Romiplostim for Primary Immune Thrombocytopenia (ITP) in Routine Clinical Practice: Results from a Multicentre Observational Study in Germany

2021 ◽  
Author(s):  
Marcel Reiser ◽  
Klaus M. Josten ◽  
Hermann Dietzfelbinger ◽  
Anouchka Seesaghur ◽  
Markus Schill ◽  
...  
Keyword(s):  
Clinical Practice ◽  
Observational Study ◽  
Immune Thrombocytopenia ◽  
Routine Clinical Practice ◽  
Newly Diagnosed ◽  
Platelet Response ◽  
Chronic Itp ◽  
Primary Immune Thrombocytopenia ◽  
Post Hoc

Introduction: The effectiveness and safety of romiplostim were evaluated by immune thrombocytopenia (ITP) phase (newly diagnosed/persistent/chronic) at romiplostim initiation. Methods: Post hoc analysis of a prospective, German, multicentre, observational study in adults with ITP who received ≥1 dose of romiplostim. Follow-up data were collected for ≤2 years. Outcomes included overall platelet response (≥1 platelet count ≥50 × 109/L at 2–24 weeks after romiplostim initiation) or durable platelet response (≥75% of measurements ≥50 x 109/L at 14–24 weeks), and adverse drug reactions (ADRs), evaluated by ITP phase. Results: Data from 96 patients were analysed (newly diagnosed, n=18; persistent, n=25; chronic, n=53). During the 2–24-week follow-up, overall platelet response was achieved in 100% (95% confidence interval [CI]: 81.5–100), 100% (86.3–100), and 96.2% (87.0–99.5) of patients with newly diagnosed, persistent, or chronic ITP, respectively; platelet responses were durable in 88.2% (63.6–98.5), 65.0% (40.8–84.6), and 69.4% (54.6–81.7) of patients. During the 2-year follow-up, ADRs occurred in 24.0–35.8% of patients across phases. Two patients with chronic ITP experienced bone marrow ADRs; no thrombotic ADRs occurred. Conclusion: Romiplostim was effective and well tolerated in patients with newly diagnosed, persistent, or chronic ITP in routine clinical practice.

scholarly journals A large observational study of patients with primary immune thrombocytopenia receiving romiplostim in European clinical practice

2016 ◽  
Vol 98 (2) ◽  
pp. 112-120 ◽  
Author(s):  
Michael Steurer ◽  
Philippe Quittet ◽  
Helen A. Papadaki ◽  
Dominik Selleslag ◽  
Jean-François Viallard ◽  
...  
Keyword(s):  
Clinical Practice ◽  
Observational Study ◽  
Immune Thrombocytopenia ◽  
Primary Immune Thrombocytopenia ◽  
Large Observational Study

Risk of skeletal-related events (SREs) in patients with prostate cancer (PC) and newly diagnosed metastases to bone.

2012 ◽  
Vol 30 (15_suppl) ◽  
pp. e15197-e15197
Author(s):  
Andrew Glass ◽  
Lois Lamerato ◽  
John Edelsberg ◽  
Kathryn E. Richert-Boe ◽  
Charu Taneja ◽  
...  
Keyword(s):  
Clinical Practice ◽  
Cumulative Incidence ◽  
Randomized Clinical Trials ◽  
Pathologic Fracture ◽  
Cord Compression ◽  
Routine Clinical Practice ◽  
Initial Diagnosis ◽  
Newly Diagnosed ◽  
Risk Of Death

