Essential versus reactive thrombocythemia in children: retrospective analyses of 12 cases

BACKGROUND: Essential thrombocythemia (ET) rarely occurs in the pediatric population and little is known about the clinical course and the molecular characteristics. PROCEDURE: In this retrospective multi-institutional study we examine the clinical, hematological, and molecular features of 12 children aged 5-16 years with thrombocytosis and a suspected diagnosis of ET. RESULTS: Median follow-up was 59 months (range 10-72). Seven patients presented with clinical symptoms potentially related to thrombocytosis. The remaining five patients were diagnosed incidentally. Median platelet count at diagnosis was 1,325 x 10(9)/L (range 600-3,050). In 11 out of 12 cases bone marrow morphology was consistent with ET, the remaining patient had chronic idiopathic myelofibrosis. Cytogenetic analyses were normal in all studied cases and only one out of nine analyzed cases harbored a JAK(V617F) allele. Within 6 months after initial presentation one patient who was initially asymptomatic developed thrombosis and another patient had mild bleeding. Eight patients were treated with acetylsalicylic acid, one patient received hydroxyurea, and two patients received anagrelide. At last follow-up, all patients were alive and none had developed leukemia. Five patients experienced hematological remission. Two children had not received any therapy. During the course of their disease, nine patients developed symptoms possibly attributable to an elevated platelet count. CONCLUSIONS: In JAK2 mutation negative cases, long-term follow-up is helpful to distinguish between primary and secondary thrombocytosis. Secondary cases are not associated with organomegaly but may present with unspecific symptoms. Indications for treatment in children remain unclear.     

Efficacy of postoperative chemotherapy using cisplatin plus etoposide in young children with brain tumors

Neuraxis radiation therapy (RT) for primary intracranial tumors is associated with major late effects if administered to very young children. To control residual tumor and to delay RT, we treated eight young children (median age 6.5 months) with primary central nervous system (CNS) tumors using combination chemotherapy: cisplatin, 20 mg/M2/day plus VP-16, 75 mg/M2/day i.v. for 5 days, given q. 3-6 weeks for 8 cycles. The tumors were medulloblastoma (one), malignant ependymoma (two), primitive neuroectodermal tumor PNET (two), malignant glioma (two), astrocytoma (one). Six had measurable disease; three had positive cerebrospinal fluid (CSF) cytopathology. All patients with measurable tumor had initial objective responses (three) complete response [CR], one partial response [PR], two minor response [MR], including cytopathology (three CR of three) and metastatic deposits (two CR of two). One patient relapsed during chemotherapy. Median time to disease progression was 17.5 months; median survival was 34 months. Three patients, none of whom received RT, have prolonged progression-free intervals of 47-67 months to date. Neurodevelopmental progress continued during and after chemotherapy. Chemotherapy toxicity was mild. Median neutrophil nadir was 312/mm3, platelets 72,000. Fever during neutropenia occurred in six of 61 courses. Moderate high-frequency auditory losses were detected in three patients, and mild renal injury (GFR less than 70 ml/min) was detected in two of seven evaluable children. This pilot study demonstrates the apparent efficacy and mild toxicity of 5 day courses of cisplatin plus VP-16, with delayed RT, in young children with CNS neoplasms. A POG treatment protocol that incorporates cisplatin plus VP-16 is evaluating primary chemotherapy with delayed radiotherapy in larger numbers of pediatric brain tumor patients.     

Alpha-interferon therapy induces improvement of platelet counts in children with chronic idiopathic thrombocytopenic purpura

