Vincristine therapy for severe platelet alloimmunization.

A 19-month-old girl with idiopathic severe aplastic anemia refractory to multi-agent immunosuppressive therapy developed severe platelet alloimmunization following several months of platelet transfusions. She became refractory to human leukocyte antigen (HLA)-matched platelet transfusions and experienced frequent episodes of bleeding. She was treated with intravenous vincristine administered weekly for three doses and showed marked improvement in both clinical and laboratory response to platelet transfusions. When vincristine was held for 3 weeks, she again became refractory to HLA-matched platelet transfusion. Reinstitution of vincristine resulted in cessation of clinical bleeding and improved response to platelet transfusion. The mechanism of response likely involves selective delivery of cytotoxic drug to macrophages. To our knowledge this is the first reported case of alloimmune thrombocytopenia responsive to vincristine.     

Randomized trial of high-dose methylprednisolone versus intravenous immunoglobulin for the treatment of acute idiopathic thrombocytopenic purpura in children

BACKGROUND: Idiopathic thrombocytopenic purpura (ITP) is an acquired disorder characterized by immune-mediated platelet destruction. The authors performed a prospective, randomized trial comparing intravenous immunoglobulin (IVIG) with high-dose intravenous methylprednisolone in the treatment of children with acute ITP. The primary aim of the study was to compare the rate of platelet increase produced by either intervention. A decision to treat was based on the clinical presentation and not an arbitrary platelet count. In general, enrolled patients exhibited extensive bruising and platelet counts less than 10 x 10 /L (10,000/microL). PATIENTS AND METHODS: Seventy-seven consecutive patients, for whom the attending hematologist determined acute treatment was warranted, were studied. Forty-two patients received IVIG (1 g/kg/dose x2) and 35 received methylprednisolone (30 mg/kg/dose x3). Patients who exhibited an increase in platelet count of more than 50,000/microL after the first IVIG dose or the second methylprednisolone dose did not receive the second IVIG dose or the third methylprednisolone dose, respectively. Patients' ages ranged from 6 months to 15 years. Platelet counts were evaluated at presentation, 24, 48, 72 hours, 1 week, and 2 to 4 weeks. RESULTS: Eighty percent of patients treated with IVIG and 60% of patients treated with methylprednisolone demonstrated an increase in platelet count of 50,000/microL or more within 48 hours. Both IVIG and methylprednisolone therapy increased platelet counts significantly above pretreatment values. In the methylprednisolone group, the mean baseline platelet count was 4,600/microL, which rose to 14,000/microL after 24 hours, 38,000/microL after 48 hours, and 65,000/microL after 72 hours. The IVIG group had a mean baseline platelet count of 4,200/microL, which rose to 32,000/microL after 24 hours, 69,000/microL after 48 hours, and 146,000/microL after 72 hours. When compared with methylprednisolone, IVIG therapy produced a greater rise in platelet counts at 24, 48, and 72 hours, with no difference at 1 week or later time points. No serious bleeding was noted in either treatment group. CONCLUSIONS: Both IVIG and methylprednisolone produce a significant early rise in platelet count that is somewhat greater with IVIG. However, the higher platelet counts produced by IVIG may not justify the additional cost and potential risks of this agent.     

Interferon-alpha therapy in idiopathic thrombocytopenic purpura

BACKGROUND: Acute idiopathic thrombocytopenic purpura (ITP) represents the most frequent hemorrhagic diathesis in childhood. Up to 30% of patients with ITP are regarded as refractory to standard therapy. The rare mortality from acute ITP in childhood is almost exclusively due to intracranial hemorrhage. This complication occurs in less than 1% of ITP patients. This study was designed to evaluate the effect of alpha-interferon (IFN-alpha) in eight patients whom did not respond to conventional therapy. METHOD: In spite of conventional therapies, the patient whose platelet count could not be increased to 50;10(9)/L were accepted as refractory ITP. Eight of these patients whose platelet count lower than 20;10(9)/L were included in the prospective cohort study. Interferon alpha 2b 5 MU/m(2) was administered subcutaneously three times a week, totalling 12 times in a month. According to the platelet count on the 28th day of therapy, we grouped the patients into three categories. After 60 days, the survey was re-evaluated according to the platelet count. RESULTS: The mean age of children was 3.5+/-2.5 (ranged between 3.5 and 9) years. Six of them were boys and two were girls. There was no response in one patient, partial response in one, and good response in six patients on the 28th day of therapy. The maximum rise in platelet count was observed from 7 to 14 days after the initiation of interferon. The median platelet count which was 15+/-5;10(9)/L before therapy, raised to 60+/-12;10(9)/L after therapy. However, on the 60th day of therapy, there were only two patients who had a platelet count over 100;10(9)/L. CONCLUSION: In our study, we did not observe the long-term benefit of IFN-alpha therapy in refractor ITP in childhood. However, in good responding patients, platelet levels were increased in a short time. Alpha-interferon may be alternative therapy for patients whom had a platelet count below 20;10(9)/L and not responding to standard therapy, or for patients whom immunosuppressive therapy is contraindicated.     

