Long-Term Safety and Efficacy of Oral Eltrombopag for the Treatment of Subjects with Idiopathic Thrombocytopenic Purpura (ITP): Preliminary Data from the EXTEND Study.

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 566-566 ◽  
Author(s):  
James B. Bussel ◽  
Gregory Cheng ◽  
Lidia Kovaleva ◽  
Mansoor N. Saleh ◽  
Balkis Meddeb ◽  
...  
Keyword(s):  
Adverse Event ◽  
Platelet Count ◽  
Idiopathic Thrombocytopenic Purpura ◽  
Who Grade ◽  
Thrombocytopenic Purpura ◽  
Safety And Efficacy ◽  
Platelet Counts ◽  
Stage 4 ◽  
Stage 1

Abstract Idiopathic thrombocytopenic purpura (ITP) is a disease caused by inadequate platelet production as well as increased platelet destruction. Eltrombopag is a first-in-class, oral, non-peptide platelet growth factor that increases platelet counts by interacting with the thrombopoietin receptor on megakaryocytes and their precursors. Accordingly, in two completed 6-week, randomized, double-blind, placebo-controlled studies of adult subjects with chronic ITP, eltrombopag produced a substantial dose-dependent increase in platelet counts. EXTEND is an ongoing, open-label extension study designed to assess the long-term safety and efficacy of oral eltrombopag. Subjects previously enrolled in an eltrombopag study are eligible to enroll in EXTEND after an intervening washout period of at least 4 weeks. Subjects are administered a starting dose of 50 mg/day (which could be increased to 75 mg/day at any time after 3 weeks) in order to reach a platelet count of ≥50,000/uL (stage 1). Then, concomitant ITP medications, if taken at study entry, are tapered to a minimal dose or discontinued entirely (stage 2), whilst maintaining a platelet count of ≥50,000/uL. Eltrombopag is then titrated to a minimal effective dose (25–75 mg/day) required to maintain platelet counts of 50,000/uL-200,000/uL (stage 3). Eltrombopag is continued for as long as the subject continues to benefit (stage 4). Bleeding incidence and severity is assessed using the WHO bleeding scale (Grade 0–4). As of August 6, 2007, data were available on 96 subjects. Ninety-four subjects were administered eltrombopag. Evaluable subjects (n=89) had a median treatment duration of 151 days (2–333 days). At baseline, 42 (44%) subjects had a platelet count ≤15,000/uL, 60 (63%) had evidence of bleeding (WHO Grade 1-4), 44 (46%) were splenectomized, and 35 (36%) were receiving concomitant ITP treatment. Of the sixty-one subjects who entered into the study with a platelet count <30,000/uL, 43 (73%) achieved a platelet count of ≥50,000/uL while on study; 10 of the 61 subjects had at least one count ≥400,000/mL during the study. Of the 94 subjects who received at least one dose of eltrombopag, 78 (83%) reported at least one adverse event; 30 (32%) reported a drug-related adverse event (AE). Most AEs were mild in severity with the most common being headache (20%). Twelve (13%) subjects reported a serious adverse event. Two deaths were reported (traffic accident and hypovolaemic shock), both not related to study medication. To date, these findings of the EXTEND study suggest that eltrombopag is well tolerated and sustains increased platelets counts during long-term treatment. Stage Description Subjects entering Median Platelet counts (/uL) WHO Grade 2–4 Bleeding n (%) Stage 1 eltrombopag administered 94 16,000 25 (27%) Stage 2 tapering ITP con meds 17 143,500 4 (24%) Stage 3 titrating eltrombpag to maintain platelet counts 46 108,500 3 (7%) Stage 4 treating with eltrombopag long-term 27 104,000 1 (4%)

Safety and Efficacy of Long-Term Treatment with Oral Eltrombopag for Chronic Idiopathic Thrombocytopenic Purpura.

