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1.
Very little has been published on the use of romiplostim to treat primary immune thrombocytopenia (ITP), refractory to previous treatments, in children. The objective of this study was to determine its efficacy and safety in pediatric patients in a university general hospital. Retrospective, longitudinal observational study of pediatric patients on treatment with romiplostim. The principal efficacy variable was platelet count. Safety was evaluated by recording possible adverse reactions to the medication, monitoring the appearance of thrombosis, thrombocytopenia during dose reduction, hemorrhage, and myelodysplastic syndromes. Three patients in the authors’ center have been treated with romiplostim (subcutaneous [SC], initial dose: 1 μg/kg/week) for ITP refractory to various treatments: 1 with newly diagnosed ITP and 2 with chronic ITP. Patients were followed up for 27 to 39 weeks after starting treatment. Responses were achieved in 7 to 28 days, and complete responses were maintained for 37% to 91% of the follow-up period, with median platelet counts between 40 × 103/μL and 215 × 103/μL. The adverse reactions observed during follow-up were headache and asthenia in one patient and mucocutaneous bleeding after dose suspension in another one. With regard to effectiveness, the response in the 3 patients was varied. The drug was considered to be safe, as there were only mild adverse reactions. Although further studies and long-term follow-up are required, these results show that romiplostim could be considered an alternative to immunosuppressive therapies, such as rituximab, or splenectomy in refractory chronic ITP.  相似文献   

2.
Aim: To describe the clinical course, morbidity and platelet recovery in an unselected Nordic cohort of children with chronic Immune Thrombocytopenic Purpura (ITP). Methods: Prospective 5‐year follow‐up of 96 children with ITP lasting more than 6 months, with reporting of hospital admissions, severity of bleeding episodes and stabilization of platelet counts above 20, 50 and 150 × 109/L. Results: The estimated 5‐year recovery rate was 52%; exclusion of 12 splenectomized children did not change the estimate. Events eliciting admission to hospital occurred in 39 (41%). Major haemorrhages occurred in eight children (8%), including a nonfatal intracranial haemorrhage in one child (1%). The overall admission rate was 0.4/year of thrombocytopenia, decreasing during follow‐up as thrombocytopenia converted to milder degrees. Early recovery within 2 years of diagnosis occurred in 35%, was associated with low morbidity and was more likely in young children with abrupt onset of symptoms. Conclusion: In a Nordic cohort of children with chronic ITP, one half had recovered 5 years after diagnosis, more than half never required hospitalization and <10% experienced serious bleeding episodes, always with a platelet count <20 × 109/L. Aggressive management can be restricted to the minority of children with continuing severe thrombocytopenia and frequent, clinically significant bleeding events.  相似文献   

3.
Very little has been published on the use of romiplostim to treat primary immune thrombocytopenia (ITP), refractory to previous treatments, in children. The objective of this study was to determine its efficacy and safety in pediatric patients in a university general hospital. Retrospective, longitudinal observational study of pediatric patients on treatment with romiplostim. The principal efficacy variable was platelet count. Safety was evaluated by recording possible adverse reactions to the medication, monitoring the appearance of thrombosis, thrombocytopenia during dose reduction, hemorrhage, and myelodysplastic syndromes. Three patients in the authors' center have been treated with romiplostim (subcutaneous [SC], initial dose: 1 μg/kg/week) for ITP refractory to various treatments: 1 with newly diagnosed ITP and 2 with chronic ITP. Patients were followed up for 27 to 39 weeks after starting treatment. Responses were achieved in 7 to 28 days, and complete responses were maintained for 37% to 91% of the follow-up period, with median platelet counts between 40 × 10(3)/μL and 215 × 10(3)/μL. The adverse reactions observed during follow-up were headache and asthenia in one patient and mucocutaneous bleeding after dose suspension in another one. With regard to effectiveness, the response in the 3 patients was varied. The drug was considered to be safe, as there were only mild adverse reactions. Although further studies and long-term follow-up are required, these results show that romiplostim could be considered an alternative to immunosuppressive therapies, such as rituximab, or splenectomy in refractory chronic ITP.  相似文献   

