Childhood Immune Thrombocytopenia: Long-term Follow-up Data Evaluated by the Criteria of the International Working Group on Immune Thrombocytopenic Purpura

Key Points After recovering from TTP, the prevalence of hypertension, depression, and systemic lupus erythematosus and risk of death are increased. TTP may be a more chronic disorder rather than a disorder of acute episodes and complete recovery.

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

Blood ◽

10.1182/blood.v112.11.4548.4548 ◽

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.

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

Blood ◽

10.1182/blood.v126.23.3259.3259 ◽

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.

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Open ADAMTS13, induced by antibodies, is a biomarker for subclinical immune-mediated thrombotic thrombocytopenic purpura

Blood ◽

10.1182/blood.2019004221 ◽

2020 ◽

Cited By ~ 4

Author(s):

Elien Roose ◽

An-Sofie Schelpe ◽

Edwige Tellier ◽

György Sinkovits ◽

Bérangère S Joly ◽

...

Keyword(s):

Thrombotic Thrombocytopenic Purpura ◽

Adamts13 Activity ◽

Thrombocytopenic Purpura ◽

Follow Up Study ◽

Open Conformation ◽

Immune Mediated ◽

Long Term Follow Up ◽

Donor Plasma

Recently, we showed that during the acute phase of immune-mediated thrombotic thrombocytopenic purpura (iTTP), ADAMTS13 circulates in an open conformation. Although the cause of this conformational change in acute iTTP remains elusive, ADAMTS13 is mainly closed in iTTP patients (i) in remission with an ADAMTS13 activity >50% and undetectable anti-ADAMTS13 autoantibodies, and (ii) after rituximab treatment, suggesting a role for anti-ADAMTS13 autoantibodies. Therefore, IgGs from 18 acute iTTP patients were purified and added to closed ADAMTS13 in healthy donor plasma. This resulted in open ADAMTS13 in 14/18 (78%) samples, proving that indeed anti-ADAMTS13 autoantibodies can induce an open ADAMTS13 conformation. To further elucidate the conformation of ADAMTS13 in iTTP patients, we studied a novel iTTP patient cohort (n=197) that also included plasma samples of iTTP patients in remission where ADAMTS13 activity was <50%. The open ADAMTS13 conformation was not only found during acute iTTP but also in patients in remission with an ADAMTS13 activity <50% and in half of the patients with an ADAMTS13 activity >50%, although free anti-ADAMTS13 autoantibodies were not always detected. Thus open ADAMTS13 is not only a hallmark of acute iTTP, but also a novel biomarker to detect subclinical iTTP in patients in remission. Finally, a long term follow-up study in one iTTP patient showed that the open conformation precedes a severe drop in ADAMTS13 activity. In conclusion, we have shown that anti-ADAMTS13 autoantibodies from iTTP patients induce an open ADAMTS13 conformation. Most importantly, an open ADAMTS13 conformation is a biomarker for subclinical iTTP and could become an important tool in TTP management.

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