e15197 Background: Bone is a common site of metastatic involvement in patients (pts) with PC. Bony metastases (mets) are often associated with SREs (spinal cord compression [SCC], pathologic fracture [PF], surgery to bone [SB], radiotherapy to bone [RT]). Skeletal complications cause significant morbidity and mortality. Current estimates of SRE risk come principally from randomized clinical trials. Information from routine clinical practice is limited. Methods: Using the tumor registry and electronic data stores at a large U.S. Midwest healthcare system that serves approximately 800,000 persons, we retrospectively identified all pts aged ≥18 yrs with primary PC and newly diagnosed bone mets between 1/1/95 and 12/31/09. Electronic medical records were reviewed by trained abstractors for evidence of SREs between date of bone mets diagnosis and death, loss to follow-up, or end of study for evidence of first SRE. Cumulative incidence of SREs was estimated in the presence of competing risk of death. Results: We identified a total of 420 men with primary PC and newly diagnosed bone mets; 42 pts had evidence of SREs at initial diagnosis of bone mets and were excluded from the analyses. Among the remaining 378 pts, mean (SD) age was 72.7 yrs (9.8 yrs); 38% were Caucasian and 58% were African-American. Median duration of follow-up after diagnosis of bone mets was 17.1 months (mos). At 12 mos, cumulative incidence of SREs was 31.6% (SCC, 6.1%; PF, 15.0%; SCC and/or PF, 19.1%; SB, 3.9%; RT, 24.4%) (Table). Corresponding figures at 24 mos were 45.3% (SCC, 12.5%; PF, 22.2%; SCC and/or PF, 30.2%; SB, 6.2%; RT, 34.9%). Relatively few pts (14.6%) received intravenous bisphosphonates prior to SRE. Conclusions: Pts with PC in routine clinical practice are at high risk of SREs following initial diagnosis of bone mets. [Table: see text]

Clinical outcomes and patient (pt) profiles in REASSURE: An observational study of radium-223 (Ra-223) in metastatic castration-resistant prostate cancer (mCRPC).

2020 ◽  
Vol 38 (6_suppl) ◽  
pp. 32-32
Author(s):  
Celestia S. Higano ◽  
Fred Saad ◽  
A. Oliver Sartor ◽  
Kurt Miller ◽  
Peter Conti ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Clinical Practice ◽  
Observational Study ◽  
Clinical Outcomes ◽  
Bone Fractures ◽  
Bone Marrow Suppression ◽  
Routine Clinical Practice ◽  
Metastatic Sites

32 Background: Ra-223 is a targeted alpha therapy that showed a survival advantage and favorable safety profile in the phase 3 ALSYMPCA trial in pts with mCRPC. REASSURE (NCT02141438) is evaluating the long-term safety of Ra-223 in routine clinical practice in pts with mCRPC over a 7-year follow-up period. Methods: In this global, prospective, single-arm, observational study, the second prespecified interim analysis (data cut-off March 2019) evaluated safety and clinical outcomes of Ra-223 in pts with mCRPC. Primary outcome measures were incidence of second primary malignancies (SPM), bone marrow suppression and short- and long-term safety in pts who had ≥1 Ra-223 dose. Secondary outcomes included overall survival (OS). Results: For 1465 pts in the safety analysis, median follow up was 11.5 months. Median PSA (n=1053), ALP (n=1048), and LDH (n=555) levels at baseline were 59 ng/mL, 135 U/L, and 269 U/L, respectively. 81% of pts had bone metastases only at baseline; 19% of pts had other metastatic sites, mostly in the lymph nodes. 19% of pts had <6 metastatic sites, 47% had 6–20 sites, 20% had >20 lesions but not a superscan, and 6% had a superscan. 45%, 38%, 37%, 9%, and 8% of pts received prior abiraterone, docetaxel, enzalutamide, cabazitaxel, or sipuleucel-T as prior therapies, respectively. Median number of Ra-223 doses received was 6; 67% of pts had ≥5 doses. SPM occurred in 1% of pts. The most common treatment-emergent drug-related adverse event (AE) of any grade was diarrhea (11%). 10% of pts had a bone-associated event, 5% had fractures, and 15% had a hematological AE. Median OS was 15.6 months (95% CI 14.6–16.5). Conclusions: In REASSURE, there was a low incidence of SPM, bone fractures, and bone marrow suppression after Ra-223 treatment, with no new AEs identified. This study confirms that in routine clinical practice, Ra-223 AE rates were low, and pts generally received ≥5 doses. Clinical trial information: NCT02141438. [Table: see text]

Risk of skeletal-related events (SREs) in patients with breast cancer (BC) and newly diagnosed metastases to bone.