Intracranial hemorrhage in immune thrombocytopenic purpura: a retrospective analysis

PURPOSE: To ascertain characteristics of children with immune thrombocytopenic purpura (ITP) and intracranial hemorrhage (ICH). METHODS: The authors identified 75 published cases of ICH in children with ITP by review of the literature from 1954 to 1998. Data pertaining to the ICH was recorded for age, gender, time from diagnosis of ITP (to ICH), platelet count, head trauma or arteriovenous malformation, concomitant medications, associated infections, other bleeding manifestations, prior treatment, and outcome.RESULTS Sixty-two cases represented 6 months to 20 years of age; 65% of patients were female. The median time from the diagnosis of ITP to ICH was 32 days (range 0 days to 8 years). Fifty of 69 ICH cases (72%) occurred within 6 months of diagnosis, but only 7 (10%) occurred within 3 days of diagnosis. The platelet count was less than 10000/microL in 71.4% of the cases. Treatment prior to the ICH was primarily steroids but also included intravenous immune globulin (IVIG), splenectomy, and others (interferon, azathioprine, or vincristine). There was no difference in mortality of patients before (56%) or after (54%) 1980. CONCLUSIONS: A very low platelet count appears permissive but not sufficient for ICH to occur in children with ITP. ICH occurs more commonly in acute ITP but can occur years after diagnosis. A significant number of patients develop an ICH despite having already initiated steroid treatment of ITP.     

Successful treatment with the monoclonal antibody rituximab in two children with refractory autoimmune thrombocytopenia

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.     

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.     

Use of intravenous methylprednisolone in acute idiopathic thrombocytopenic purpura

Twenty newly diagnosed children with acute idiopathic thrombocytopenic purpura (ITP) with a platelet count (PC) of less than 20,000 were treated with intravenous methyl prednisolone (IVMP) 5 mg/kg/day in four divided doses until the platelet count increased to 50,000/mm3. They were then placed on oral prednisone at conventional doses for a total of three weeks of steroid therapy. The platelet counts increased by an average of 55,000/mm3 within 48 h of treatment. Within 72 h (day 3) of treatment all patients achieved a PC of greater than 20,000/mm3. By day 5, all 20 patients achieved a PC of greater than 50,000. IVMP is highly effective in rapidly raising the PC in acute ITP above the critical level of 20,000, thereby reducing the duration of risk for intracranial hemorrhage (ICH).     

Aggressive combination therapy in the successful management of life-threatening intracranial hemorrhage in a patient with idiopathic thrombocytopenic purpura

We describe acute therapy for a 13-year-old female from Panama with chronic idiopathic thrombocytopenia purpura (ITP) refractory to steroids, splenectomy, vinca alkaloids, and azathioprine. She presented with neurologic deterioration from a posterior fossa intracranial hemorrhage (ICH). This followed a 3-month history of severe dysfunctional uterine bleeding, progressive from menarche, which had required multiple red cell transfusions. Steroid and vinblastine therapy and transfusion of 40 U of platelets failed to increase the platelet count above 10,000/microliter. Development of a second larger ICH (frontal) produced morbid increase in intracranial pressure that necessitated neurosurgical decompression. Plasma exchange and colloid repletion with intravenous gamma globulin (1 g/kg) and an infusion of 20 U of platelets resulted in a transient rise in platelet count to 160,000/microliter, permitting surgery without bleeding. Danazol (800 mg/day) and conjugated estrogen (Premarin 25 mg/day) were begun to control the uterine bleeding. Intensive plasma exchange and i.v. IgG infusions were continued daily for 24 days, then twice weekly for several weeks. Platelet-bound IgG decreased almost 500% over the first 10 days of therapy, and platelets increased dramatically to 600,000/microliter after 3 weeks of therapy. The patient has remained amenorrheic with a normal platelet count for more than 24 months on daily danazol therapy and monthly infusions of i.v. IgG (0.4 g/kg/dose).     