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 3432-3432 ◽  
Author(s):  
James B Bussel ◽  
Gregory Cheng ◽  
Mansoor N Saleh ◽  
Balkis Meddeb ◽  
Christine Bailey ◽  
...  
Keyword(s):  
Idiopathic Thrombocytopenic Purpura ◽  
Term Treatment ◽  
Thrombocytopenic Purpura ◽  
Once Daily ◽  
Safety And Efficacy ◽  
Platelet Counts ◽  
Chronic Itp ◽  
Long Term Treatment ◽  
Previously Treated

Abstract INTRODUCTION: Eltrombopag (PROMACTA®/REVOLADE®; GlaxoSmithKline, Collegeville, PA) is the first oral, small molecule, non-peptide thrombopoietin receptor agonist under investigation for the treatment of thrombocytopenia due to various causes, including idiopathic thrombocytopenic purpura (ITP). Chronic ITP is characterized by autoantibody-induced platelet destruction and reduced platelet production, leading to chronically low peripheral platelet counts. Eltrombopag treatment has previously demonstrated a significant increase in platelet counts and a reduction in clinically relevant bleeding symptoms in 2 placebo-controlled trials evaluating a total of >200 patients with chronic ITP after up to 6 weeks of treatment. EXTEND is an ongoing open-label, phase III extension study to assess the long-term safety and efficacy of oral eltrombopag in ITP patients that have previously completed an eltrombopag trial. METHODS: Patients with previously treated, chronic ITP who completed a prior eltrombopag study were eligible to participate in EXTEND. Eltrombopag treatment was initiated at 50 mg once daily and then adjusted in order to maintain platelet counts ≥ 50,000/μL and <400,000/μL, with doses between 75 mg once daily and 25 mg once daily or less often than once daily, if necessary. Patients who achieved platelet counts ≥ 50,000/μL during treatment with eltrombopag were considered responders. Bleeding events were prospectively evaluated using the WHO Bleeding Scale: Grade 0 = no bleeding, Grade 1 = mild bleeding, Grade 2 = moderate bleeding, Grade 3 = gross bleeding and Grade 4 = debilitating blood loss. RESULTS: At the time of this analysis, 207 patients (median age, 50 years; 67% female) had received eltrombopag on this study. At baseline, 33% were receiving concomitant ITP medication and 40% were splenectomized. The majority of patients (70%) enrolled with baseline platelet counts <30,000/μL, followed by 18% and 12% with baseline platelet counts from ≥ 30,000/μL to ≤ 50,000/μL, and >50,000/μL, respectively. The duration of eltrombopag treatment ranged from 3 to 523 days. Seventy-nine percent (159/201) of patients achieved a platelet count ≥ 50,000/μL, and 24% (18/75) of patients who had received eltrombopag for at least 25 weeks maintained platelet counts ≥ 50,000/μL continuously for ≥ 25 weeks. Patients responded to eltrombopag regardless of splenectomy status (non-splenectomized: 78%, splenectomized: 81%) and use of baseline concomitant ITP medications (no baseline ITP medications: 79%, baseline ITP medications: 80%). Median platelet counts remained ≥ 50,000/μL throughout the observation period of the study (Figure 1) with only 3 exceptions, when the median platelet counts remained >40,000/μL. At baseline, 59% of patients reported bleeding symptoms (WHO Grades 1–4) compared with approximately 30% at months 1, 3, and 6. Adverse events (AEs) were reported in 150 patients (72%) while on therapy, the majority of which were mild to moderate. Headache (15%) was the most commonly reported on-therapy AE, followed by upper respiratory tract infection (13%), diarrhea (10%), and nasopharyngitis (9%). Six thromboembolic events were reported during the study. No clinically relevant effects of eltrombopag on patient bone marrow were detected. Thirty-nine serious AEs were reported by 17 patients (8%) while on therapy +1 day. Four deaths were reported in the study (2 deaths on therapy and 2 deaths >30 days after the last dose of eltrombopag); none were considered related to study medication. CONCLUSION: Oral eltrombopag is effective at raising platelet counts and decreasing bleeding symptoms during long-term treatment, regardless of splenectomy status or the use of baseline ITP medications. Eltrombopag is well tolerated during long-term treatment in patients with previously treated chronic ITP. Figure 1. Median platelet counts.a
 BL, median baseline value.
 aDotted line indicates 50,000 platelets/μL. Figure 1. Median platelet counts.a
 BL, median baseline value.
 aDotted line indicates 50,000 platelets/μL.

Multiple Cycles of Recombinant Human Thrombopoietin Therapy in a Patient with Chronic Refractory Idiopathic Thrombocytopenic Purpura.

Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 3933-3933
Author(s):  
Yongqiang Zhao ◽  
Baolai Hua ◽  
Nong Zou ◽  
Shujie Wang ◽  
Tienan Zhu
Keyword(s):  
Platelet Count ◽  
Idiopathic Thrombocytopenic Purpura ◽  
Immunosuppressive Agents ◽  
Plasma Concentrations ◽  
Thrombocytopenic Purpura ◽  
Platelet Production ◽  
Platelet Counts ◽  
Multiple Cycles ◽  
Recombinant Human Thrombopoietin ◽  
Refractory Itp

Abstract Thrombopoietin (TPO) is the key regulator of megakaryocytepoiesis and platelet production. TPO binds to its specific receptor, c-Mpl, on the surfaces of megakaryocytes, and may promote the proliferation, differentiation and maturation of megakaryocytes, and finally increase the circulating platelet count. The role of TPO in the pathogenesis of idiopathic thrombocytopenic purpura (ITP) is not certain. Plasma concentrations of TPO in ITP patients were similar to or little lower than that in healthy subjects. Therefore it is possible that supplemental TPO could significantly promote platelet production and increase platelet counts in ITP patients. Here, we report the result of multiple cycles of recombinant human thrombopoietin (rhTPO) therapy in a patient with refractory ITP. The patient, a 42-year-old woman, was admitted to our department on December 30, 2003. She had suffered from chronic ITP for more than 4 years. The patient had been treated with glucocorticosteroids, immunosuppressive agents and splenectomy. No sustained response could be achieved. The diagnosis of chronic refractory ITP was made. There were petechiae and gingival bleeding on admission. Liver and spleen were not palpable. Hemoglobin was 142g/L, white blood cell count 7.6×10 9/L, platelet count 15×10 9/L. Bone marrow aspiration revealed that erythroid and myeloid development were normal, megakaryocytes were increased in number and no dysplastic features. After an informed consent was obtained from the patient, rhTPO (Sunshine Pharmaceutical Corporation, China) was administrated subcutaneously at dosage of 1.0 μg/kg, daily for 14 days or until platelet count sustained more than 50×109/L. Anti-rhTPO antibodies were determined weekly by ELISA. Three cycles of rhTPO therapy was given with 6, 13 and 8 dosing for each cycle. The platelet counts before each cycle were all less than10×109/L and increased above 50×109/L on day 5, 11 and 8 of rhTPO administration, respectively. The peak platelet counts of 456, 130 and 82×109/L were reached on day 9, 15 and 13 for each cycle. Then platelet count decreased gradually. The durations of platelet count more than 50×109/L in 3 cycles were 13, 7 and 10 days respectively. No increase of WBC count and Hb level occurred. No liver and kidney function damage, abnormal coagulation functions or thrombosis developed during the treatment. rhTPO antibodies were not detectable. The result indicated that rhTPO could transiently increase peripheral platelet counts of the patient with chronic refractory ITP. It was uncertain why peak platelet counts declined and durations of platelet count more than 50×109/L shortened when multiple cycles of rhTPO were given.

Safety and Efficacy of Laparoscopic Splenectomy in Patients with Refractory Immune Thrombocytopenic Purpura. A Long-Term Survey

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 4548-4548
Author(s):  
Nicola Cascavilla ◽  
Matteo Scaramuzzi ◽  
Michele Nobile ◽  
Matteo Dell’Olio ◽  
Antonietta Pia Falcone ◽  
...  
Keyword(s):  
Platelet Count ◽  
Immune Thrombocytopenic Purpura ◽  
Laparoscopic Splenectomy ◽  
New Drugs ◽  
Thrombocytopenic Purpura ◽  
Safety And Efficacy ◽  
Haematological Response ◽  
Short And Long Term