2012 ◽  
Vol 30 (15_suppl) ◽  
pp. e12024-e12024
Author(s):  
Charu Taneja ◽  
Lois Lamerato ◽  
Andrew Glass ◽  
Kathryn E. Richert-Boe ◽  
John Edelsberg ◽  
...  
Keyword(s):  
Clinical Practice ◽  
Cumulative Incidence ◽  
Randomized Clinical Trials ◽  
Pathologic Fracture ◽  
Cord Compression ◽  
Routine Clinical Practice ◽  
Initial Diagnosis ◽  
Newly Diagnosed ◽  
Risk Of Death

e12024 Background: Bone is a common site of metastatic involvement in patients (pts) with BC. Bony metastases (mets) are often associated with SREs (spinal cord compression [SCC], pathologic fracture [PF], surgery to bone [SB], radiotherapy to bone [RT]). Skeletal complications cause significant morbidity and mortality. Current estimates of SRE risk come principally from randomized clinical trials. Information from routine clinical practice is limited. Methods: Using the tumor registry and electronic data stores at a large US Midwest healthcare system that serves approximately 800,000 persons, we retrospectively identified all pts aged ≥18 yrs with primary BC and newly diagnosed bone mets between 1/1/95 and 12/31/09. Electronic medical records were reviewed by trained abstractors for evidence of SREs between date of bone mets diagnosis and death, loss to follow-up, or end of study. Cumulative incidence of SREs was estimated in the presence of competing risk of death. Results: We identified a total of 378 pts with primary BC and newly diagnosed bone mets; 87 pts had evidence of SREs at initial diagnosis of bone mets and were excluded from the analyses. Among the remaining 291 pts, mean (SD) age was 58.2 yrs (14.3 yrs), and 99% were women; 46% were Caucasian and 48% were African-American. Median duration of follow-up after diagnosis of bone mets was 16.1 months (mos). At 12 mos, cumulative incidence of SREs was 44.5% (SCC, 5.2%; PF, 21.0%; SCC and/or PF, 23.3%; SB, 7.6%; RT, 34.3%) (Table). Corresponding figures at 24 mos were 53.8% (SCC, 7.5%; PF, 29.3%; SCC and/or PF, 32.5%; SB, 9.4%; RT, 41.7%). Approximately one-half (45.0%) of study subjects received intravenous bisphosphonates prior to SRE. Conclusions: Pts with BC in routine clinical practice are at high risk of SREs following initial diagnosis of bone mets. [Table: see text]

Risk of skeletal-related events (SREs) in patients with lung cancer (LC) and newly diagnosed metastases to bone.

2012 ◽  
Vol 30 (15_suppl) ◽  
pp. e18107-e18107
Author(s):  
Kathryn E. Richert-Boe ◽  
John Edelsberg ◽  
Charu Taneja ◽  
Lois Lamerato ◽  
Andrew Glass ◽  
...  
Keyword(s):  
Clinical Practice ◽  
Cumulative Incidence ◽  
Randomized Clinical Trials ◽  
Pathologic Fracture ◽  
Cord Compression ◽  
Routine Clinical Practice ◽  
Initial Diagnosis ◽  
Newly Diagnosed ◽  
Risk Of Death