PURPOSE: To investigate alpha-interferon (IFN) therapy for children with chronic idiopathic thrombocytopenic purpura (ITP). PATIENTS AND METHODS: Patients with refractory ITP lasting more than 12 months from diagnosis were included if they had platelet counts <50 x 10(9)/L and had received no treatment during the past month. Patients received IFN (3 x 10(6) U/m2 per dose), three times per week for 4 weeks; if partial (<150 x 10(9)/L) or no response was obtained, the same dose was continued for another 8 weeks. In patients with favorable response and subsequent decrease to pre-treatment values, an additional 4 weeks of treatment could be administered. RESULTS: Fourteen patients (ages 4-20 y) receiving 17 IFN courses were included. Mean initial platelet count was 29 +/- 15 x 10(9)/L. A significant increase was achieved during 14 of 17 courses (82.4%). All but two responses were transitory, and platelets returned to initial values after IFN discontinuation (mean 44 +/- 26 days). Considering the best response achieved by each patient, we observed: 1) 10 patients who achieved a sustained improvement of platelet count throughout the treatment period, decreasing to initial values after therapy was stopped; 2) one patient who achieved platelet count >150 x 10(9)/L, remaining with normal platelets at 18 months; 3) one patient who achieved platelet count >150 x 10(9)/L, remaining with platelets between 100 and 140 x 10(9)/L at 48 months; 4) one patient who had no response; and 5) one patient in whom therapy worsened the thrombocytopenia. A mild to moderate flu-like syndrome and a moderate decrease of the absolute neutrophil count were the only side effects observed. CONCLUSION: Interferon therapy induces a significant increase of platelet count and seems to be a valid alternative therapy to attempt the achievement of prolonged remission in refractory ITP, to defer splenectomy in younger children, or to improve platelet count before planned splenectomy.     

Single-center experience: use of recombinant factor VIIa for acute life-threatening bleeding in children without congenital hemorrhagic disorder

Coagulopathy is an important cause of mortality in critically ill children. Traditional therapies to correct coagulopathy lead to great time delays and cause fluid overload in patients. The authors report the effectiveness and safety of the activated recombinant factor VII (rFVIIa) administration in a series of 13 nonhemophiliac children with acute, life-threatening bleeding. In this retrospective study, the records of the patients who were not diagnosed with congenital hemorrhagic disorder and were administered rFVIIa due to any other reason in Ege University Faculty of Medicine, Department of Pediatrics, between February 2002 and February 2007 were reviewed retrospectively. Thirteen nonhemophiliac patients with acute life-threatening bleeding and ages ranging from 2 days to 15 years received rFVIIa over a 5-year period. Three patients were diagnosed with hemaphagocytic lymphohistiocytosis, 4 with prematurity, sepsis, and disseminated intravascular coagulation (DIC), 5 with sepsis, multiple organ dysfunction syndrome, and DIC, and 1 with acute liver failure. Severe bleeding resulted from pulmonary (n = 3), lower gastrointestinal system (n = 2), esophagus varices (n = 1), pulmonary and gastrointestinal system (n = 4), pulmonary, gastrointestinal system, and intracranial hemorrhage (n = 1), and gastrointestinal system and intracranial hemorrhage (n = 2). Median frequency of rFVIIa administration was 3 per patient (range 2-15) and median dose of rFVIIa was 90 microg/kg, ranging from 60 to 135 microg/kg each administration. All of the patients were given fresh frozen plasma and if necessary platelet transfusion (n = 10) or fibrinogen concentrate (n = 3) before administration of rFVIIa. In 6 patients, lack of success to control bleeding by conventional methods was the only cause to start rFVIIa. In 7 patients, the need for volume restriction was also a significant contributing factor in deciding to start rFVIIa. Median PT was 32.9 s (range: 19-65) before rFVIIa administration and it was decreased to 11.6 s (range: 10.7-12.8), 2-3 h after rFVIIa infusion. Bleeding was stopped completely in 10 patients at least for 24 h and decreased in 3 patients 30-45 min after rFVIIa administration. Two patients had thrombotic complications attributed to rFVIIa administration. No other complication was observed in the other patients. In this retrospective study, rFVIIa was found to be effective at controlling severe hemorrhagic symptoms of different etiologies in children without congenital hemorrhagic disorder. In addition to the rapid control of bleeding, administration of this agent improved fluid balance and led to a reduction in blood product requirements in critically ill children. However, survival was still poor (23%), and 2/13 (15.4%) patients developed venous and arterial thrombosis within 3 h of treatment. The authors emphasize that in acquired, acute life-threatening bleeding, simultaneous administration of rFVIIa with conventional treatment may contribute to patient survival. However, the risk of thromboembolism should be considered before this treatment is given.     