4.
We investigated the effect of subcutaneous anti‐D IgG as platelet enhancing therapy in children with idiopathic thrombocytopenic purpura (ITP). Twenty‐three children were treated with subcutaneous anti‐D 50 µg/kg. The median platelet count increased from 7 × 109 to 31 × 109/L on day 3 (P < 0.01). The median decline in hemoglobin was 1.3 g/dl. Two children experienced minor fever and chills within 24 hr of treatment. Pain at the injection site was common but self‐limiting with no effect on activity level. These results suggest subcutaneous anti‐D IgG 50 µg/kg as an effective and well‐tolerated treatment option in childhood ITP. Pediatr Blood Cancer 2009; 53:1315–1317. © 2009 Wiley‐Liss, Inc.  相似文献   

5.
Thrombocytopenia is a major risk factor for cirrhotic liver disease. Patients with thrombocytopenia may have esophageal or gastric varices secondary to portal hypertension, leading to variceal bleeding which exposes the liver to further damage. Here, we present a female pediatric patient with PSC and CD, whose progressive thrombocytopenia was successfully controlled by romiplostim, a TPO receptor agonist. The patient developed bloody diarrhea at four yr of age, and was subsequently diagnosed with PSC and CD when seven yr old. While CD was well‐controlled by immunomodulators, the patient's thrombocytopenia gradually progressed resulting in petechiae (platelet count of 11 × 109/L) when she was 10 yr and four months old. She responded poorly to immunoglobulin and corticosteroids. Weekly subcutaneous injection of romiplostim was therefore initiated, and platelet counts were maintained over at 50 × 109/L. She was able to undergo successful LDLT without platelet transfusion seven months after the initiation of romiplostim. Romiplostim was not required after LDLT with improved platelet counts. This case report suggests that romiplostim may be effective in the treatment of thrombocytopenic children with liver cirrhosis and portal hypertension, and in eliminating the need for platelet transfusion during the peri‐transplant period.  相似文献   

6.
Agranulocytosis and thrombocytopenic purpura occurred simultaneously following measles infection in a 2-year-old girl who had received a living-related orthotopic liver transplantation. Neutrophil and platelet counts rose to more than 0.5 × 103/mm3 and 30 × 103/mm3, respectively, 8 weeks after the onset of neutropenia and thrombocytopenia, and within 2 weeks from the start of subcutaneous injection of granulocyte colony-stimulating factor. Simultaneous occurrence of measles infection, neutropenia and thrombocytopenia has not been described before in the literature. An autoimmune mechanism was suspected because serum levels of platelet-associated immunoglobulin G (IgG) and anti-neutrophil antibody were elevated during the acute phase.  相似文献   

7.
Nine newly diagnosed, previously untreated children (mean age: 4.2 years, range: 1-9 years) with severe acute idiopathic thrombocytopenia purpura (mean platelet count: 5.8 X 10(9)/L, range: 1-12 X 10(9)/L) were treated with high-dose steroids (prednisone 4-8 mg/kg/day). Steroid dose was based on platelet count at presentation: Group I (platelets less than 5 X 10(9)/L) was started on 8 mg/kg/day; Group II (platelets 5-15 X 10(9)/L) received 6 mg/kg/day. All patients had serologic and histologic evidence of acute idiopathic thrombocytopenia purpura. On this protocol, it took a mean number of 1.9 days (1-3 days) to reach a platelet count of at least 20 X 10(9)/L and 9.2 days (3-26 days) to reach a normal platelet count. No significant toxicity was observed except for weight gain ranging from 3-10% and mild behavioral problems. Both groups were on high-dose steroids (4-8 mg/kg/day) for 7.3 +/- 2.1 days. Only one patient had a brief relapse to a platelet count of 18 X 10(9)/L while on therapy (day 14), but responded promptly to an increase in prednisone dose. Presently, all nine patients are in remission and have not required maintenance medication.  相似文献   