e18107 Background: Bone is a common site of metastatic involvement in patients (pts) with LC. Bony metastases (mets) are often associated with SREs (spinal cord compression [SCC], pathologic fracture [PF], surgery to bone [SB], radiotherapy to bone [RT]). Skeletal complications cause significant morbidity and mortality. Current estimates of SRE risk come principally from randomized clinical trials. Information from routine clinical practice is limited. Methods: Using the tumor registry and electronic data stores at a large U.S. Midwest healthcare system that serves approximately 800,000 persons, we retrospectively identified all pts aged ≥18 yrs with primary LC and newly diagnosed bone mets between 1/1/95 and 12/31/09. Electronic medical records were reviewed by trained abstractors for evidence of SREs between date of bone mets diagnosis and death, loss to follow-up, or end of study. Cumulative incidence of SREs was estimated in the presence of competing risk of death. Results: We identified a total of 272 pts with primary LC and newly diagnosed bone mets; 66 pts had evidence of SREs at initial diagnosis of bone mets and were excluded from the analyses. Among the remaining 206 pts, mean (SD) age was 65.7 yrs (10.5 yrs) and 66% were male; 47% were Caucasian and 50% were African-American. Median duration of follow-up after diagnosis of bone mets was 3.0 months (mos). At 6 mos, cumulative incidence of SREs was 45.6% (SCC, 6.9%; PF, 20.6%; SCC and/or PF, 25.0%; SB, 4.1%; RT, 34.7%) (Table). Corresponding figures at 12 mos were 50.8% (SCC, 6.9%; PF, 24.1%; SCC and/or PF, 28.3%; SB, 4.1%; RT, 39.8%). Relatively few pts (17.5%) received intravenous bisphosphonates prior to SRE. Conclusions: Pts with LC in routine clinical practice are at high risk of SREs following initial diagnosis of bone mets. [Table: see text]

Risk of skeletal-related events (SREs) in patients with breast cancer (BC) and newly diagnosed metastases to bone.

2012 ◽  
Vol 30 (27_suppl) ◽  
pp. 91-91
Author(s):  
Charu Taneja ◽  
Lois Lamerato ◽  
Andrew Glass ◽  
Kathryn E. Richert-Boe ◽  
John Edelsberg ◽  
...  
Keyword(s):  
Clinical Practice ◽  
Cumulative Incidence ◽  
Randomized Clinical Trials ◽  
Pathologic Fracture ◽  
Cord Compression ◽  
Routine Clinical Practice ◽  
Initial Diagnosis ◽  
Newly Diagnosed ◽  
Risk Of Death

91 Background: Bone is a common site of metastatic involvement in patients (pts) with BC. Bony metastases (mets) are often associated with SREs (spinal cord compression [SCC], pathologic fracture [PF], surgery to bone [SB], radiotherapy to bone [RT]). Skeletal complications cause significant morbidity and mortality. Current estimates of SRE risk come principally from randomized clinical trials. Information from routine clinical practice is limited. Methods: Using the tumor registry and electronic data stores at a large U.S. Midwest healthcare system that serves approximately 800,000 persons, we retrospectively identified all pts aged ≥18 yrs with primary BC and newly diagnosed bone mets between 1/1/95 and 12/31/09. Electronic medical records were reviewed by trained abstractors for evidence of SREs between date of bone mets diagnosis and death, loss to follow-up, or end of study. Cumulative incidence of SREs was estimated in the presence of competing risk of death. Results: We identified a total of 378 pts with primary BC and newly diagnosed bone mets; 87 pts had evidence of SREs at initial diagnosis of bone mets and were excluded from the analyses. Among the remaining 291 pts, mean (SD) age was 58.2 yrs (14.3 yrs), and 99% were women; 48% were African-American and 46% were Caucasian. Median duration of follow-up after diagnosis of bone mets was 16.1 months (mos). At 12 mos, cumulative incidence of SREs was 44.5% (SCC, 5.2%; PF, 21.0%; SCC and/or PF, 23.3%; SB, 7.6%; RT, 34.3%) (Table). Corresponding figures at 24 mos were 53.8% (SCC, 7.5%; PF, 29.3%; SCC and/or PF, 32.5%; SB, 9.4%; RT, 41.7%). Approximately one-half (45.0%) of study subjects received intravenous bisphosphonates prior to SRE. Conclusions: Pts with BC in routine clinical practice are at high risk of SREs following initial diagnosis of bone mets. [Table: see text]

