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2263 Usefulness of Eltrombopag in Secondary ITP Patients in Clinical Practice

Disorders of Platelet Number or Function
Program: Oral and Poster Abstracts
Session: 311. Disorders of Platelet Number or Function: Poster II
Sunday, December 6, 2015, 6:00 PM-8:00 PM
Hall A, Level 2 (Orange County Convention Center)

Tomás José González-López, MD1*, María Teresa Alvarez-Roman, MD2*, Cristina Pascual, MD3*, Blanca Sánchez-González, MD4*, Fernando Fernández-Fuentes, MD5*, Gloria Pérez-Rus, MD6*, José Angel Hernández-Rivas, MD7*, Silvia Bernat, MD8*, Montserrat Cortés, MD9*, José María Bastida, MD10*, Violeta Martínez-Robles, MD11*, Angeles Fernández-Rodríguez, MD12*, Pavel Olivera, MD13*, Estefanía Bolaños, MD14*, Rafael Alonso, MD15*, Laura Entrena, MD16*, Marta Gómez-Nuñez, MD17*, Nuria Bermejo, MD18*, María Paz Martínez-Badas19*, María Jesús Peñarrubia, MD20*, Arancha Alonso, MD21*, Inmaculada Soto, MD12*, María Yera Cobo, MD22*, Alberto Casaus, MD23*, Isabel Caparrós, MD24*, Marí­a Perera, MD25*, Reyes Jiménez Bárcenas, MD26*, Cecilia Heras, MD7*, María Calbacho, MD27*, Maria Tenorio, MD28*, Armando Luaña, MD29*, Elsa Lopez-Ansoar, MD30*, Gerardo Hermida, MD1*, Javier García-Frade, MD31*, José Ramón González-Porras, MD32* and Miguel A. Sanz, MD, PhD33

1Department of Hematology, Hospital Universitario de Burgos, Burgos, Spain
2Department of Hematology, Hospital Universitario La Paz, Madrid, Spain
3Hematology, Hospital General Universitario Gregorio Marañón, Madrid, Spain
4Department of Hematology, Hospital del Mar, Barcelona, Spain
5Department of Hematology, Hospital Universitario Insular de Gran Canaria, Las Palmas de Gran Canaria, Spain
6Department of Hematology, Hospital General Universitario Gregorio Marañón, Madrid, Spain
7Department of Hematology, Hospital Infanta Leonor, Madrid, Spain
8Hospital de La Plana, Department of Hematology, Castellón, Spain
9Department of Hematology, Fundació Hospital Asil de Granollers, Barcelona, Spain
10Hospital Universitario de Salamanca, Salamanca, Spain
11Department of Hematology, Hospital de León, León, Spain
12Department of Hematology, Hospital Universitario Central de Asturias, Oviedo (Asturias), Spain
13Department of Hematology, Hospital Universitario Valle Hebrón, Barcelona, Spain
14Department of Hematology, Hospital Clínico San Carlos, Madrid, Spain
15Department of Hematology, Hospital Universitario Doce de Octubre, Madrid, Spain
16Hematology Department, Hospital Virgen de las Nieves, Granada, Spain
17Department of Hematology, Parc Sanitari Taulí, Sabadell (Barcelona), Spain
18Department of Hematology, Hospital San Pedro de Alcántara, Cáceres, Spain
19Department of Hematology, Hospital de Avila, Avila, Spain
20Department of Hematology, Hospital Clínico de Valladolid, Valladolid, Spain
21Department of Hematology, Hospital Universitario Quirón Madrid, Madrid, Spain
22Department of Hematology, Hospital Universitario Puerta del Mar, Cádiz, Spain
23Department of Hematology, Hospital Punta de Europa, Algeciras (Cádiz), Spain
24Department of Hematology, Hospital Clínico de Málaga, Málaga, Spain
25Department of Hematology, Hospital Universitario de Gran Canaria Doctor Negrín, Las Palmas de Gran Canaria, Spain
26Department of Hematology, Hospital Universitario Virgen del Rocío, Sevilla, Spain
27Hospital Universitario Ramon y Cajal, Madrid, Spain
28Department of Hematology, Hospital Universitario Ramón y Cajal, Madrid, Spain
29Hematology Department, Hospital Arnau de Vilanova, Lérida, Spain
30Hematology Department, Complejo Hospitalario Universitario de Ourense, Orense, Spain
31Department of Hematology, Hospital Universitario Río Hortega, Valladolid, Spain
32Department of Hematology, Hospital Clínico de Salamanca, Salamanca, Spain
33Servicio de Hematologia, Hospital Univ. La Fe, Valencia, Spain

Background: Eltrombopag is an oral thrombopoietin receptor agonist (TPO-RA) drug approved in primary chronic ITP. Lack of clinical trials in secondary ITP avoids a clear demonstration of its potential in terms of safety and efficacy on secondary ITP.