Abstract Background: Despite the popularity of splenectomy has decreased dramatically in the past few years, the surgical approach remains the best therapy for patients with refractory Immune Thrombocytopenic Purpura (ITP) in terms of high and durable rate of response (Vesely et al, Ann Intern Med2004; 140: 112). The recent introduction of anti-CD20 antibodies and thrombopoietins of second generation such as AMG 531 and Eltrombopag may have a relevant role (Kuter et al, Lancet2008; 371: 362) but their long-term safety and efficacy have not been still established. In parallel with new drugs, there has been an evolution in the surgery of splenectomy as well (Dolan et al, Am J Hematol2008; 83: 93). Actually, the laparoscopic surgery is considered the standard approach and the ITP represents the most common indication in 50–80% of all the laparoscopic splenectomies. Methods: The aim of this study is to evaluate the long-term complete and partial haematological response (CR + PR), as well as the short and long-term complications, of 40 patients (30 females and 10 males; median age: 38 years - range 6–71) with unresponsive ITP after one or more medical approaches and underwent laparoscopic splenectomy at our Institution from 1999 through 2006. The 40 patients accounted for 22.2% of 181 patients diagnosed in those years. An abdominal CT scan to evaluate the presence of accessory spleens was performed in all cases. All patients received meningococcal, pneumococcal and haemophilus influenzae vaccine one week before splenectomy. For 4 or 5 days before splenectomy the patients were treated with high doses of intravenous Immunoglobulins. Anti-thrombotic prophylaxis was performed with low molecular weight heparin (LMWH) for 10 days and afterwards with cardioaspirin (ASA) if the platelet count exceeded 500x10E9/L. Results: No cases required conversion to laparotomic splenectomy. An accessory spleen was found in 2 patients (5%). Immediate haematological response rate was of 100%. At date, after a median follow-up of 78 months (range 28–112 months), 36 patients (90%) remain in CR or PR with a platelet count more than 50x10E9/L and 2 patients are taking ASA. Four patients (10%) relapsed; out of which, 2 patients have a platelet count less than 10x10E9/L. Short and long-term mortality rate was 0%. Immediate postoperative complications rate was 5%: we observed 2 cases of hemoperitoneum related to a trocar’s tube and to an active bleeding, respectively, both resolved with new laparoscopic approach. The mean postoperative hospital stay was 4,5 days (range 4–8). Neither cases of bacterial sepsis in the postoperative or during the follow-up time, nor cases of splenic-portal vein thrombosis (SPVT) and no cases of neoplasms occurred. Conclusions: Our experience suggests that laparoscopic splenectomy is an excellent approach to patients with refractory ITP in terms of safety, efficacy and costs. With respect to laparotomic splenectomy, the use of laparoscopy is likely to make the splenectomy even safer and therefore suitable for a larger number of patients. Undoubtedly there is a great expectation for the new drugs (Rodeghiero et al, Am J Hematol2008; 83: 91) and we agree that only controlled comparative clinical trials (Vianelli et al, Haematologica2005; 90: 72) will be able or not to say a final word and to challenge the role of splenectomy.

scholarly journals Treatment of adult chronic autoimmune thrombocytopenic purpura with repeated high-dose intravenous immunoglobulin

Blood ◽  
1993 ◽  
Vol 82 (5) ◽  
pp. 1415-1421 ◽  
Author(s):  
B Godeau ◽  
S Lesage ◽  
M Divine ◽  
V Wirquin ◽  
JP Farcet ◽  
...  
Keyword(s):  
Body Weight ◽  
Platelet Count ◽  
Treatment Protocol ◽  
Complete Response ◽  
Long Term Results ◽  
High Dose ◽  
Thrombocytopenic Purpura ◽  
Autoimmune Thrombocytopenic Purpura ◽  
Platelet Counts