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.     

High-dose dexamethasone therapy for a 14-month-old boy with refractory idiopathic thrombocytopenic purpura

A 14-month-old boy with refractory idiopathic thrombocytopenic purpura (ITP), who was successfully treated with pulsed high-dose oral dexamethasone therapy is reported. The platelet count increased after six scheduled courses of treatment (10 mg/day × 4 days, six courses). Twenty-four months later, the platelet count remained over 10.0 × 10⁴/μL. No obvious side effects were observed during or after the therapy. This treatment could be taken into consideration not only for adults but also for young children with refractory ITP. It is effective, safe, easy to administer, patient comfort is taken into consideration, and hospitalization duration and costs are minimized.     

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.     

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.     

Evaluation of clinical characteristics, diagnosis and management in childhood immune thrombocytopenic purpura: a single center's experience

Diagnostic evaluation and management in childhood immune thrombocytopenic purpura (ITP) are controversial. We reviewed the files of 162 children with ITP to evaluate clinical characteristics, response to treatment and outcome. History of antecedent infection, vaccination and serologic evidence for acute viral infection were present in 48%, 5% and 17% of the patients, respectively. At diagnosis, two-thirds of the patients had a platelet count of <10,000/microl but only 10% had major bleedings. Intracranial hemorrhage was seen in two patients (1.2%) with a mortality rate of 0.6%. Sixteen percent developed chronic ITP. The rate of platelet recovery with mega-dose methylprednisolone (30 mg/kg/d for 3 and 20 mg/kg/d for 4 days) was similar to that obtained with intravenous immunoglobulin or oral prednisolone. Four of seven patients with ITP responded to splenectomy. These data show that mode of treatment has no effect on the clinical course and prognosis of childhood ITP.     

婴幼儿特发性血小板减少性紫癜401例临床特点

目的 分析婴幼儿特发性血小板减少性紫癜(ITP)的临床特点,并比较婴儿与幼儿ITP的疗效.方法 收集401例婴幼儿ITP,均给予激素冲击治疗和静脉滴注免疫球蛋白治疗,疗效判定依据血小板计数的高低和出血症状的改善分为完全缓解、有效、无效.401例婴幼儿ITP按年龄分为婴儿组(≤1岁)和幼儿组(>1～3岁),按病程分为急性(病程≤6个月)和慢性(病程>6个月),对其临床资料进行回顾性分析,应用SPSS 12.0软件进行统计分析.结果 1.婴儿组与幼儿组均为男童比例高,但2组间性别比较差异无统计学意义(χ2=0.682,P>0.05).2.婴儿组入院时血小板中位计数低于幼儿组,差异有统计学意义(Z=2.668,P<0.05).3.婴儿组骨髓巨核细胞增高比例低于幼儿组,差异有统计学意义(χ2=16.322,P<0.001);婴儿组产板巨核细胞中位数低于幼儿组,差异有统计学意义(Z=2.065,P<0.05).4.婴儿组经治疗后血小板计数达到或超过100×109 L-1的时间短于幼儿组,差异有统计学意义(Z=3.542,P<0.001).5.急性患儿入院时血小板中位计数低于慢性患儿,差异有统计学意义(Z=2.100,P<0.05).6.输注血小板患儿的住院时间与未输注血小板患儿相比,差异无统计学意义(Z=1.385,P>0.05).结论 婴幼儿ITP中,男童比例高,大部分无明显诱因,以皮肤黏膜出血为主;婴儿入院时血小板中位计数较低,血小板上升至正常的时间较短,对治疗反应较幼儿好,幼儿急性ITP可以变为慢性;输注血小板并不能缩短ITP患儿的住院时间.     