Aims: To evaluate the efficacy and safety of eltrombopag in secondary ITP patients in daily clinical practice in Spain.

Methods: Ninety-eight secondary ITP patients (aged 18 years or more) from 30 Spanish centers, treated with eltrombopag and included in the Spanish Eltrombopag Registry were retrospectively evaluated. Our study was performed in accordance with the standards of the Helsinki declaration and approved by the Hospital Universitario de Burgos Ethics Committee.

Results:

Our case series included 98 patients we allocated to four categories: immune disorders (n=47), infections (n=23), lymphoproliferative disorders (n=20), and neoplasms (n=8).

The median age of the cohort was 62 (IQR, 40-71) years with 38 men and 60 women. At diagnosis, 34 patients had a Charlson Comorbidity Index score of 2 or more. Median time from ITP diagnosis to eltrombopag initiation was 13 (IQR, 2-66) months. Median number of therapies against thrombocytopenia before eltrombopag was 2 (IQR, 1-3), including rituximab (24), splenectomy(18) and romiplostim (13). Median platelet count when treatment started was 15 x 109/L (IQR, 5-43 x 109/L). Meanwhile, 44 patients had bleeding symptoms. Concomitant therapy was administered to 55 ITP (corticoids in 33) (Table I).

Whole cohort eltrombopag response rate was 59% of responses (R; platelet count ≥30 x109/L and at least 2-fold increase the baseline count and absence of bleeding) with 52% of complete responses (CR; platelet count >100 x 109/L). Regarding the disease associated to ITP we observed  high response rates in immune disorders and infection groups (67% of R, 76 % of R, respectively). Nevertheless, in lymphoproliferative disorders and  neoplastic groups efficacy rates were much lower (36 % of R, 37 % of R respectively). The proportion of patients achieving platelet response was quite similar regardless the other studied parameters: age, sex, concomitant treatment, bleeding and platelet count at start of eltrombopag treatment.

30 adverse events were reported with eltrombopag, being 18 of them grade 3-4. 14 deaths were observed but only two were caused by bleeding. The remaining causes of death were: 4  because of bacterial sepsis and another 4 due to progression of basal disease. 2 secondary neoplasms, 1 aspergillosis and one death due to a non-treated severe anemia were also reported (Table II).

Conclusion:  

The use of eltrombopag for treating secondary ITP is effective and safe. To point out, its efficacy in lymphoproliferative disorders and in neoplasm-associated ITP is lower than in benign diseases.

Certainly, more studies are needed to confirm usefulness of TPO-RAs in secondary ITP cases.

Table I.  Patient characteristics

Variable

Total

(n = 98)

Type of disease, n

Immune disorders

   SLE

   Evans Syndrome

   Antiphospholipid Syndrome

   Sjögren Syndrome

   Rheumatoid Arthritis

   Immunodeficiencies

   Autoimmune Hepatitis

   Primary Biliary Cirrhosis

   Psoriatic arthritis

   Evans Syndrome-Immunodeficiencies

   Evans Syndrome-HCV

   Graves-Basedow disease

   Inflammatory Bowel disease

Lymphoproliferative disorders

   Lymphoproliferative diseases

   HCV-Lymphoma

   HIV-Lymphoma

Infections

   Hepatitis C Virus

   HIV

   HCV-HIV

Neoplasms

   Myeloid Neoplasms

Age, years, median [Q1;Q3]

13

8

6

5

3

3

2

2

1

1

1

1

1

16

3

1

16

5

2

8

62[40;71]

Men/Women n

38/60

Bleeding at start of eltrombopag treatment , n

Concomitant treatment, n

   Corticoids

   Immunoglobulins

   Corticoids and Immunoglobulins

44

55

33

6

7

Table II.  Adverse events with Eltrombopag

Variable

n

   Total, n

   Serious Adverse Events (Grade 3-4), n

         Progression of basal disease

         Severe Bacterial Infections

         Deep venous thrombosis

         Stroke

         Medullary fibrosis

         Severe Bleeding

         Aspergillosis

         Pulmonary Embolism

         Secondary neoplasms

         Acute Pancreatitis

         Acute Myocardial Infarction

   Deaths, n

         Bacterial Infections

         Progression of basal disease

         Secondary neoplasms

         Severe Bleeding

         Aspergillosis

         Severe Anemia due to negative of patient to transfusion

30

18

4

3

3

2

2

1

1

1

1

1

1

14

4

4

2

2

1

1

Disclosures: Off Label Use: We describe the possibility of using eltrombopag, an oral thrombopoietin receptor analog, for secondary ITP patients..

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