Intravenous (i.v.) infusions of Ig concentrates are an effective but expensive treatment for patients with autoimmune thrombocytopenic purpura (AITP). The optimal treatment protocol and the long-term results are uncertain, and the precise mechanism by which the platelet count increases is poorly understood. Twenty adult patients with chronic AITP were enrolled in a prospective study to compare the respective efficacy of two high-dose IVIgG induction regimens (1 g v 2 g/kg body weight) and the long-term effect of six 1 g/kg body weight i.v. IgG reinfusions. An initial response was observed in all 18 evaluable patients: the platelet count increased to a mean value of 251 x 10(9)/L (range 72 to 836 x 10(9)/L) and the mean pretreatment platelet count was multiplied by 14.6. No difference in efficiency was observed between the two i.v. IgG dosages. The degree of the platelet count increment correlated in both groups with the increase in the clearance of antibody-coated red blood cells, measured by an isotopic method, but not with the serum IgG elevation. Treatment was considered to have failed in 11 patients, 90 days after the last i.v. IgG reinfusion (D90), because the platelet counts were comparable with pretreatment values. In contrast, a complete response was observed at D90 in five patients (mean platelet count: 184 x 10(9)/L; range: 150 to 250 x 10(9)/L) and a partial response at D90 was obtained in the remaining two patients (platelet counts: 70 and 104 x 10(9)/L). Five of the 7 responders at D90 kept a platelet count above 50 x 10(9)/L during the entire follow-up period (mean 33 months; range: 5 to 66) with no further treatment; unfortunately, no clinical or biologic criteria were found to be predictive of the long-term response. This study shows that an i.v. IgG infusion regimen of 1 g/kg body weight could safely replace the classical 2 g/kg body weight dosage, at least in patients who do not have life-threatening thrombocytopenia. Moreover, repeated i.v. IgG reinfusion could be an alternative for AITP patients in whom splenectomy is contraindicated.

scholarly journals Long term follow up of splenectomised patients with idiopathic thrombocytopenic purpura

2002 ◽  
Vol 49 (3) ◽  
pp. 29-34 ◽  
Author(s):  
Ivo Elezovic ◽  
Darinka Boskovic ◽  
Milica Colovic ◽  
Dragica Tomin ◽  
Nada Suvajdzic-Vukovic ◽  
...  
Keyword(s):  
Platelet Count ◽  
Idiopathic Thrombocytopenic Purpura ◽  
Immune Globulin ◽  
Response Rate ◽  
Long Term Results ◽  
Intravenous Immune Globulin ◽  
Definitive Treatment ◽  
Thrombocytopenic Purpura

Splenectomy is definitive treatment for idiopathic thrombocytopenic purpura (ITP) because it removes both the sites of autoantibody producing cells and also the major site of platelet destruction. The purpose of this study was to evaluate long term results of splenectomised patients with ITP and to determine predictor factors for good response. A 167 patients with chronic ITP (136 females, 31 males), median aged 35 years (17-74) was splenectomised after 2 to 160 months (Median 12) from diagnosis of ITP. Indications for splenectomy were: 6 weeks of steroid therapy with platelet count below 10x10^9/l or 3 months with platelet count under 30xl0^9/l, or treatment with prednisone above 30 mg more of 6 months to increase platelet count over 30x10^9/l, or repeated relapses. Postoperative complications developed in 16 patients (9.5%), 3 of them died (1.8%) due to thromboembolism and 17 patients discontinued later controls. During follow up to 172 months (Median 62) 111/147 splenectomised patients were in remission (75.5%), 99 in complete (above 100x10^9/l), 12 in partial (50-100x109/l) and 36 patients (24.5%) were relapsed (below 50x10^9/l). Remission was achieved in 79/88 patients (89.8%) with good response to prednisone before splenectomy toward 32/62 patients (51.6%) with poor response to prednisone (p<0.01). Remission was obtained in 9/11 patients (81.8%) who responded well to intravenous immune globulin (0.4 g/kg x 5d) and only in 1/8 who did not (p<0.05). Higher response rate was achieved in patients under 40 years of age (81.6%) than in older ones (63.4%) (p<0.05). No difference was shown between sex and time intervals (3, 6, 12, 24, 36 or over 36 months) from diagnosis to splenectomy. Splenectomy is an effective treatment of refractory ITP with response rate of 75.5% after median follow up of 62 months. In our patients better results on splenectomy were associated with age less than 40 years, good responses to steroid, and intravenous immune globulin.

scholarly journals Treatment of adult chronic autoimmune thrombocytopenic purpura with repeated high-dose intravenous immunoglobulin

Blood ◽  
1993 ◽  
Vol 82 (5) ◽  
pp. 1415-1421 ◽  
Author(s):  
B Godeau ◽  
S Lesage ◽  
M Divine ◽  
V Wirquin ◽  
JP Farcet ◽  
...  
Keyword(s):  
Body Weight ◽  
Platelet Count ◽  
Treatment Protocol ◽  
Complete Response ◽  
Long Term Results ◽  
High Dose ◽  
Thrombocytopenic Purpura ◽  
Autoimmune Thrombocytopenic Purpura ◽  
Platelet Counts