Chronic immune thrombocytopenic purpura in children: a survey of the canadian experience

BACKGROUND: Immune thrombocytopenic purpura (ITP) in children is a common pediatric bleeding disorder with heterogeneous manifestations and a natural history that is not fully understood. To better understand the natural history of chronic ITP and detect response trends and outcomes of therapy, we conducted a 10-year retrospective survey of children from age 1 to 18 years with a diagnosis of chronic ITP. RESULTS: Data on 198 patients from 8 Canadian Pediatric Hematology/Oncology centers were analyzed. The majority of patients were female (58%), and were previously diagnosed with acute (primary) ITP (85%). The age at diagnosis of chronic ITP ranged from 1.1 to 17.2 years with a mean of 8.2+/-4.4 years. Ninety percent of patients received some form of treatment. Untreated patients had a higher mean platelet count at diagnosis of chronic ITP (P=0.009) despite similarities in mean age at first presentation and mean duration of follow-up. Thirty-four (17%) patients underwent splenectomy. Splenectomized patients tended to be significantly older, had a lower mean platelet count at diagnosis of chronic ITP, and had a longer duration of follow-up. CONCLUSIONS: The results from this study are consistent with published reports.     

硬化剂治疗食管静脉曲张破裂出血的远期疗效分析

目的 探讨经硬化剂治疗食管静脉曲张破裂出血后的远期疗效。方法 1996-2003年在内镜下用硬化剂(1％乙氧硬化醇)注射治疗15例食管静脉曲张破裂出血患儿,年龄3—14岁,采用静脉内和静脉旁联合注射。注射后长期临床与内镜随访,观察再出血率及曲张静脉消失率与并发症发生率。结果 15例共作了43次硬化剂注射治疗,依治疗时间先后不同,随访时间从3年4个月到7年2个月不等,平均5年6个月。10例经2次治疗,5例经3次治疗,曲张静脉基本消退,内镜随访发现曲张静脉平均消退时间为3-6个月。15例长期随访发现仅3例曲张静脉复发。再出血率明显减少,15例中仅3例发生再出血,其中2例因长期失访,直至再出血来院检查,发现1例系十二指肠溃疡,1例为静脉曲张出血,而治疗前几乎每1-2个月出血1次。此外未见一例食管穿孔、食管狭窄的并发症。结论 食管静脉曲张硬化剂疗法治疗儿童食管静脉曲张安全有效,尤其是肝外血管畸形所致的门脉高压症效果显著,是挽救患儿生命,提高患儿生命质量的有效内科治疗方法。     

High-dose steroids in childhood acute idiopathic thrombocytopenia purpura

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.     

The use of recombinant coagulation factor VIIa in uncontrolled postoperative bleeding in children undergoing cardiac surgery with cardiopulmonary bypass.

OBJECTIVE: To assess the hemostatic efficacy of recombinant coagulation factor VIIa (rFVIIa) in the management of uncontrolled bleeding in postcardiac surgery with cardiopulmonary bypass in children. DESIGN: An open-label study. SETTING: A postoperative intensive care unit. PATIENTS: Eight consecutive pediatric patients with excessive bleeding after cardiac surgery with cardiopulmonary bypass that met the criteria for reexploration and did not respond to optimal transfusions of platelets and fresh frozen plasma. INTERVENTIONS: rFVIIa 30 microg/kg was given as a bolus injection. A higher dose of 60 microg/kg was used if a patient had preoperative coagulopathy, preoperative multiple-organ failure, or indications that required an emergency operation. The same dose was repeated 15 mins after the previous injection if the bleeding had not decreased. If the bleeding had decreased but still exceeded 10 mL/hr for body weight 5 kg, the same dose was repeated 2 hrs after the previous injection. A maximum of four doses could be given before rFVIIa was considered ineffective and a reexploration was needed. MEASUREMENTS AND MAIN RESULTS: Postoperative blood loss was estimated from the volume of chest tube drainage. rFVIIa successfully controlled bleeding and prevented reexploration in all seven patients who received treatment according to the protocol. One patient who received only one dose of rFVIIa required reexploration because a second dose was not available. No adverse events related to rFVIIa were seen. CONCLUSIONS: rFVIIa may be useful in preventing reexploration in uncontrolled postoperative bleeding in children undergoing cardiac surgery with cardiopulmonary bypass. Randomized, placebo-controlled studies are needed to confirm the safety and efficacy of rFVIIa in this clinical setting.     