Idiopathic thrombocytopenic purpura: a 10-year natural history study at the childrens hospital of alabama

Childhood idiopathic thrombocytopenic purpura (ITP) is a common disorder. However, single-institution, long-term, natural history data are limited. The objective of this paper is to review presenting features, response to therapy, and natural history of ITP treated at a single pediatric academic medical center. A retrospective chart review was made for all children (ages birth-18 years) diagnosed with ITP (ICD 287.3) and treated at the Childrens Hospital of Alabama/University of Alabama at Birmingham between 1993 and 2003. Four hundred nine patients were identified (49% male, 51% female; mean age: 5.85 years; range: 1 month-17 years). There was no seasonal variation of presentation. The mean platelet count was 19k (0-120k). Bone marrow aspiration (BMA) was performed in 72% but altered the diagnosis or therapy in no patient. Treatment consisted of corticosteroids in 256 (92% response), intravenous immunoglobulin (IVIG) in 125 (87% response), Win-Rho D in 58 (91% response), and no therapy in 71 (100% response). Response was defined as increase in platelet count to > 50k. There was no difference in response to any therapy. No patients died. One patient presented with a CNS hemorrhage at presentation, responded to therapy, and survived. Twenty-three of 409 patients (6%) experienced clinical bleeding requiring hospitalization or blood transfusion. Chronic ITP (persistence > 6 months) was noted in 99 patients (24%). Chronic patients presented at an older age (7.8 vs 5.2 years for acute only, p<0.001), and with higher platelet counts (27k vs 17k, p<0.001). The risk of chronic ITP was partially predicted by presenting platelet count > 50k and age > 10 years, or both; 50% of patients presenting with these features developed chronic ITP vs 24% overall rate. Splenectomy was curative in 30/31 (97%) patients. There was no postsplenectomy sepsis. Of 99 patients with chronic ITP, 25 responded to splenectomy, 37 resolved at a mean of 20.3 months after diagnosis (7-96 months), 36 had persistent mild thrombocytopenia (50k-125k), and 1 failed to respond to any treatment including splenectomy. Overall, 91% of cases resolved with therapy or observation. ITP is a common pediatric disease presenting at any age with low morbidity and mortality. Most cases can be managed by pediatricians without hematology referral. Several equally successful therapeutic options exist. Chronic cases present at an older age with higher platelet counts. Up to 50% of cases of chronic ITP will resolve with ongoing follow-up. The overall prognosis in childhood ITP is excellent.     

Diamond-blackfan anemia and cyclosporine therapy revisited

A girl with Diamond-Blackfan anemia diagnosed in infancy started cyclosporine A (CSA) therapy at 9 years and 8 months of age after experiencing unacceptable side effects while receiving prednisone. Since then, she has been followed-up for more than 4 years. She exhibited a dramatic response to CSA, with weaning and then cessation of steroid therapy after 5 months. She has remained transfusion-independent. Attempts to discontinue CSA therapy have been unsuccessful. Relapse of the anemia has occurred in the context of viral infections with missed CSA doses. The major clinical problem during treatment has been recurrent oral aphthous ulceration, which responds to topical therapy. She is currently maintained on CSA 100 mg twice daily with a hemoglobin of 10.2 g/dL and a reticulocyte count of 1.6%. A trial of CSA therapy should be considered in patients with Diamond-Blackfan anemia in whom steroid therapy has failed before a transfusion program is instituted or alternative donor stem cell transplantation is entertained.     

A cost-utility analysis of treatment for acute childhood idiopathic thrombocytopenic purpura (ITP)

BACKGROUND: The primary objective in the treatment of acute pediatric idiopathic thrombocytopenic purpura (ITP) is to rapidly increase the platelet count. METHODS: We built a decision analytic model to evaluate the cost-utility of four commonly used treatment strategies: intravenous immunoglobulin G (IVIG) 0.8 g/kg, anti-D 75 mcg/kg, methylprednisolone (30 mg/kg for 3 days), and prednisone (4 mg/kg/day for 4 days). In our baseline model, all children were hospitalized upon presentation, and discharged once the platelet count reached > or =20,000. We performed a literature search to estimate time to platelet count > or =20,000 for each strategy, as well as the probability of side effects. We obtained cost data and quality of life measures from institutional and published data sources. RESULTS: Total cost of one-time treatment for a 20 kg child was US dollars 786 with prednisone, US dollars 1,346 with methylprednisolone, US dollars 2,035 with anti-D, and US dollars 2,492 with IVIG. The strategies of IVIG and methylprednisolone were less effective and more expensive than anti-D and prednisone, respectively. Although anti-D caused the most rapid rise in platelet counts, the incremental cost-utility ratio (costs incurred by using anti-D instead of prednisone divided by health benefit of using anti-D instead of prednisone) was US dollars 7,616 per day of severe thrombocytopenia avoided, primarily due to the much higher medication cost of anti-D. Utilizing an outpatient model, the cost difference between anti-D and prednisone was even more striking. CONCLUSIONS: The clinical benefit of anti-D is offset by a substantial cost increase. Although often overlooked in favor of newer agents, a brief course of high-dose prednisone is an inexpensive and effective treatment for acute ITP.     