Romiplostim safety and efficacy for immune thrombocytopenia in clinical practice: 2-year results of 72 adults in a romiplostim compassionate-use program

Blood ◽  
2011 ◽  
Vol 118 (16) ◽  
pp. 4338-4345 ◽  
Author(s):  
Mehdi Khellaf ◽  
Marc Michel ◽  
Philippe Quittet ◽  
Jean-François Viallard ◽  
Magda Alexis ◽  
...  
Keyword(s):  
Platelet Count ◽  
Clinical Practice ◽  
Immune Thrombocytopenia ◽  
Intravenous Immunoglobulins ◽  
Interquartile Range ◽  
Concomitant Treatment ◽  
Platelet Response ◽  
Compassionate Use ◽  
Chronic Itp ◽  
Bleeding Score

Abstract Romiplostim, a thrombopoietic agent with demonstrated efficacy against immune thrombocytopenia (ITP) in prospective controlled studies, was recently licensed for adults with chronic ITP. Only France has allowed romiplostim compassionate use since January 2008. ITP patients could receive romiplostim when they failed to respond to successive corticosteroids, intravenous immunoglobulins, rituximab, and splenectomy, or when splenectomy was not indicated. We included the first 80 patients enrolled in this program with at least 2 years of follow-up. Primary platelet response (platelet count ≥ 50 × 109/L and double baseline) was observed in 74% of all patients. Long-term responses (2 years) were observed in 47 (65%) patients, 37 (79%) had sustained platelet responses with a median platelet count of 106 × 109/L (interquartile range, 75-167 × 109/L), and 10 (21%) were still taking romiplostim, despite a median platelet count of 38 × 109/L (interquartile range, 35-44 × 109/L), but with clinical benefit (lower dose and/or fewer concomitant treatment(s) and/or diminished bleeding signs). A high bleeding score and use of concomitant ITP therapy were baseline factors predicting romiplostim failure. The most frequently reported adverse events were: arthralgias (26%), fatigue (13%), and nausea (7%). Our results confirmed that romiplostim use in clinical practice is effective and safe for severe chronic ITP. This trial was registered at www.clinicaltrials.gov as #NCT01013181.

scholarly journals IMPACT of Therapeutic Strategy and Time to Therapy Initiation on Clinical Evolution of Patients with NEWLY Diagnosed Chronic Myelomonocytic Leukemia. a Report from Erasme Study

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 5607-5607
Author(s):  
Brayan Marcel Merchan Ruiz ◽  
Teresa Bernal ◽  
Montserrat Arnan ◽  
Mar Tormo ◽  
Jose Angel Hernandez Rivas ◽  
...  
Keyword(s):  
Clinical Practice ◽  
Disease Progression ◽  
Therapeutic Strategy ◽  
Routine Clinical Practice ◽  
Chronic Myelomonocytic Leukemia ◽  
Cell Transplant ◽  
Newly Diagnosed ◽  
Active Therapy ◽  
Myelomonocytic Leukemia

