Elective splenectomy in children with idiopathic thrombocytopenic purpura

PURPOSE: The aim of this study was to review the safety and efficacy of elective splenectomy in children with idiopathic (immune) thrombocytopenic purpura (ITP). METHODS: The authors reviewed the medical records of children with ITP treated with elective splenectomy at Children's Medical Center of Dallas since 1961. Indication for splenectomy was symptomatic thrombocytopenia unresponsive to medical management. RESULTS: Thirty-eight evaluable patients who had elective splenectomy for ITP were identified. Twenty-one (55%) were girls and 17 (45%) were boys. Twenty-two had splenectomy since January 1990. Age at diagnosis ranged from 6 months to 15.9 years (median 9 years), and age at splenectomy ranged from 3.6 to 16.4 years (median 11.8). Laparoscopic splenectomy was performed in 11 patients. No patient died and only one (2.6%) had postoperative hemorrhage. There were no other complications related to surgery. No cases of postsplenectomy sepsis were observed. At follow-up ranging from 1 month to 19.9 years (median 2.1 years), 29 patients (76.3%) had a normal platelet count (>150 x 109/L) and 4 (10.5%) had a platelet count between 50 and 150 x 109/L. Only two of the five (13.2%) remaining patients who continued to have a platelet count less than 50 x 109/L had hemorrhagic manifestations necessitating intermittent therapy with corticosteroids. CONCLUSION: Laparoscopic or open splenectomy is a safe and effective procedure for children with chronic or refractory ITP and should be considered when medical management fails or causes excessive toxicity.     

选择性脾切除术治疗儿童ITP的疗效探讨

目的探讨近年来儿童慢性特发性血小板减少性紫癜(ITP)患者行选择性脾切除术的有效性及安全性。方法收集1986年～2000年新华医院及上海儿童医学中心行选择性脾切除术治疗ITP的患儿资料,以术后血小板计数的稳定最低值判断疗效,回顾性研究其相关因素。结果16例慢性ITP患儿行选择性脾切除术,其中9例男孩,7例女孩。治愈7例(43.75%),好转5例(31.25%)。术后随访未有感染并发症发生。治愈患儿的术后血小板峰值均超过400×109/L,而其余患儿中仅2例超过400×109/L,经Fisher精确检验,两组间有显著差异(P<0.01)。结论选择性脾切除术是治疗儿童慢性ITP安全有效的方法。脾切除术后的疗效与术后血小板最高峰值相关,术后高的血小板计数峰值将提示着良好的预后。     

Pulsed high-dose dexamethasone therapy in children with chronic idiopathic thrombocytopenic purpura

The effectiveness of pulsed high-dose oral dexamethasone therapy in children with refractory chronic idiopathic thrombocytopenic purpura (ITP) is evaluated. Thirteen children with severe chronic ITP were enrolled in the study from an outpatient pediatric hematology clinic (ages 2-14 years), 5 boys and 7 girls. They did not maintain a response to other forms of therapy (IVIg, Anti-D, conventional steroids, danazol) and one girl relapsed after splenectomy. Dexamethasone was administered orally at a dosage of 40 mg/M2/day (maximum 40 mg/day) for 4 consecutive days. The cycle was repeated once a month for 6 months. The immediate response to therapy was excellent as the mean platelet count at day 1 was 15 x 10(9)/L, while mean platelet count at day 4 was 158 x 10(9)/L. At the end of 6 cycles 3 patients maintained a platelet count of >150 x 10(9)/L and 4 patients showed partial response. At the end of the first year and second year (12 and 24 months after onset of treatment) 3 patients still had complete response, 3 patients had partial response, and 7 patients were failures. Six of the failures underwent splenectomy and one was shifted to dapsone, had no response, and refused splenectomy. Side effects were tolerable. They included bloating, nausea, vomiting, insomnia, anxiety, and depression, and transient glucosuria; however, they were not severe enough to discontinue the cycles. Mean duration of illness prior to start of dexamethasone was not significantly different in between responders and nonresponders. Dexamethasone given orally in high doses is an effective drug in achieving short-term platelet responses. Long-term remission is obtained in nearly half the patients with well-established chronic ITP. Its effectiveness in almost half the patients, minimal side effects, and low cost indicate that this treatment should be considered in patients with chronic ITP who do not tolerate the disease well before considering splenectomy.     