Short term efficacy of intravenous dexamethasone and methylprednisolone therapy in steroid resistant nephrotic syndrome

OBJECTIVE: To compare the short term efficacy of intravenous pulses of methylprednisolone and dexamethasone in treatment of steroid resistant nephrotic syndrome in children. METHOD: We prospectively treated 81 children with idiopathic steroid resistant nephrotic syndrome with six alternate-day pulses of intravenous dexamethasone (5 mg/kg) or methylprednisolone (30 mg/kg). Fifty-nine patients received dexamethasone and 22 were treated with methylprednisolone. Two patients in dexamethasone and one in methylprednisolone group developed serious infection during administration of alternate-day pulses and could not complete the therapy. RESULTS: The median age at treatment was 38 (36-74.7) months. Of patients who completed therapy, 20 (35.1 percent) (95 PERCNT CI 22.9-48.9) and 7 (33.1 percent) (95 percent CI 14.6-56.9) patients in dexamethasone and methylprednisolone group, respectively achieved complete remission. Following alternate day pu1ses the median urinary albumin to creatinine ratio decreased from 9.2 to 1.5 (P less tha 0.005) in dexamethasone group and from 12.1 to 0.7 (P less than 0.005) in methylprednisolone group. The median reduction in urinary albumin to creatinine ratio was 54.1 PERCNT (95 percent CI 32.7- 83.9) and 63.2 percent (95 percent CI 23.5- 100) in dexamethasone and methylprednisolone group respectively. The chief side effects of therapy were transient hypertension or worsening of preexisting hypertension, which occurred in 31 (54.4 percent) patients in dexamethasone group and 10 (47.6 percent) in the methylprednisolone group. The hypertension was satisfactorily controlled on antihypertensive drugs. One or more side effects were observed in 66.7 percent (95 percent CI 52.9-78.6) children receiving dexamethasone therapy and 61.9percent (95 percent CI 38.4-81.9) receiving methylprednisolone, which was comparable. CONCLUSIONS: We conclude that intravenous dexamethasone is as effective as methylprednisolone in inducing remission in patients with steroid resistant nephrotic syndrome.     

Treatment of an infant with severe acute refractory immune thrombocytopenic purpura using combination therapy including rituximab

Some infants with acute immune thrombocytopenic purpura (ITP) do not respond to first‐line therapy, and currently there is no consensus on therapy for these refractory cases. We describe a 12‐week‐old infant with acute ITP who was unresponsive to intravenous immunoglobulin and corticosteroid, and developed gastrointestinal bleeding. Several combination therapies were unsuccessful. After four doses of rituximab followed by intravenous immunoglobulin and corticosteroid, his platelet counts gradually increased. Combined therapy which includes rituximab may be a promising treatment for severe acute refractory ITP. Pediatr Blood Cancer 2009;53:203–205. © 2009 Wiley‐Liss, Inc.     

Successful α-interferon therapy in a child with chronic refractory idiopathic thrombocytopenic purpura: A case report

We treated a sixteen month old male with chronic refractory idiopathic thrombocytopenic purpura (ITP) in whom α-interferon (IFN) therapy was effective. He developed ITP which did not respond to various treatments. Six months after admission, we began to treat him with IFN. The patient's platelet count rapidly responded to the therapy and rose above normal range. Serum levels of platelet associated immunoglobulin G (PA-IgG) showed a tendency to decrease with the administration of IFN. After stopping the IFN therapy for a duration of 3 months, the platelet count remained normal. No serious adverse side effects, except transient fever, were observed. From the experience of this case we propose that IFN is one of the therapeutic options for treatment of refractory ITP not only in adults but also in children.