The prevalence of bleeding disorders among healthy pediatric patients with abnormal preprocedural coagulation studies

BACKGROUND: We evaluated the prevalence of hemostatic disorders among pediatric patients with abnormal screening coagulation tests. PROCEDURE: We analyzed 48 consecutive referrals for abnormal prothrombin times, partial thromboplastin times, or closure times obtained as preprocedural screens. Patients were evaluated by uniform diagnostic testing. RESULTS: Seventeen patients (35%) had an isolated nonspecific inhibitor (NSI). Six patients (12.5%) presented with mildly low factor activity with a concomitant NSI. These deficiencies were of unclear clinical significance. One patient (2%) had a lupus anticoagulant. Only 9 patients (19%) had a possible or true mild bleeding disorder: 5 patients (10%) had isolated low von Willebrand factor levels, 2 patients (4%) had possible type I von Willebrand disease, and 2 (4%) had platelet aggregation disorders. In all patients, personal and family bleeding history had a positive predictive value of 45% for hemostatic disorders. CONCLUSIONS: The most common diagnosis among the patients referred to us for abnormal preoperative coagulation tests was a NSI, which is not associated with an increased risk of operative bleeding complications. Less than 20% had a possible or true mild bleeding disorder. Although certain bleeding disorders can be occult in children and are associated with perioperative bleeding risks, our study demonstrates the inherent limitations in making a laboratory diagnosis of a bleeding disorder in pediatric patients preoperatively. Our findings contribute to existing doubt about the usefulness of prothrombin times, partial thromboplastin times, and closure times to identify occult bleeding disorders in this population.     

Chronic childhood idiopathic thrombocytopenia purpura: long-term follow-up

An understanding of the natural history of childhood chronic idiopathic thrombocytopenia purpura (ITP) could contribute to a rational therapeutic approach to its treatment, which remains controversial. In our retrospective study of 92 children with ITP, 22 had a chronic course and were followed for 3–14 years (median 8.6 years). Treatment, when indicated, was individualized: 4 patients (18.2%) did not receive any treatment, 14 (63.6%) received steroids only, while 4 (18.2%) were treated with steroids and one of the following: high-dose gamma globulin (4 patients), splenectomy (2 patients) or immunosuppressive therapy (2 patients). During follow-up, 14 patients (63.6%) achieved complete remission, 5 (22.7%) partial remission and only 3 (13.5%) remained severely thrombocytopenic, with minimal bleeding tendency. Eleven patients (50%) responded to the initial prednisone course (1–5mg/kg/day), but showed a marked decrease in platelet count when steroids were tapered off. In view of the high rates of complete and partial remission and the mild course of the few non-responding patients, it is suggested that with adequate supportive therapy, follow-up problems and fatalities can be kept to a minimum. We believe that aggressive therapy, such as splenectomy, should be reserved for the rare symptomatic and severely thrombocytopenic patient.     

Nasopharyngeal carcinoma in the pediatric age group: the northern Israel (Rambam) medical center experience, 1989-2004

Nasopharyngeal carcinoma (NPC) is rare in children, accounting for less than 1% of all malignancies. Radiation therapy has been the mainstay of treatment of many years, but to improve survival, the use of chemotherapy has been advocated. This is a retrospective analysis of 13 patients less than 20 years of age treated for NPC the Rambam Medical Center during 1989 to 2004. Eight boys and five girls with a median age of 14.5 years (range 10-19) were included. Median follow up (including patients who died) was 6.15 years (range 1-15 years). Duration of symptoms was 1 to 24 months (median 5 months). Of the 13 patients, one patient had stage I, 6 had stage III, 5 had stage IV-A, and 1 had stage IV-B disease. Ten patients (77%) had undifferentiated carcinoma (WHO type III) and three patients (23%) had nonkeratinizing carcinoma (WHO type II). Most of the children received two or three courses of neoadjuvant multiagent chemotherapy consisting of cisplatin and 5-FU, followed by radiotherapy with doses in excess of 60 Gy. One child received concomitant chemoradiation. Ten of the 13 patients (77%) are alive without disease 6 years after diagnosis (range 1-15 years). One patient developed local and distant metastases 1 year after diagnosis and is currently receiving combined radiochemotherapy. Two patients died. Overall survival was 84%; event-free survival was 77%. Nine patients (69%) developed moderate to severe long-term complications. Pediatric NPC is curable by combined radiation and chemotherapy, with doses of radiation in excess of 60 Gy. Long-term follow-up is important for early detection of second malignancies as well as for radiation-induced endocrinologic deficiencies and other normal tissue complications.     