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.     

Splenectomy in children with idiopathic thrombocytopenic purpura: A prospective study of 134 children from the Intercontinental Childhood ITP Study Group

BACKGROUND: Splenectomy is an effective procedure for children and adults with severe or refractory idiopathic thrombocytopenic purpura (ITP). Data regarding pediatric patients are limited. PROCEDURE: Sixty-eight Intercontinental Childhood ITP Study Group (ICIS) investigators from 57 institutions in 25 countries participated in a splenectomy registry. Data from 153 patients were submitted, of whom 134 had a splenectomy and were analyzed. RESULTS: The median age at splenectomy was 11.8 (2.7-20.7) years. The median postsplenectomy follow-up was 2.0 (0.1-4.5) years. Pre-splenectomy vaccination was not administered in 21 children (15.7%). Open and laparoscopic splenectomy procedures were performed in 67 and 65 evaluable children, respectively. Surgical technique was not reported in two children. Overall immediate platelet response to splenectomy was achieved in 113 patients (86.3%). Eighty percent of responders maintained their status of response during the following 4 years. Older age, longer duration of ITP, and male gender correlated with a complete response. Post-splenectomy sepsis was reported in seven patients without lethal outcome, although sepsis might be differently defined at participating institutions. CONCLUSIONS: Splenectomy is effective in children with ITP. Management varies greatly in different institutions. These Registry data may serve as a basis for future clinical trials to assess the indication and timing of splenectomy.     

腹腔镜与开腹脾切除术治疗儿童特发性血小板减少性紫癜的疗效比较

目的探讨腹腔镜脾切除术（laparoscopic splenectomy,LS）和开腹脾切除术（open splenectomy,OS）治疗儿童特发性血小板减少性紫癜（idiopathic thrombocytopenic purpura,ITP）的临床疗效。方法2003年1月至2012年2月作者实施脾切除术治疗ITP30例,其中12例采取LS,为LS组;18例采取OS,为OS组。两组术后均随访3—36个月。结果30例患儿均成功实施脾切除术,LS组和OS组平均手术时间分别为140min、118min,术中平均出血量分别为26．67mL、43．18mL,术后平均恢复饮食的时间分别为2d、2．5d,术后平均住院时间分别为8．5d、11．5d。治疗有效率：LS组为91．67％（11／12）,OS组为94．44％（17／18）,术后1周LS组和OS组平均血小板分别升高至458×10^9／L和411×10^9／L。长期随访有效率：LS组为80％,OS组为75％。结论①LS治疗ITP的疗效与OS相近,二者均可作为ITP的有效治疗措施,治疗效果与手术方式无关;②IS较OS创伤小,术后恢复快,并发症少,住院时间短,伤口小而美观。③LS治疗儿童ITP安全有效,可替代OS成为治疗儿童ITP的措施。     