Abstract Introduction Chronic myelomonocytic leukemia (CMML) is a clonal hematopoietic stem cell disorder characterized by a heterogeneous clinical and morphological expression that shares features of both myelodysplastic syndromes (MDS) and chronic myeloproliferative disorders. In the last years therapy of CMML has undergone a change with the inclusion of the demethylating agents but data regarding their impact on the “real life” setting are still scarce. The aim of our study was to evaluate the use of the different therapies and the time to therapy in an unselected Spanish population within the ERASME study. Materials and methods The ERASME study (CEL-SMD-2012-01) is an observational, post-authorization, prospective, multicenter study that will include a total of 600 patients with MDS and CMML and follow them during a minimum of three years (or until death). The primary objective of this study is to describe the disease progression in routine clinical practice, based on the initial therapeutic strategy, in patients with newly diagnosed MDS and CMML. We present here the results of a pre-specified interim analysis with data of CMML patients enrolled in the ERASME study. Initial patient management strategy is classified in three groups: Observation (OB) & support (SP) (including blood and platelet transfusions and growth factors), active therapy (AT) (including chemotherapy, azacitidine, lenalidomide, etc) and allogenic hematopoietic cell transplant (HCT) (including those patients receiving other therapies before transplant). Results A total of 41 CMML patients (34% women) with a median age of 80 years (range 49-91) have been recruited between January 2013-June 2014. The median follow-up time was 6.7 months (range 0.4-15.1). Morphological subtypes according WHO classification were CMML-1 (blasts count <10%) in 35 patients (85%) and CMML-2 (blasts count 10% to 19%) in 6 (15%). According to FAB criteria, 30 patients (73%) had CMML-MD depending on absolute leukocyte count at diagnosis (WBC ≤13x109/L) and 11 (27%) had CMML-MP (WBC >13x109/L). Karyotype was normal in 32 patients (86%). Five patients displayed cytogenetic abnormalities; 3 out of 5 patients with trisomy 8 (isolated or with one additional abnormality). The CMML-GESMD cytogenetic risk classification was low/intermediate/high risk in 83%/10%/5% of patients, respectively. The CPSS was low/int-1/int-2/high in 46%/32%/15%/5% of patients, respectively. Nine out of 41 patients were transfusion dependent at diagnosis. Median bone marrow blast count was 3% (range 0-33). Hemoglobin, platelet and neutrophil count was: 11.1 g/dL (range 7.8-16.7), 106x103/µL (4.2-415), and 3.98x109/L (range 0.48-57.2), respectively. After diagnosis, 33, 7 and 1 of CMML patients were considered candidate to SP/OB, AT and HCT strategy, respectively. The main reasons for treatment selection were risk-disease (90%), symptomatology (83%), age (73%), and comorbidities (46%).The median time to AT initiation from diagnosis for AT/OB&SP was 0.52/2.5 months (range 0.22-2.29) and (range 1.0-4.7) for each group, respectively. Patients in active therapy received azacitidine (n=2, 29%), other low-dose chemotherapy (n=4, 57%) and other therapy (erythropoietin and azacitidine) (n=1, 14%), respectively. Only one patient was considered candidate for HCT and this patient received azacitidine prior the transplant. At last follow-up, a total of 5 (12%) of patients have died (2, 29% of active therapy and 3, 9% of support group) after a median of 3.6 months (range 3.1-4.1) and 1.7 months (range 0.7-10), for each group respectively. Conclusions CMML patients were treated on an individualized therapy strategy after diagnostic evaluation and prognosis assessment. More data on disease progression in routine clinical practice may be useful in characterizing the newly diagnosed CMML patients. Our prospective study confirms that azacitidine has been considered a therapy for CMML patients, including for HCT candidates. Disclosures Off Label Use: Vidaza, erythropoietin stimulating agents, revlimid. Valcarcel:Celgene: Honoraria, Speakers Bureau. Rafel:Celgene: Employment. Garcia:Celgene, Novartis: Consultancy, Speakers Bureau.

scholarly journals A Comparative Prospective Observational Study of Children and Adults with Immune Thrombocytopenia: 2-Year Follow-up

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 3741-3741 ◽  
Author(s):  
Thomas Kuehne ◽  
Alexandra Schifferli
Keyword(s):  
Platelet Count ◽  
Research Funding ◽  
Immune Thrombocytopenia ◽  
Initial Diagnosis ◽  
Diagnostic Methods ◽  
Newly Diagnosed ◽  
Time Points ◽  
Platelet Counts ◽  
Chronic Itp