8.
Two patients with a long history of unexplained thrombocytopenia, eventually diagnosed with von Willebrand''s disease (vWD) type 2B are reported. In one patient with platelet counts of 80× 109/l 1-desamino-8-D-arginine vasopressin (DDAVP) had a favourable effect during bleeding episodes. The second patient received intermediate purity von Willebrand''s factor (vWF)/factor VIII concentrate (Haemate HS), which helped haemostasis during tooth extraction. It increased platelet counts from 15 to 30 × 109/l, whereas platelet transfusions produced no increase, nor prevented severe bleeding during abdominal surgery. Thus the treatment of vWD type 2B might depend on the degree of thrombocytopenia. It is recommended that in patients with mild to moderately decreased platelet counts, DDAVP treatment can be tried, whereas in patients with severely decreased platelet counts intermediate purity vWF/factor VIII concentrate substitution is preferred.
In addition, vWD type 2B should be considered in the differential diagnosis of any child with chronic thrombocytopenia as the treatment strategy is different.

  相似文献   

9.
Rituximab is a chimeric monoclonal antibody directed against normal and malignant mature B-lymphocytes and results in prolonged and severe B-cell depletion. Recently, rituximab has been successfully used in adult and paediatric disorders of B-lymphocytes such as autoimmune haemolytic anaemia and Werlhof disease. We report on two children with chronic immune thrombocytopenic purpura (ITP) refractory to steroids and immunoglobulins who achieved complete normalisation of their platelet counts after treatment with rituximab, 375 mg/m2 given weekly in four doses. In both cases the B-lymphocyte count dropped to zero after the second dose of rituximab and an unsupported platelet count >100×109/l was achieved during treatment. Six and 12 months after treatment, both patients remain well with normal platelet counts. Conclusion: this report supports the concept that rituximab may also be a valuable therapeutic option in children with chronic immune thrombocytopenic purpura refractory to standard treatment. Controlled clinical trials are needed to evaluate the efficacy and long-term side-effects of rituximab in this group of patients.Abbreviations ITP immune thrombocytopenic purpura - IVIG intravenous immunoglobulinsSupported in part by the Regione del Veneto Ricerca Sanitaria Finalizzata n 107/02.  相似文献   

10.
Objective  To evaluate the prevalence of thrombocytopenia in neonates born to mothers with pregnancy induced hypertension (PIH) and identify the associated material and neonatal characteristics. Methods  In the current, prospective study, platelet counts were assessed serially. Maternal and neonatal characteristic were recorded in pre-designed proforma. Primary outcome measures were thrombocytopenia defined as platelet count of <150000/mm3 and severe thrombocytopenia if counts were <30000/mm3 or <50000/mm3 with bleeding. Results  Of 97 neonates born to PIH mothers 35 (36.1%) had thrombocytopenia. In 20 (20.6%) thrombocytopenia was severe. Higher percentage of thrombocytopenia was associated with male gender (47.7%), low birth weight (71.4%) and prematurity (67.4%). Severe thrombocytopenia was significantly associated with low birth weight (OR: 4.58; 95% CI: 0.98–21.3; p<0.03) and prematurity (OR: 2.52; 95% CI: 0.87–7.24; p<0.05). Material parity, onset of PIH, and medications did not seem to be associated significantly. Conclusion  Premature and low birth weight neonates born to mothers with pregnancy induced hypertension would require scrutiny for thrombocytopenia during early neonatal period.  相似文献   

11.
12.
Auto‐SCT is a common approach for metastatic neuroblastoma with the intention to rescue hematopoiesis after megadose chemotherapy. PBSC or BM is the usual stem cell source for auto‐SCT. Auto‐CBT for neuroblastoma has very rarely been performed. Currently, case reports are available for two patients only. We performed 13 auto‐SCTs for high‐risk neuroblastoma from 2007 to 2013, including four cases of metastatic neuroblastoma aged 11–64 months treated with auto‐CBT. All four patients had partial or CR to upfront treatments before auto‐CBT. Nucleated cell dose and CD34+ cell dose infused were 2.8–8.7 × 107/kg and 0.36–3.9 × 105/kg, respectively. Post‐thawed viability was 57–76%. Neutrophil engraftment (>0.5 × 109/L) occurred at 15–33 days, while platelet engraftment occurred at 31–43 days (>20 × 109/L) and 33–65 days (>50 × 109/L) post‐transplant, respectively. There was no severe acute or chronic complication. Three patients survived for 1.9–7.7 yr without evidence of recurrence. One patient relapsed at 16 months post‐transplant and died of progressive disease. Cord blood may be a feasible alternative stem cell source for auto‐SCT in patients with stage 4 neuroblastoma, and outcomes may be improved compared to autologous PBSC or BM transplants.  相似文献   