PULSED HIGH-DOSE DEXAMETHASONE THERAPY IN CHILDREN WITH CHRONIC IDIOPATHIC THROMBOCYTOPENIC PURPURA

The effectiveness of pulsed high-dose oral dexamethasone therapy in children with refractory chronic idiopathic thrombocytopenic purpura (ITP) is evaluated. Thirteen children with severe chronic ITP were enrolled in the study from an outpatient pediatric hematology clinic (ages 2-14 years), 5 boys and 7 girls. They did not maintain a response to other forms of therapy (IVIg, Anti-D, conventional steroids, danazol) and one girl relapsed after splenectomy. Dexamethasone was administered orally at a dosage of 40 mg/M2/day (maximum 40 mg/day) for 4 consecutive days. The cycle was repeated once a month for 6 months. The immediate response to therapy was excellent as the mean platelet count at day 1 was 15 &#50 10 9 /L, while mean platelet count at day 4 was 158 &#50 10 9 /L. At the end of 6 cycles 3 patients maintained a platelet count of >150 &#50 10 9 /L and 4 patients showed partial response. At the end of the first year and second year (12 and 24 months after onset of treatment) 3 patients still had complete response, 3 patients had partial response, and 7 patients were failures. Six of the failures underwent splenectomy and one was shifted to dapsone, had no response, and refused splenectomy. Side effects were tolerable. They included bloating, nausea, vomiting, insomnia, anxiety, and depression, and transient glucosuria; however, they were not severe enough to discontinue the cycles. Mean duration of illness prior to start of dexamethasone was not significantly different in between responders and nonresponders. Dexamethasone given orally in high doses is an effective drug in achieving short-term platelet responses. Long-term remission is obtained in nearly half the patients with well-established chronic ITP. Its effectiveness in almost half the patients, minimal side effects, and low cost indicate that this treatment should be considered in patients with chronic ITP who do not tolerate the disease well before considering splenectomy.     

¿Cuál es el pronóstico de la trombopenia inmunitaria crónica?

ObjectivesTo analyse the characteristics of children with chronic ITP (chronic immune thrombocytopenia) in the Hospital Infantil Universitario Niño Jesús (HIUNJ) between 2003 and 2008. To also evaluate whether clinical variables as age, gender, initial platelet count, and treatment have any prognostic significance on the outcome of ITP.Patients and methodsData were retrospectively collected from 288 patients diagnosed with «Purpura and other haemorrhagic illnesses». Forty-two out of these 288 satisfied the criteria for «chronic ITP».ResultsTen patients out of 42 (23.8%) achieved remission with splenectomy, and 25 (almost 60%) achieved it without splenectomy (14 were complete remissions and 11 were partial remissions). Eight patients (almost 20% of patients with chronic ITP) had spontaneous remissions between 6 and 12 months from initial diagnosis. None of the clinical variables analysed were related to the outcome of the disease and the prognosis of the disease.ConclusionsAlmost 60% of children with chronic ITP achieve remission without treatment regardless of age, gender, initial treatment or platelet count. Splenectomy is one of the treatments with best results; however the high spontaneous recovery rate in children with cITP, the low percentage of bleeding, and the generally benign outcome should encourage delaying this as long as possible. As it is possible to have a remission between 6 and 12 months from the initial diagnosis, the term «chronic» should be reserved for patients with ITP lasting more than 1 year.     

个体化剂量丙种球蛋白联合地塞米松治疗儿童重症特发性血小板减少性紫癜78例疗效观察

目的观察个体化剂量静脉注射用丙种球蛋白(IVIG)联合地塞米松治疗儿童特发性血小板减少性紫癜(ITP)的疗效。方法重症ITP患儿入院后均给地塞米松及IVIG[400 mg/(kg.d)],3 d后测外周血血小板计数,≥100×109/L停用IVIG,继用地塞米松;若<100×109/L,再继续用原剂量IVIG 2 d。结果治疗3 d后,78例患儿中,37例外周血血小板计数升至100×109/L以上,余41例继续治疗后,31例升至100×109/L以上。结论个体化剂量IVIG联合地塞米松是治疗儿童重症ITP的有效办法,可节省一定医疗费用。     

Immune thrombocytopenic purpura in childhood in Norway: a prospective, population-based registration

A prospective, population-based registration of children with immune thrombocytopenic purpura (ITP) was performed in Norway in 1996 and 1997. Ninety-two cases were identified, indicating an incidence of 5.3 per 100,000 children under 15 years. The sex ratio (female/male) was 1.2/1. Fifty-six percent presented with cutaneous signs only. The lowest platelet count was < 20 x 10(9)/L in 91%. In spite of mild bleeding symptoms, medical treatment was given in 68%, in most cases (57/63) with intravenous immunoglobulin. A total of 41/44 patients with platelet counts of < or = 5 x 10(9)/L were treated, regardless of whether they had mucous bleedings or not. Eighteen percent had platelet counts < 150 x 10(9)/L at 6 months, and 9% at 12 months following diagnosis. One patient with therapy-resistant chronic ITP died 16 months after diagnosis from an anesthesia complication related to profound epistaxis. This study shows a relatively high incidence. As in other studies, there was a tendency to treat platelet counts rather than bleeding symptoms.     

