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.

scholarly journals Demographics and Long-Term Outcome of Incident Immune Thrombocytopenic Purpura: A Twelve-Years Nationwide Population-Based Study in Taiwan

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 3259-3259
Author(s):  
Bor-Sheng Ko ◽  
Grace Hui-Min Wu ◽  
Yu-Chiao Wang ◽  
Ming Yao ◽  
Churn-Shiouh Gau ◽  
...  
Keyword(s):  
General Population ◽  
Immune Thrombocytopenic Purpura ◽  
Population Based ◽  
Male Gender ◽  
Term Outcome ◽  
Thrombocytopenic Purpura ◽  
Long Term Outcome ◽  
Chronic Itp

Abstract Background and Objectives Immune thrombocytopenic purpura (ITP) is a rare disease, and the epidemiology and long-term outcome are still rarely characterized. This study is then aimed to provide a population-based assessment for the demographics and outcome about ITP in Taiwan, an island in Southeastern Asia with around 23 million inhabitants. Material and Methods This study used claims data from Taiwan's National Health Insurance Research Database (NHIRD). The database included information from a nationwide, mandatory-enrollment and single-payer healthcare system with more than 99% coverage rate in Taiwan since March, 1995. To address adequate medical history tracking and outcome follow-up, only those patients with the first ITP diagnosis from Jan 1st, 2001 to Dec 31st, 2012 were included. Incident ITP was identified first with ICD-9 codes; but those cases with codes for potential ITP-confounding diseases within 6 months from the first ITP code were excluded. Next, only those patients with meaningful pharmacological treatment or splenectomy within 3 months were included in the final analysis. Chronic ITP was defined for those with ICD-9 ITP codes and continuous drug exposure for more than 3 months, or with rituximab or splenectomy. Sex- and age-matched cohorts with 1:10 ratio were selected from Taiwan general population for survival comparison. Results Of the 30673 patients with ITP codes from Jan 1st, 2001 to Dec 31st, 2012, 11437 were identified as incident ITP. The mean age was 42.9+/-27.5 y/o, and 5445 (47.6%) cases had Charlson Comorbidity Index (CCI) score more than 2. The average incidence was 4.16 per 100,000 person-year, and the details are shown in Table 1. The incidence for female was higher than that for male (4.97 vs. 3.38 per 100,000 person-year), and the incidences across the age represented a U-shape distribution, with the highest ones in those aged 0-9 y/o and more than 70 y/o (7.21 and 13.3 per 100,000 person-year, respectively). Some geographic distribution of the incidences existed, with the highest in central part and the lowest in Eastern part of Taiwan (5.33 and 2.64 per 100,000 person-year, respectively). Secondary causes could be identified in 3560 (31.0%) cases, and malignant neoplasma (1743, 49.0%) were most frequently noted. Viral hepatitis B or C were found in 785 (22.1%) cases. Chronic ITP was diagnosed during follow-up in 29.1% (n=3324) of incident ITP patients. Those incident ITP patients aged 0-9 y/o (431/2169 vs. 2893/9268, p<0.001) or male gender (1118/4697 vs. 2206/6740, p<0.001) had a less chance to develop chronic ITP. As compared with the matched cohort from general population, the 10-yr survival rate was significantly inferior for all ITP patients, no matter in those aged below 20 y/o (96.9+/-0.5% vs. 98.8+/-0.1%, p<0.0001) or above 20 y/o (62.5+/-0.8% vs. 83.2+/-0.2%, p<0.0001), as in Figure 1. For chronic ITP, the disadvantaged 10-yr survival rates persisted (for age below 20 y/o: 96.5+/-1.0% vs. 98.6+/-0.2%, p<0.0001; for age above 20 y/o: 72.7+/-1.3% vs. 86.7+/-0.4%, p<0.0001, as in Figure 2). Elder age, male gender and high CCI scores predicted worse survival in multi-variate analysis. Conclusions This study is the largest population-based epidemiology report at nationwide scale till now. Not only the results can provide a valuable demographic description for ITP in Eastern Asia, but also they confirm an inferior long-term outcome for ITP patients, which necessitates more attention to their health care. SD: standard deviation Table 1. Table 1. Figure 2. Figure 2. Figure 3. Figure 3. Disclosures Tang: Novartis: Consultancy, Honoraria.

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.

Morbidity and mortality risk ratios are elevated in severe supine dominant OSA: a long-term follow-up study

2014 ◽  
Vol 19 (2) ◽  
pp. 653-660 ◽  
Author(s):  
Antti Kulkas ◽  
Anu Muraja-Murro ◽  
Pekka Tiihonen ◽  
Esa Mervaala ◽  
Juha Töyräs
Keyword(s):  
Mortality Risk ◽  
Morbidity And Mortality ◽  
Follow Up Study ◽  
Long Term Follow Up ◽  
Risk Ratios

Long-term salvage therapy with cyclosporin A in refractory idiopathic thrombocytopenic purpura

