient groups was significantly higher than the control group (0.78 mg/L; range 0.21–70.0) (p<0.0001). The median CRP level in Group 1A (7.42 mg/L) and Group 1B (6.94 mg/L) were significantly higher than group 1C patients (2.24 mg/L). There were no significant difference between the SCA groups considering median serum PCT levels (Group 1A: 0.18 ng/ml, Group 1B: 0.11 ng/ml, Group 1C: 0.13 ng/ml, p>0.05). These values were significantly higher than control group (0.08 ng/ml) (p<0.0001). LBP level in Group 1A (median:11.5 μg/ml) was significantly higher than Group 1B (median: 8.9μg/ml), Group 1C (median: 7.7μg/ml) and control group (median:5.9μg/ml) (p<0.0001). Also serum LBP in both Group 1B and Group 1C were higher than control group (p<0.0001). Conclusion: PCT level is not affected from the acute inflamation originates from the vasoocclusive crisis. Thus, serum PCT level can be considered a good marker in the differentional diagnosis of fever in patients with SCA. Disclosures: No relevant conflicts of interest to declare.

Microparticle Profile During Painful Crisis and Steady State Period In Sickle Cell Anemia

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 4681-4681
Author(s):  
Anil Atmis ◽  
Ilgen Sasmaz ◽  
Bulent Ali Antmen ◽  
Keyword(s):  
Tissue Factor ◽  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Complete Blood Count ◽  
Conflicts Of Interest ◽  
Systemic Disease ◽  
Control Group ◽  
Healthy Children ◽  
Crisis Period ◽  
Painful Crisis

Introduction Sickle cell anemia is a disease which is characterized with hemolytic anemia, hypercoagulopathy and painful crisis. Microparticles are 0,1-1 µm sized little membrane particles which are derived during activation or apoptotic phase of cell cycle. It is reported that microparticles are increased in many systemic disease including sickle cell anemia. Aim In this study we aimed to investigate the role of microparticles on during crisis and non-crisis periods in sickle cell anemia patients. Materials and method Twenty nine patients, following by Cukurova University, Department of Pediatric Hematology, are included in this study. Blood samples were collected in 26 of these patients in non-crisis period. Control group formed with 18 healthy children without any systemic disease. Complete blood count, hemoglobin electrophoresis and biochemical parameters were studied in both groups. Also patients’ total microparticle levels, erythrocyte (CD235a), endothelial (CD106), monocyte (CD14) particle levels and tissue factor expressing (CD142) microparticle levels were studied by flow cytometry and whole data was statistically analyzed. Findings Hemoglobin and hematocrit levels were significantly low in sickle cell anemia patients (p<0,001). Levels of HbS were significantly high during crisis period comparing with mean HbS levels during non-crisis period (p<0,001). Total microparticle levels were significantly high in sickle cell anemia patients with painful crisis comparing with control group (p<0,05). Erythrocyte and monocyte microparticle levels were significantly high in patients with painful crisis comparing with non-crisis periods (p<0,05). Endothelial and tissue factor expressing microparticle levels were high in patiens with crisis comparing to non-crisis period but this was not statistically significant (p>0,05). There was not any significant relation with frequency of crisis and microparticle levels (p>0,05). Microparticle levels were low in patients whose were taking hydroxiurea treatment comparing with non-hydroxiurea treatment but this data was not statistically significant (p>0,05). Result As a result we found high levels of total microparticle, erythrocyte and monocyte microparticles in sickle cell anemia patients during painful crisis period. This important clue of crises was need further studies in order to understand the effect of microparticles on pathophysiology of sickle cell anemia. Disclosures: No relevant conflicts of interest to declare.

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