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.     

Antiinfectious prophylaxis in asplenia

Asplenia in childhood may be congenital (e.g. Ivemark-syndrome) or acquired (functional hyposplenism in sickle cell disease; after splenectomy or bone marrow transplantation). Hereditary spherocytosis is the most common indication for splenectomy in childhood. Virtually every patient without spleen has a significantly increased risk of severe postsplenectomy infection (mostly caused by Streptococcus pneumoniae). Therefore, vaccinations against pneumococci, haemophilus influenzae and, under certain circumstances, meningococci are recommended. In addition a continuous prophylaxis with antibiotics should be performed for at least three years (or even longer depending on the disease) after splenectomy followed by lifelong interventional application of broad spectrum antibiotics in case of any unclear infection or high fever. This prophylaxis must be started as early as four months of age in sickle cell disease. In future the use of penicillin may be hampered by the growing resistance of pneumococci. Due to this fact the indication for splenectomy in childhood should be restricted to patients with hematologic disease (spherocytosis and other hemolytic anemias, chronic ITP etc.) and moderate to severe symptoms. It is unclear whether partial splenectomy for spherocytosis (and other hemolytic anemias) is an alternative regarding both longlasting reduction of hemolysis and prevention of severe infection. After trauma every effort should be undertaken to preserve a splenic remnant.     

Guidelines for therapy of idiopathic thrombocytopenic purpura (ITP) in childhood

The term "idiopathic thrombocytopenic purpura (ITP)" comprises a rather heterogeneous group of diseases with different etiology and pathogenesis. 80% of the cases are acute forms following viral infections. Within the first 6 months the spontaneous remission rate is higher than 80%. The remaining patients develop an intermittent or chronic form of the disease. Lethal complications, commonly cerebral hemorrhage, are rare (less than 2%). Following a review of the established and the recent experimental therapeutic approaches, the authors try to give comprehensive therapeutical guide-lines for the management of the various clinical forms of the disease. If there is only a minor bleeding diathesis, it is recommended to withhold therapy for 2 to 3 weeks, irrespective of the platelet count. If no spontaneous remission occurs, we suggest therapy with corticosteroids. In case of failure of this therapy, infusions of high-dose immunoglobulins are advisable. Short-lasting successes may even be prolonged with further infusions (once a week or at longer intervals)--thus postponing splenectomy. In addition, a combination of high-dose immunoglobulins and corticosteroids may be effective. Following pneumococcal vaccination and penicillin prophylaxis, splenectomy should be performed not earlier than one year after diagnosis of ITP and only in children older than 5 years. If splenectomy does not lead to recovery of the platelet count, immunoglobulin infusions--possibly combined with corticosteroids--may be repeated. Finally, if all the above proposed therapeutic actions have failed, immunosuppressive drugs (azathioprime or vincristine) may be necessary.(ABSTRACT TRUNCATED AT 250 WORDS)     

Prevention and therapy of bacterial infections for children with asplenia or hyposplenia

The asplenic state, whether functional or anatomic, is associated with an increased risk of life threatening infection or postsplenectomy sepsis (PSS). Because the risk of bacteremia with encapsulated bacteria is enhanced in children with asplenia, vaccination to prevent infection by Streptococcus pneumoniae, Haemophilus influenzae type b (Hib) and Neiserria meningitidis is recommended. Despite the increasing prevalence of penicillin-resistant S pneumoniae, prophylactic penicillin use is also recommended for children with asplenia who are younger than five years of age, and for at least one year following splenectomy. Continuation of antibiotic prophylaxis beyond these times depends on individual clinical circumstances and the prevalence of penicillin-resistant S pneumoniae in the community. When children with asplenia develop fever or nonspecific symptoms, they should be assessed immediately. If bacterial sepsis is suspected, blood and other appropriate body fluids should be cultured, and treatment should begin immediately with parenteral broad spectrum antibiotics that are also effective against the strains of S pneumoniae circulating in the community. In children with asplenia with overwhelming S pneumoniae bacteremia, the mortality rate is high, even with prompt initiation of appropriate antibiotic therapy. Thus, preventive measures are important.     