Abstract Intravenous (i.v.) infusions of Ig concentrates are an effective but expensive treatment for patients with autoimmune thrombocytopenic purpura (AITP). The optimal treatment protocol and the long-term results are uncertain, and the precise mechanism by which the platelet count increases is poorly understood. Twenty adult patients with chronic AITP were enrolled in a prospective study to compare the respective efficacy of two high-dose IVIgG induction regimens (1 g v 2 g/kg body weight) and the long-term effect of six 1 g/kg body weight i.v. IgG reinfusions. An initial response was observed in all 18 evaluable patients: the platelet count increased to a mean value of 251 x 10(9)/L (range 72 to 836 x 10(9)/L) and the mean pretreatment platelet count was multiplied by 14.6. No difference in efficiency was observed between the two i.v. IgG dosages. The degree of the platelet count increment correlated in both groups with the increase in the clearance of antibody-coated red blood cells, measured by an isotopic method, but not with the serum IgG elevation. Treatment was considered to have failed in 11 patients, 90 days after the last i.v. IgG reinfusion (D90), because the platelet counts were comparable with pretreatment values. In contrast, a complete response was observed at D90 in five patients (mean platelet count: 184 x 10(9)/L; range: 150 to 250 x 10(9)/L) and a partial response at D90 was obtained in the remaining two patients (platelet counts: 70 and 104 x 10(9)/L). Five of the 7 responders at D90 kept a platelet count above 50 x 10(9)/L during the entire follow-up period (mean 33 months; range: 5 to 66) with no further treatment; unfortunately, no clinical or biologic criteria were found to be predictive of the long-term response. This study shows that an i.v. IgG infusion regimen of 1 g/kg body weight could safely replace the classical 2 g/kg body weight dosage, at least in patients who do not have life-threatening thrombocytopenia. Moreover, repeated i.v. IgG reinfusion could be an alternative for AITP patients in whom splenectomy is contraindicated.

scholarly journals PLATELET COUNT RESPONSE TO HELICOBACTER PYLORI ERADICATION IN IRANIAN ‎PATIENTS WITH IDIOPATHIC THROMBOCYTOPENIC ‎PURPURA

2012 ◽  
Vol 4 (1) ◽  
pp. e2012056 ◽  
Author(s):  
Mohammad Erfan Zare
Keyword(s):  
Helicobacter Pylori ◽  
Platelet Count ◽  
Idiopathic Thrombocytopenic Purpura ◽  
Eradication Therapy ◽  
Complete Response ◽  
Thrombocytopenic Purpura ◽  
Platelet Counts ◽  
Count Response ◽  
Significant Difference ◽  
H Pylori

Idiopathic thrombocytopenic purpura (ITP) is an autoimmune hematological disordercharacterized by auto antibody-mediated platelet destruction. Although the main cause of ITPremains unclear, but its relationship with some infection was demonstrated. In recent years, many studies have demonstrated improvement of platelet counts in ITP patients after treating Helicobacter pylori infection. The aim of this study was to investigate the effects of H. pylori eradication on platelet count response in Iranian ITP patients.A total of 26 patients diagnosed with both ITP and H. pylori infection. ITP were diagnosed whose platelet counts were less than 100×103/μL. These patients were tested for H. pylori infection by Urea Breath Test and serum H. pylori antibody. All patients received triple therapy for 7 or 14 days to eradicate H. pylori infection. These patients followed for six months.Prevalence of H. pylori was 67.3%. H. pylori eradication achieved in 89.5% (26/29). Of the 26 patients, 15 (57.7%) exhibited a complete response (CR) and 11 (42.3%) were unresponsive. We did not find partial responders. There was a significant difference in the baseline platelet count of responders and non-responders patients (p<0.001). All responders had platelet count ≥50×103/μLand all non-responders had platelet count <50×103/μL.Results of this study revealed that eradication therapy of H. pylori infection can improve platelet counts in ITP patients especially with mild thrombocytopenia and support routine detection andtreatment of H. pylori infection in ITP patients in populations with a high prevalence of this infection.