13.
We evaluated the effects of platelet transfusions and intravenous immunoglobulin (IVIG) in neonates with fetal and neonatal alloimmune thrombocytopenia (FNAIT) with and without antenatal treatment with IVIG. Records of neonates with FNAIT admitted between January 2000 and November 2005 were reviewed. The patients were divided into group I, treated antenatally with IVIG for known FNAIT, and group II, postnatally diagnosed with FNAIT. The primary outcome was the time interval to reach a platelet level above 100 × 109/L in relation to the type of treatment. Nineteen neonates with FNAIT were identified, 13 in group I and 6 in group II. In group I, four children were born with a platelet count above 100 × 109/L and never needed treatment, and four received a single matched platelet transfusion at birth with a maintained response. Five neonates received IVIG and one matched transfusion, with all but one rapidly responding. In antenatally treated cases, postnatal IVIG had no apparent effect on the platelet count. In group II, two neonates died on day 1 with severe intracranial hemorrhage. Two of the four other patients responded to a number of unmatched platelet transfusions, with one neonate rapidly responding after one matched transfusion, while another needed nine matched transfusions before a persistent adequate platelet count was reached after 9 weeks. Postnatal IVIG had no apparent effect on the platelet count in any of our cases. In neonates with FNAIT treated antenatally with IVIG, neonatal management using a single matched platelet transfusion was adequate in all cases. In neonatally diagnosed cases not treated before birth, multiple matched platelet transfusions may be required. We found no evidence to support the use of IVIG in neonates with FNAIT.  相似文献   

14.
Background: Idiopathic thrombocytopenic purpura (ITP) is an autoimmune disorder with a variable clinical course. Methods: A retrospective analysis was carried out of ITP patients presenting to a pediatric hematology‐oncology department during a period of 20 years, with a focus on treatment and outcome. Results: One hundred and twenty‐four cases were recorded (mean patient age, 8.4 years). Forty‐nine children (39.5%) had platelet counts <10 000/µL at diagnosis. No episode of severe bleeding was observed. Peak incidence was observed during spring and summer. Respiratory infections proceeded in 58% of cases. Treatment consisted of i.v. immunoglobulin (IVIG) in 93 children at four dosing schedules. Sixteen children received corticosteroids, 10 children received anti‐D immunoglobulin and 14 received no treatment. Recovery was observed in 67% of children on IVIG and in 50% on anti‐D globulin. Eight patients did not respond initially and received corticosteroids. Three children with refractory thrombocytopenia received anti‐CD20 (rituximab). Fourteen children (11%) had persistent/chronic disease. In 10 of them recovery was observed in 13 months–8 years. Splenectomy was performed in six children with resistant/chronic disease. Conclusion: ITP has a benign course in the majority of cases. Anti‐D globulin can effectively be used as an alternative first‐line treatment. Rituximab can successfully be used in refractory cases, while splenectomy has currently limited indications.  相似文献   