血浆置换术后联合小剂量激素治疗溶血尿毒综合征

目的探讨血浆置换术后联合小剂量激素治疗溶血尿毒综合征(HUS)的疗效。方法回顾性分析近5 a来本院肾内科11例HUS患儿临床资料。男6例,女5例;年龄2～9岁,平均年龄5.2岁。重症10例,轻症1例。其中8例进行血浆置换治疗,每例2、3次,术后应用泼尼松(1.0～1.5 mg.kg-1)或甲泼尼龙(1 mg.kg-1)维持;其中1例联合血液透析治疗,1例联合连续性血液滤过治疗。轻症1例采用大剂量丙种球蛋白(丙球)治疗。1例重症HUS外院进行血液透析1个月余转本院继续血液透析治疗。1例重症HUS外院采用大剂量丙球冲击联合甲泼尼龙治疗后继续甲泼尼龙1 mg.kg-1治疗和肠道透析。结果 19例次血浆置换治疗均顺利实施,无明显并发症;8例血浆置换后联合小剂量激素治疗者中7例肝酶、心肌酶、肾功能恢复正常,尿常规镜下血尿或并轻中度蛋白尿出院,追踪观察2～26个月,复查肾功能均正常,尿蛋白阴性,5例镜下轻微血尿[RBC(9～36)×106L-1],1例感染后轻微镜下血尿,1例尿常规正常。轻症1例出院时尿常规和肾功能均正常,门诊随诊38个月尿常规正常。2例外院治疗的重症HUS患儿转入本科时病程1周～1个月,血小板已恢复正常,Hb无继续下降,尿常规示肉眼血尿和中量蛋白尿,处于肾衰竭期。其中1例血液透析6次,肾功能稍好转、肉眼血尿并中量蛋白尿,放弃治疗出院。1例经肠道透析和口服激素等措施后放弃治疗出院。结论重症HUS患儿宜早期应用血浆置换治疗,血浆置换治疗后联合小剂量激素治疗可改善重症HUS患儿预后,减少后遗症。     

Disease of the liver following bone marrow transplantation in children: incidence, clinical course and outcome in a long-term perspective

Sixty-four consecutive cases of allogeneic ( n = 16), autologous ( n = 47) or syngeneic ( n = 1) bone marrow transplantation (BMT) in children with haematological or lymphoid malignancy, aplasia or metabolic disease were reviewed to assess the incidence, clinical presentation and outcome of liver disease. Median follow-up time was 5 y (1.0-10). No liver diagnosis was established at the pre-transplant check-up. During the first 100d post-transplant, 81% of the patients had impaired liver function as documented by various biochemical parameters. Three of 64 patients (5%) met diagnostic criteria for veno-occlusive disease. Four (25%) of the 16 receiving allografts were diagnosed as having acute graft vs host disease (GVHD) with liver involvement (grades II-III). No patient died of liver disease. During the late post-transplant follow-up, one patient developed HCV hepatitis after packed erythrocyte transfusion. Four patients were diagnosed as having chronic GVHD with liver involvement; three of them also had an episode of CMV hepatitis. At their latest follow-up, the patients with chronic GVHD had aminotransferase values 1.5–3 times the normal, whereas all other long-term survivors had normal or near-normal liver function tests. We conclude that the incidence of serious liver disease was low in this paediatric population of bone marrow recipients.     

儿童获得性血栓性血小板减少性紫癜五例分析

目的:探讨儿童获得性血栓性血小板减少性紫癜(aTTP)的临床特点、治疗情况以及预后。方法:回顾性病例总结,以2016年1月至2019年7月于首都医科大学附属北京儿童医院住院治疗的5例aTTP患儿为研究对象,分析患儿临床表现、实验室检查、治疗及预后情况。结果:纳入5例aTTP患儿,占同期血栓性血小板减少性紫癜患儿的5/11,其中男2例、女3例,发病年龄8.9(0.8~14.5)岁。5例患儿均存在血小板减少和微血管病性溶血性贫血,仅1例存在经典五联征,3例患儿伴神经系统症状,3例有发热,而肾功能损伤相对少见(1例)。5例患儿均存在重度血小板减低[7(4~14)×10⁹/L]及血红蛋白下降[70(58~100)g/L];血生化检查示3例总胆红素水平升高,均以间接胆红素升高为主,5例乳酸脱氢酶水平均升高,1例尿素氮升高。骨髓穿刺提示巨核细胞数目不低。ADAMTS13活性检查均为0,4例ADAMTS13抑制物阳性,1例为阴性。5例患儿均接受糖皮质激素治疗,并且在疾病早期应用利妥昔单抗治疗,3例患儿接受血浆置换。5例患儿血小板恢复正常的时间为开始治疗后的19(9~29)d。1例患儿在治疗9个月后出现复发,再次予糖皮质激素及利妥昔单抗治疗后病情稳定,随访3年以上确诊为系统性红斑狼疮。截至2020年12月1日,随访24(16~57)个月,5例患儿临床症状消失,未次随访血小板计数为159(125~269)×10⁹/L。结论:儿童aTTP患者较为少见,各年龄段均有发病,临床表现以血小板减少及微血管病性溶血为主,血浆ADAMTS13活性及抑制物检测有助于aTTP的诊断。血浆置换及利妥昔单抗治疗有效,该病需长期随诊监测。     