Retrospective evaluation of long-term efficacy and safety of splenectomy in chronic idiopathic thrombocytopenic purpura in children

Aim: To review the long-term efficacy and safety of splenectomy in children with chronic idiopathic thrombocytopenic purpura (cITP). Patients and methods: Data from 33 splenectomized children were retrospectively analysed (median follow up period: 18.8 y from the removal of the spleen). The median age of children at splenectomy was 12 y and the median ITP duration 3.3 y. Indications for splenectomy were: persistent severe thrombocytopenia with extensive purpura, epistaxis and/or gum bleeds, menorrhagia (n = 5) and severe or recurrent haemorrhage from various sites (n = 11). Results: Eighty-five per cent of the patients showed an excellent (n = 26) or partial response to splenectomy. Five children (15%), all females, failed to respond. Of the responders, 25% experienced a transient recurrence of thrombocytopenia within 6 mo to 4 y from splenectomy. The mortality rate due to severe sepsis was 3%. However, the majority of the splenectomized patients have not so far suffered any severe or mild bacterial infection, despite incomplete vaccination and/or antibiotic prophylaxis.

Conclusion: Splenectomy remains the only effective therapeutic modality for children with cITP, although it is associated with transient recurrence and rarely with post-splenectomy sepsis, which could be fatal. Nonetheless, splenectomy should be the last treatment option for the cITP patient, after all available therapeutic modalities have been exhausted and the child still remains profoundly thrombocytopenic and symptomatic.     

Does treatment of newly diagnosed idiopathic thrombocytopenic purpura reduce morbidity?

AIM: To explore whether early treatment of children with idiopathic thrombocytopenic purpura (ITP) with immunoglobulin and/or corticosteroids reduces subsequent morbidity. METHODS: Centres participating in a Nordic ITP study were divided according to whether they had treated more than 2/3, from 1/3 to 2/3, or less than 1/3 children within 14 days of diagnosis. The course of disease from 15 days to 6 months after diagnosis was compared for children managed at the three centre categories. The comparison was restricted to children in whom at least one platelet count <20x10(9)/l was measured, numbering 156, 143 and 84 in the three different categories, respectively. RESULTS: The three groups of children were clinically similar but were managed with initial treatment rates of 89%, 57% and 14%, respectively. By day 15, the platelet count had stabilised to >20x10(9)/l in 67%, 67% and 52% (p<0.05) and to >150x10(9)/l in 38%, 29% and 29% (p<0.20). At 1 month after diagnosis there was no difference in recovery rates. Chronic ITP developed in 27%, 22% and 25% in the three groups. During follow-up, one or more disease-related events occurred in 23%, 22% and 19%, with no difference in the average numbers of episodes with mucosal bleeding. Treatment courses were administered to 19%, 13% and 11%, respectively. CONCLUSION: Active treatment policies accelerated platelet recovery in children with short-lasting ITP but did not avert the development of chronic ITP and did not cause a reduction in morbidity during follow-up.     

Giant platelet disorders in African-American children misdiagnosed as idiopathic thrombocytopenic purpura.

A retrospective chart review of six African-American children with a diagnosis of macrothrombocytopenias (MTCP) was performed to evaluate the accuracy of their diagnosis. The following was diagnosed in the six children with MTCP: Fechtner syndrome (two children), Sebastian syndrome (one child), and unnamed MTCP (three children). In five families, chronic idiopathic thrombocytopenic purpura (ITP) was diagnosed in the propositus, which resulted in therapy using steroids, intravenous immunoglobulin (IVIG), and in one case splenectomy. Bleeding symptoms were generally mild. All six patients had thrombocytopenia ranging from 10 to 125 x 10(9)/L with mean platelet volume of 8 to 20 fL. Bleeding times were abnormal in two of three patients, and platelet aggregation was abnormal in three of four patients tested. Bone marrow aspirates were reported as increased megakaryocytes in the three patients on whom the procedure was performed. Ultrastructural morphology of platelets and leukocytes was performed in all six patients demonstrating giant platelets in all six patients and leukocyte inclusions in three patients. Differentiating MTCP from the more common ITP can be difficult but important in avoiding unnecessary diagnostic studies and potentially harmful therapy associated with ITP.     