15.
Objective To investigate the thrombocytopenia and platelet transfusion related outcome in very preterm infants. Methods Cases (n=94) with at least one episode of thrombocytopenia (platelet counts <150X109/L) and controls (n=70) were identified from a database of 1054 neonates with gestational age ≤32 weeks admitted to a level III NICU. Thrombocytopenia and platelet transfusion related morbidity (IVH, sepsis, NEC, and bleeding) and mortality were analyzed with respect to gestational age (<28 weeks and 28–32 weeks), severity of thrombocytopenia (mild if platelet count ≥ 100 and <150X109/L, moderate if count ≥ 50 and <100X109/L, and severe if platelets <50X109/L), age of thrombocytopenia onset (early <72 hours and late ≥72 hours). Results The majority of thrombocytopenia (67.0%) was diagnosed after 72 hours of age, and was mild in 12.8%, moderate in 36.2% and severe in 51.0% of the cases. Neonates with severe and moderate thrombocytopenia were more frequently born at lower gestational age and birth weight. NEC and sepsis especially that caused by Candida infection, were associated with severe thrombocytopenic events. The development of IVH was strongly associated with lower gestational age but not the severity and age of thrombocytopenia onset. Mucocutaneous bleeding complicated 18.4% of cases with severe and late-onset thrombocytopenia (7/38). Platelets were transfused to 85.4% of infants with severe and 64.7% of infants with moderate thrombocytopenia (P<0.02). The gestational age of the majority of the platelet transfused neonates (49/60, 81.7%) was <28 weeks. Mean gestational age and birth weight, and rates of severe thrombocytopenia, IVH, sepsis and mortality were comparable in transfused vs not-transfused infants with gestational age 28–32 weeks. Platelet transfused neonates with gestational age <28 weeks had lower birth weights, were more often severely thrombocytopenic, and died more frequently than infants of a similar gestational age who were not transfused. Conclusion Platelet transfusions did not lower mortality in very premature born infants with moderate and severe thrombocytopenia during the NICU admission.  相似文献   

16.
目的探讨持续性免疫性血小板减少症(persistent ITP,pITP)和慢性免疫性血小板减少症(chronic ITP,cITP)患儿临床特征和疗效。方法对2002年12月-2009年12月间于我院诊断和治疗的pITP和cITP患儿103例的临床资料进行回顾性分析。结果 (1)103例患儿中96.1%年龄分布在学龄前期及以后;男女性别比例为1.24∶1。(2)73.8%有诱因,其中上呼吸道感染占89.5%;患儿病原血清学IgM阳性率为45.0%,混合感染率为36.1%。(3)有黏膜出血者占61.2%,失血性贫血者占25.2%,以轻度贫血为主。就诊时血小板计数<25×109/L者占71.9%。黏膜出血组与非黏膜出血组的血小板计数比较差异无显著性;而血小板减少与贫血程度间比较差异亦无显著性(P>0.05)。(4)遵医嘱维持用药治疗者占59.2%,该组患儿黏膜出血的发生率(55.7%)低于维持间断治疗组(69.0%);而维持用药组患儿失血性贫血发生率(8.2%)显著低于间断治疗组(50.0%),差异有极显著性(χ2=23.034,P<0.001)。(5)单用激素组(79.7%)、激素+IVIG(静脉注射用人免疫球蛋白)组(78.6%)治疗有效率高于激素+VCR(长春新碱)组(40.0%);激素+IVIG与激素+VCR组间疗效差异有显著性(χ2=4.441,P=0.035);单用激素组与激素+VCR组间疗效差异有显著性(χ2=9.772,P=0.002)。结论 (1)pITP和cITP患儿主要见于学龄前期及学龄期儿童,性别差异不明显。(2)上呼吸道感染是儿童pITP和cITP发生的主要诱因,病毒感染在ITP病情反复中起着一定的作用。(3)pITP和cITP患儿出血程度较轻,血小板减少以重型、极重型居多;而出血表现、贫血程度与血小板减少间无相关性。(4)维持用药可减轻pITP和cITP患儿出血症状,降低失血性贫血发生率。(5)单用激素组、激素+IVIG组的治疗有效率显著高于激素+VCR组。  相似文献   

17.
Abstract Neonatal thrombocytopenia related to maternal idiopathic thrombocytopenic purpura (ITP) is reportedly uncommon but may have severe complications. The present report reviews records of 15 infants born to mothers with ITP during a 10-year period, and the incidence of neonatal thrombocytopenia and the risk of hematological complications is examined. Severe thrombocytopenia (platelets < 50 000/μL) was seen in three infants despite successful therapy with high-dose gamma globulin prior to delivery, which elevated maternal platelet counts. Although the platelet counts of these three infants fell to < 10 000/μL, none had severe complications. Moreover, no infants required treatment such as adrenocorticosteroids, platelets transfusion, or high doses of gamma globulin. No maternal markers predicted the degree of neonatal thrombocytopenia. The risk of complications arising from neonatal thrombocytopenia is low, but careful observation is required for the thrombocytopenic newborn of ITP mothers even when the infant has no bleeding complications at delivery.  相似文献   