Carboplatin and VP 16 in medulloblastoma: A phase II study of the French Society of Pediatric Oncology (sfop)

The purpose of this study is to evaluate the antitumor activity of combination carboplatin and etoposide in measurable medulloblastoma. From January '89 to January '92, 26 patients with medulloblastoma were included in a multicentric phase II study of 2 courses of carboplatin 160 mg/m²/d day 1 to day 5 and VP16 100 mg/m²/d day 1 to day 5. Median age was 10 years (19 months-14.5 years). First treatment was surgery alone in 1 patient, surgery + radiotherapy in 4 patients, surgery + chemotherapy in 2 patients less than 3 years old, surgery + radiotherapy + chemotherapy in 19 patients (“8 drugs in 1 day” based:17, SIOP I:1, SIOP II:1). Previous treatment included cisplatin (20 cases), carboplatin (1 case), and VP16 (7 cases). Measurable disease was evaluated by CT scan, MRI or myelogram and CSF. Response rate (RR) was 72 ± 10%:8 complete responses (CR), 10 partial responses (PR), 1 objective effect (OE), 6 progressive disease (PD), 1 non-evaluable. Thirty-six courses were evaluated for toxicity. Median duration of WHO grade 4 neutropenia was 8 days (0–23). One patient died at day 18 after the first course because of diffuse haemorrhage during septic aplasia. Five other non-life-threatening septicemias were recorded. Median number of platelet transfusions was 1 (0–4). One child who had achieved a PR after two courses died from CNS bleeding after the third course. This drug combination achieves a high response rate in childhood medulloblastoma. Severe toxicity has been mainly encountered in previously heavily treated patients. Tolerance may be acceptable in newly diagnosed children, but careful hematological follow-up and platelet transfusional support are definitely mandatory. © 1994 Wiley-Liss, Inc.     

Pediatric sinovenous thrombosis

PURPOSE: To determine if anticoagulation therapy is effective for preventing progression of pediatric sinovenous thrombosis, to determine the safety of anticoagulation therapy in the pediatric population, and to outline risk factors and clinical presentation in the authors' population of patients with sinovenous thrombosis. METHODS: A retrospective chart review was conducted of 17 consecutive pediatric patients with sinovenous thrombosis at the authors' institution regardless of treatment option and outcome. RESULTS: Fifteen children underwent anticoagulation therapy; two did not. Surgical intervention was undertaken in two patients. None of the children died. None had recurrence after anticoagulation was initiated. Of the patients who had follow-up studies performed, 33% had some resolution of the clot, 60% had complete resolution, and 13% had no change. Both children who did not undergo anticoagulation therapy had resolution of the thrombus. All of the children had improvement of their symptoms at presentation. No patient had worsening of radiologic findings during the follow-up period. CONCLUSIONS: Anticoagulation therapy did not result in bleeding complications. Fifteen of 17 patients were safely anticoagulated. All children had improvement of their presenting symptoms. There was no worsening of radiologic findings in any patient, and there was improvement in 13 patients. One patient has long-term neurologic sequelae (a learning disability). This patient underwent extensive surgeries for subdural and epidural empyemas.     