Blood ◽  
2002 ◽  
Vol 99 (4) ◽  
pp. 1482-1485 ◽  
Author(s):  
Giovanni Emilia ◽  
Monica Morselli ◽  
Mario Luppi ◽  
Giuseppe Longo ◽  
Roberto Marasca ◽  
...  
Keyword(s):  
Cyclosporin A ◽  
Idiopathic Thrombocytopenic Purpura ◽  
Complete Response ◽  
Term Treatment ◽  
Thrombocytopenic Purpura ◽  
Long Term Treatment ◽  
Life Threatening ◽  
Difficult Challenge

Treatment of severe, chronic idiopathic thrombocytopenic purpura (ITP) refractory to most usual therapies is a difficult challenge. Little information exists on the clinical use of cyclosporin A (CyA) in the treatment of ITP. This report describes long-term treatment with CyA (median, 40 months) and follow-up (median, 36.8 months) in 12 adult patients with resistant ITP. CyA used in relatively low doses (2.5-3 mg/kg of body weight per day) led to a clinical improvement in 10 patients (83.3%). Five had a complete response (41.1%), 4 a complete response to maintenance therapy (33.3%), and one a partial response (8.3%). Two patients had no response. Most patients with a response (60%) had a long-term remission (mean, 28.6 months) after discontinuation of CyA. One patient had a relapse of ITP 4 years after CyA therapy was stopped. Side effects were moderate and transient, even in patients dependent on continued CyA treatment. CyA seems to represent reasonable salvage treatment in severe, potentially life-threatening, refractory ITP.

Oral Eltrombopag Treatment Reduces the Need for Concomitant Medications in Patients with Chronic Idiopathic Thrombocytopenic Purpura.

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 3424-3424 ◽  
Author(s):  
Patrick F. Fogarty ◽  
James B Bussel ◽  
Gregory Cheng ◽  
Mansoor N Saleh ◽  
Balkis Meddeb ◽  
...  
Keyword(s):  
Idiopathic Thrombocytopenic Purpura ◽  
Phase Iii ◽  
Term Therapy ◽  
Chronic Idiopathic Thrombocytopenic Purpura ◽  
Thrombocytopenic Purpura ◽  
Once Daily ◽  
Platelet Counts ◽  
Chronic Itp ◽  
Rescue Treatment

Abstract INTRODUCTION: Eltrombopag (PROMACTA®/REVOLADE®; GlaxoSmithKline, Collegeville, PA) is the first oral, small molecule, non-peptide thrombopoietin receptor agonist developed as a treatment for thrombocytopenia of various etiologies, including chronic idiopathic thrombocytopenic purpura (ITP). In 2 placebo-controlled studies totaling over 200 patients with chronic ITP, eltrombopag has demonstrated a significant increase in platelet counts and a reduction in clinically relevant bleeding 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. Although the primary objective of this study was to raise platelet counts to a safe level (≥50,000/μL), the ability of eltrombopag treatment to allow patients to reduce concomitant ITP medications and avoid the adverse events associated with those therapies was also of interest. METHODS: Adult 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 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. The effect of eltrombopag treatment on the ability of patients to reduce and/or discontinue baseline concomitant ITP medications was evaluated. RESULTS: As of the clinical cut-off date (January 7, 2008), 207 patients (median age, 50 years; 67% female) had received eltrombopag. At baseline, 69 (33%) patients reported the use of ITP medications; of these, 65 patients had at least 1 post-baseline visit by the clinical cut-off date and were evaluable for response status. Eighty percent (52/65) of these patients responded to eltrombopag with a platelet count of ≥50,000/μL during the study. Forty-eight percent (33/69) of patients attempted to reduce or discontinue their concomitant ITP medications during the study. Seventy percent (23/33) of these patients discontinued or had a sustained reduction of their baseline ITP medication and did not require any subsequent rescue treatment as of the clinical cut-off date; of these, 65% (15/23) had maintained the discontinuation or reduction for at least 24 weeks as of the clinical cut-off date. Sixty-one percent (20/33) of patients discontinued at least 1 baseline ITP medication, and 55% (18/33) discontinued all baseline ITP medications, without subsequent rescue treatment. Discontinued or reduced medications included prednisone (n = 11); prednisolone (n = 8); danazol (n = 5); and azathioprine, dexamethasone, mycophenolic acid, and oxymetholone (n = 1 each). Only 12% (8/69) of patients increased the dose of concomitant ITP medication from baseline. CONCLUSION: In this study, long-term therapy with oral eltrombopag allowed 80% of patients with chronic ITP who were also receiving a concomitant ITP medication at baseline to maintain elevated platelet counts sufficient to permit a reduction in the use of concomitant ITP medications without the need for rescue therapy.

Long-Term Follow-Up of Adult Patients with Idiopathic Thrombocytopenic Purpura after Splenectomy

2011 ◽  
Vol 4 (3) ◽  
pp. 285-289 ◽  
Author(s):  
Marica Pavkovic ◽  
Slobodanka Trpkovska-Terzieva ◽  
Arif Latifi ◽  
Oliver Karanfilski ◽  
Lidija Cevreska ◽  
...  
Keyword(s):  
Idiopathic Thrombocytopenic Purpura ◽  
Adult Patients ◽  
Thrombocytopenic Purpura ◽  
Long Term Follow Up

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%)

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