Morbidity and mortality in adults with idiopathic thrombocytopenic purpura

Blood ◽  
2001 ◽  
Vol 97 (9) ◽  
pp. 2549-2554 ◽  
Author(s):  
Johanna E. A. Portielje ◽  
Rudi G. J. Westendorp ◽  
Hanneke C. Kluin-Nelemans ◽  
Anneke Brand
Keyword(s):  
General Population ◽  
Idiopathic Thrombocytopenic Purpura ◽  
Mortality Risk ◽  
Hospital Admissions ◽  
Morbidity And Mortality ◽  
Refractory Disease ◽  
Thrombocytopenic Purpura ◽  
Platelet Counts

Abstract To study outcomes of adults with idiopathic thrombocytopenic purpura (ITP), we performed a follow-up study in a cohort of 152 consecutive patients who were treated according to a well-defined algorithm. Long-term outcomes were determined relative to the response 2 years after diagnosis, because most (93%) patients who ultimately attained platelet counts above 30.0 × 109/L (30 000/μL) did so within this time frame. Complete follow-up for mortality could be studied in 99% of patients and for morbidity in 95% of patients, with a mean of 10.5 years. Within 2 years after diagnosis, 4 patients died, 2 were lost to follow-up, and 12 were reclassified as having secondary immune thrombocytopenia. Of the remaining 134 patients, 114 (85%) had obtained platelet counts above 30.0 × 109/L while all therapies had been discontinued. These patients had a long-term mortality risk equal to the general population. Twelve of 134 patients (9%), all with severe thrombocytopenia, had refractory disease and suffered a mortality risk of 4.2 (95% confidence interval, 1.7-10.0). Bleeding and infection equally contributed to the death of these patients. Another 8 patients (6%) had platelet counts above 30.0 × 109/L while on maintenance therapy. Similar to patients with refractory disease, these latter patients had considerably increased ITP-related hospital admissions, but mortality was only slightly higher than in the general population. In conclusion, most adults with ITP have a good outcome with infrequent hospital admissions and no excess mortality. The absence of gross morbidity and mortality in patients with moderate thrombocytopenia supports clinical practice refraining from further treatment.

A retrospective 11-year analysis of obstetric patients with idiopathic thrombocytopenic purpura

Blood ◽  
2003 ◽  
Vol 102 (13) ◽  
pp. 4306-4311 ◽  
Author(s):  
Kathryn E. Webert ◽  
Richa Mittal ◽  
Christopher Sigouin ◽  
Nancy M. Heddle ◽  
John G. Kelton
Keyword(s):  
Platelet Count ◽  
Retrospective Study ◽  
Epidural Analgesia ◽  
Idiopathic Thrombocytopenic Purpura ◽  
Vaginal Bleeding ◽  
Severe Bleeding ◽  
Thrombocytopenic Purpura ◽  
Platelet Counts ◽  
Itp In Pregnancy ◽  
In Pregnancy

AbstractNumerous studies have examined the outcomes of infants born to mothers with idiopathic thrombocytopenic purpura (ITP). Fewer studies have discussed the morbidity of obstetric patients with ITP. We describe a retrospective study of 92 women with ITP during 119 pregnancies over an 11-year period. Most women had thrombocytopenia during pregnancy. At delivery, women in 98 pregnancies (89%) had platelet counts lower than 150 × 109/L; most had mild to moderate thrombocytopenia. For many, the pregnancy was uneventful; however, women had moderate to severe bleeding in 25 pregnancies (21.5%). Women in 37 pregnancies (31.1%) required treatment to increase platelet counts. During delivery, 44 women (37.3%) received epidural analgesia without complications, with most having a platelet count between 50 and 149 × 109/L. Most deliveries (82.4%) were vaginal. Bleeding was uncommon at delivery. Infant platelet counts at birth ranged from 12 to 436 × 109/L; 25.2% of infants had platelet counts lower than 150 × 109/L, and 9% had platelet counts lower than 50 × 109/L. Eighteen infants (14.6%) required treatment for hemostatic impairment. Two fetal deaths occurred. One was caused by hemorrhage. ITP in pregnancy carries a low risk, but mothers and infants may require therapy to raise their platelet counts. (Blood. 2003;102:4306-4311)

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