Immunosuppressive therapy versus bone marrow transplantation for children with aplastic anemia

A total of 15 patients 1 to 16 years of age were treated for aplastic anemia (13 of a severe degree) and followed-up for a mean of 24 months (range 2 to 64 months). Six patients had an HLA-matched sibling and underwent allogeneic bone marrow transplantation. Nine patients who lacked a suitable donor were given immunosuppressive therapy. Antithymocyte globulin was the initial treatment for eight of these nine patients. Two patients who failed to respond to antithymocyte globulin were then treated with cyclosporine A. Pretreatment age, hematologic measurements, duration of follow-up, and interval prior to therapy were similar between the two groups. All of the patients receiving bone marrow transplants had a complete response and now have normal blood cell counts. Six of nine patients (67%) responded to antithymocyte globulin and are now transfusion free, although three have mild thrombocytopenia. Both patients given cyclosporine A had a good response and are also transfusion free. Patients who underwent marrow transplantation had a significantly shorter period of transfusion dependence for RBCs (9 v 4 weeks, P less than .005) and platelets (5 v 21 weeks, P less than .05). The patients given immunosuppressive therapy have significantly lesser platelet counts in follow-up but have similar values for both hemoglobin and absolute granulocyte counts. Although HLA-matched bone marrow transplantation leads to a faster and more complete recovery for children with aplastic anemia, immunosuppressive therapy can provide a good outcome for children with this disorder.     

Mesenterico-left portal vein bypass in children with congenital extrahepatic portal vein thrombosis: a unique curative approach

OBJECTIVES: Current management of extrahepatic portal vein thrombosis (EPVT) comprises endoscopic eradication therapy of esophageal varices and conventional shunt surgery. The authors have used the novel technique of mesenterico-left portal bypass (Rex shunt) in seven children with symptomatic EPVT, and report their results here. METHODS: Median age of the children was 12 years (range, 2-16 years). All children had portal hypertension with hypersplenism and recurrent bleeding from esophageal varices. Furthermore, one patient suffered from a severe hepatopulmonary syndrome. Preoperative evaluation included liver function tests, liver biopsy, hepatic duplex ultrasonography, and radiologic evaluation of the intrahepatic and extrahepatic vascular anatomy. The internal jugular vein was used as vein graft in all patients. RESULTS: Median follow-up period was 15 months (range, 3-28 months). Ultrasound scans revealed sufficient perfusion in all shunts (median, 35 cm/s; range, 28-60 cm/s). The intrahepatic portal perfusion in segment 4 improved from a median of 6 cm/s before surgery to 18 cm/s postoperatively. The platelet count increased within 3 months from a mean of 50,625/microL to 137,750/microL. The clinical signs of hypoxemia in the child with hepatopulmonary syndrome disappeared within 6 months. CONCLUSIONS: In accordance with the limited experience published by others, the authors' data confirmed the mesenterico-portal Rex shunt as the therapy of choice for children with EPVT. Furthermore, this report is the first to show that a hepatopulmonary syndrome can be abolished by mesenterico-portal Rex shunt.     

Incidence of high erythrocyte count in infants and young children with iron deficiency anemia: re-evaluation of an old parameter

PURPOSE: To investigate the frequency of high erythrocyte count (red blood cell count >or=5.0 x 106/microL) in infants and young children with iron deficiency anemia and to document the differences in hematologic parameters at diagnosis and during iron therapy in IDA patients with and without a high erythrocyte count. PATIENTS AND METHODS: A total of 140 infants and young children aged 6 to 48 months with nutritional IDA without a history of any bleeding disorder were the subjects of this study. The patients were divided into three groups according to the severity of anemia. Group A1 children had Hb values 8.0 g/dL or less (severe anemia); group A2, 8.1 to 10.0 g/dL (moderate anemia); and group A3, 10.1-11.0 g/dL (mild anemia). All children received oral iron (3-5 mg/kg per day) for 12 weeks. Complete blood counts were done weekly during treatment. RESULTS: A total of 36 of the 140 patients (26%) had a high erythrocyte count. Of the 140 patients, 37 were in group A1, 80 in A2, and 23 in A3. The frequency of high erythrocyte count was 11%, 23%, and 61% in groups A1, A2, and A3, respectively. The patients with a high erythrocyte count had significantly higher Hb and Hct but significantly lower mean corpuscular volume and mean corpuscular hemoglobin (MCH) values than those with a low erythrocyte count (n = 104). A continuous elevation in the erythrocyte count has been observed in patients with a high red cell count, as in those with a low red cell count, after the institution of iron therapy. CONCLUSIONS: A high erythrocyte count is a common feature of iron deficiency anemia in infants and young children, with an increasing frequency from severe to moderate to mild anemia. High erythrocyte count cannot be regarded as a reliable preliminary parameter in differentiating iron deficiency from thalassemias in infants and children aged up to 48 months.