18.
We retrospectively analyzed sequential therapy with romiplostim and eltrombopag in 23 children with immune thrombocytopenia: switching from romiplostim to eltrombopag (10 patients) or vice versa (13 patients). The median age of patients at enrollment in the study was 5.6 years (2‐15 years). Switching from romiplostim to eltrombopag was effective in eight (80%) patients, whereas switching from eltrombopag to romiplostim was effective in eight (62%) patients. The response rate was similar in patients failing the first thrombopoietin receptor agonist and those who had previous response. To date, all responders continue to maintain platelets over 50 × 109/L at 13‐39 months after switching.  相似文献   

19.
The evaluation and management of thrombocytopenia is a frequent challenge for neonatologists, as it affects 22-35% of infants admitted to the neonatal intensive care unit. Multiple disease processes can cause neonatal thrombocytopenia, and these can be classified as those inducing early thrombocytopenia (72 h). Most cases of neonatal thrombocytopenia are mild to moderate, and do not warrant intervention. In approximately 25% of affected neonates, however, the platelets count is <50x10(9)/L, and therapy with platelet transfusions is considered to decrease the risk of hemorrhage. The existing evidence to establish platelet transfusion triggers in neonates is very limited, but it suggests that transfusing platelets to non-bleeding neonates with platelet counts >50x10(9)/L does not decrease the risk of intraventricular hemorrhage (IVH), and that 30x10(9)/L might be an adequate threshold for stable non-bleeding neonates. However, adequately powered multi-center studies are needed to conclusively establish the safety of any given set of neonatal transfusion guidelines.  相似文献   

20.
INTRODUCTION: Thrombocytopenia has been shown to be an independent predictor of mortality and prolonged hospital length of stay in critically ill adults. Studies are lacking in the pediatric intensive care unit population. We evaluated the relationship between platelet counts at admission, platelet course, and outcomes. HYPOTHESES: 1) Thrombocytopenia at the time of admission to the pediatric intensive care unit is a risk factor for increased mortality and prolonged length of stay. 2) Thrombocytopenia at any point during pediatric intensive care unit stay is associated with increased mortality and length of stay. 3) Falling platelet counts during a pediatric intensive care unit course are associated with greater mortality and longer length of stay. METHOD: Prospective observational study. STUDY POPULATION: All patients admitted to a multidisciplinary tertiary care pediatric intensive care unit in a University Hospital over the course of a year. ANALYSIS OF DATA: Data were analyzed using logistic and linear regression. RESULTS: Thrombocytopenia (platelet count <150 x 10/L) was present in 17.3% of pediatric intensive care unit patients on admission. Mortality was higher in thrombocytopenic patients (17.6% vs. 2.47%, p < 0.001). The median length of stay in the thrombocytopenia and nonthrombocytopenia groups was 4 days vs. 1.6 days, respectively (p < 0.001). The pediatric intensive care unit patients (25.3%) were thrombocytopenic at some point in their stay. They had higher mortality (17.1% vs. 0.9%, odds ratio [OR] 23.8, 95% confidence interval [CI] 5.2-108.6, p < 0.0005) and longer length of stay (median 6.6 days vs. 1.5 days, p < 0.0005) compared with those who were never thrombocytopenic. For every 10% fall in platelet count from the time of admission, the OR for mortality was 1.4 (95% CI 1.1-1.8) and the length of stay was longer (p < 0.0005). Patients with normal platelet counts at admission who later developed thrombocytopenia had increased mortality (OR 18.6, 95% CI 3.2-107.3) and longer length of stay (p < 0.0005) compared with those who did not develop thrombocytopenia. CONCLUSION: Thrombocytopenia and falling platelet counts are associated with increased risk of mortality and length of stay in the pediatric intensive care unit.  相似文献   

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