Abstract Introduction It is widely accepted that immune thrombocytopenia (ITP) of children differs from that of adults in the clinical course, such as the rate of spontaneous remission, the bleeding risk and the need of treatment. However, this assumption is limited by incongruity of study populations and divergences of collected information, definitions, study objectives and end-points. Surprisingly, data of the Pediatric and Adult Registry on Chronic ITP (PARC-ITP) at initial diagnosis demonstrated far less differences in clinical and laboratory findings between children and adults than expected (Kühne et al. Haematologica 2011). This suggests that newly diagnosed ITP may be driven by similar pathophysiological mechanisms. Differences may occur in the ability of restoring tolerance. We analyzed 6-, 12-, and 24-month follow-up data of children and adults recorded in the PARC-ITP Registry. Design and Methods PARC-ITP is an international multi-center registry designed to collect data prospectively of children and adults with newly diagnosed ITP, and was opened in May 2004. Demographic information, diagnostic methods, clinical data, and efficacy and safety of management are continuously registered at the time of diagnosis, 6 and 12months and then yearly. Patients younger than 3 months (n=167) and those with a platelet count of >100x109/l were excluded from the analysis. Patients with missing follow-up data at certain time-points were not excluded. Remission of ITP was defined as a platelet count of >100x109/l at any time point and regardless of therapy. Platelet counts of chronic ITP were defined as being <100x109/l at 12 or 24 months. The data were analyzed with descriptive statistics. Results A total of 3'780 evaluable patients with the initial diagnosis of primary ITP were recorded in the PARC-ITP database between 2004 and 2015. There were 3360 children (3 months - 16 years) and 420 adults (≥16 years). The pediatric female: male ratio was 1:1.09, and that of adults was 1:0.54. Follow-up information was available for 67% of children at the 6-month, 49% at the 12-month and 31 % at the 24-month evaluation and in adults in 77%, 64%, and 47%, respectively. In children remission was seen at 6, 12 and 24 months in 70%, 70%, and 71%, and in adults in 45%, 49%, and 56%, respectively. Of the patients with a platelet count of <100x109/l at 6 months, 212/590 children (36%) and 42/152 adults (28%) achieved again a remission at 12-months. The platelet counts of children and adults with chronic ITP at 12 months were 46±30x109/l and 51 ±26x109/l. Adults with a diagnosis of chronic ITP at 12 and 24 months reported having no bleeding in 69% and 65% for the last follow-up period, children in 37% at both time-points. Children with thrombocytopenia at 6, 12 and 24-months received platelet-enhancing drugs in 58%, 46% and 47% and adults in 58%, 52% and 40%, respectively. The diagnosis of secondary ITP and other causes of thrombocytopenia was reported for 123 children, i.e. 3.5%, 1.9% and 1.3% at 6, 12 and 24 months, respectively and 21 adults, i.e. 3.7%, 2.3% and 1.7% at 6, 12 and 24 months, respectively. The reported cause was an infectious disease in both children (49%) and adults (52%). Discussion The PARC-ITP Registry is the first cohort of ITP patients including a mixed pediatric and adult population. Limitations include the variety of participating centers (n=74), data registration on a voluntary basis, a high percentage of loss of follow-up and an unbalanced number of children and adults. Preliminary analyses of follow-up data demonstrate similarities between children and adults in much more areas, than previously assumed. Differences in remission rates where confirmed but in a smaller extent than expected. Treatment requirement in patients with active disease was very similar in both age groups. Surprisingly, adults with a diagnosis of chronic disease exhibited a greater number of a non-bleeding phenotype than children. Conclusion Understanding differences or similarities among children and adults with ITP may guide in finding immune modulatory strategies with the goal of achieving early sustained responses. Disclosures Kuehne: Amgen: Research Funding; UCB Biosciences GmbH: Consultancy. Schifferli:Amgen: Research Funding.

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