Duration and morbidity of chronic immune thrombocytopenic purpura in children: five-year follow-up of a Nordic cohort

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.     

Randomized study of IVIg and high-dose dexamethasone therapy for children with chronic idiopathic thrombocytopenic purpura

PURPOSE: To investigate whether pulsed high-dose dexamethasone is more efficacious than intravenous immunoglobulin (IVIg) as treatment of symptomatic chronic idiopathic thrombocytopenic purpura (ITP) in childhood. METHODS: In a 2:1-randomized study, 23 children with chronic ITP received dexamethasone (0.6 mg/kg per day for 4 consecutive days once monthly for 6 months, n = 15) or IVIg (800 mg/kg intravenously once monthly for 6 months, n = 8). After four courses of treatment a crossover was offered to nonresponders. A total of 20 children received dexamethasone and 11 received IVIg. RESULTS: One of the 8 IVIg patients and 2 of the 15 dexamethasone patients achieved complete response, defined as a platelet count of at least 150 x 10(9)/L for more than 3 months without treatment. Two of the 15 dexamethasone patients achieved partial response, defined as a platelet count of at least 30 x 10(9)/L for more than 3 months without treatment. One of the 8 IVIg patients and 5 of the 15 dexamethasone patients discontinued treatment. Five patients crossed over from IVIg to dexamethasone (one complete response) and three from dexamethasone to IVIg (none responded). In summary, 5 of the 20 dexamethasone patients achieved a complete or partial response and 1 of the 11 IVIg patients achieved a complete response. Platelet counts of at least 30 x 10(9)/L by day 3 were reached in 9 of the 12 (75%) dexamethasone patients and all 8 (100%) IVIg children using available data. Five years after study completion, two of the three children who achieved a complete response and one of the two with a partial response to dexamethasone were in remission, as was the child with a complete response to IVIg. CONCLUSIONS: Treatment with pulsed high-dose dexamethasone is not always effective in children with chronic ITP, but it is worth trying in severe symptomatic chronic childhood ITP.     

Clinical characteristics of chronic idiopathic thrombocytopenia in Chinese children

PURPOSES: Clinical course and treatment outcome of childhood chronic ITP are quite variable in the literature. We report in the current paper our observation on the clinical behavior of chronic ITP in Chinese children. PATIENTS AND METHODS: We performed a retrospective review (Jan. 1990 to Dec. 2000) of children having low platelet count (plt <150 x 10(9)/L) for more than 6 months without identifiable cause. The indication for treatment was plt < or =20 x 10(9)/L. Remission is defined as plt > or =150 x 10(9)/L. RESULTS: Thirty-four children were identified within these 11 years. Their median age at diagnosis was 6.7 years (range from 0.4 to 16.8 years). The M:F ratio was 16:18. Bone marrow aspiration was performed in 30/34 cases. The median plt count at presentation was 24 x 10(9)/L (range 2 to 135 x 10(9)/L). Fourteen of 34 (41%) children eventually achieved durable remission. The chance of remission at 5 years was 66.62% with a median follow-up time of 5.86 years (range 0.72 to 10.41 years). Concerning therapy, 17/34 (50%) required no treatment while for the remaining 17, treatment included steroid (n = 16), IVIG (n = 7) or splenectomy (n = 3). In spite of temporary improvement in most, treatment induced prolonged complete remission (plt >150 x 10(9)/L) in only 2 patients. Twenty of 31 tested had abnormal immune marker(s) at presentation but none evolved into specific autoimmune disease later on. There was no correlation between the remission status, response to treatment, and the presence of autoimmune markers. CONCLUSION: About half of our chronic ITP patients achieved remission within 5 years. Medical treatment does not seem to alter the natural course of the disease but induced a transient response in most cases. Positive autoimmune markers are common among chronic ITP patients and have no significance in predicting outcome.