Introduktion: Immunologisk trombocytopeni, ITP är en autoimmun sjukdom. Hos ITP patienter bildas autoantikroppar mot trombocyter som leder till trombocyt eliminering och därmed till trombocytopeni. ITP kan delas in i olika grupper beroende på durationen. Duration i mindre än tre månader kallas för nydiagnostiserad ITP, duration mellan 3 och 12 månader kallas för persistent ITP och duration i mer än 12 månader kallas för kronisk ITP. ITP kan behandlas med olika terapier som anti-CD20-antikroppar som rituximab och trombopoietin-receptor-agonister eltrombopag.  Syfte: Examensarbetes syfte är att undersöka samt jämföra effektivitet mellan rituximab och eltrombopag mot kronisk ITP patienter. Metod: Det här arbetet är baserat på en litteratursökning via databasen PubMed med sökord ”eltrombopag and immune thrombocytopenia” och ” rituximab and immune thrombocytopenia” och därefter valdes 4 Randomized Controlled Trial studier.  Resultat: Studie 1 visade bra effektivitet av rituximab mot kronisk ITP och 28% av patienter kunde nå trombocytantal över 50 x109/L i över 6 månader efter behandlingens slut. Studie 2 visade bra effektivitet av rituximab mot kronisk ITP och 30,8% av patienter kunde nå trombocytantal över 50 x109/L i över 6 månader efter behandlings slut. Studie 3 visade mycket bra effektivitet av eltrombopag mot kroniskt ITP och 60% av patienter kunde nå trombocytantal över 50 x109/L under behandlingstiden. Effekten försvann efter 2 veckor av behandlingens slut.  Studie 4 visade mycket bra effektivitet av eltrombopag mot kroniskt ITP och 59% av patienter kunde nå trombocytantal över 50 x109/L under behandlingstiden. Effekten försvann efter 2 veckor av behandlingens slut.  Slutsats: Både rituximab och eltrombopag visade bra effekt hos ITP patienter, men eltrombopag visade bättre effekt mot kronisk ITP än rituximab. Eltrombopag kunde ge bättre effektivitet samt snabbare svar på ökning av trombocytantalet hos de flesta patienter som behandlades i jämförelse med rituximab. Dock försvann effekten av eltrombopag snabbare efter utsatt behandling. / Introduction: Immunological thrombocytopenia, ITP is an autoimmune disease that can develop in our bodies. Autoantibodies develop in ITP patients against platelets, which are small cells without a nucleus and are formed from the megakaryocytes and play an important role in hemostasis and blood clotting. This leads to platelet elimination and thus to thrombocytopenia. Thrombocytopenia is a condition that means low levels of platelets in the individual and which can lead to various severe symptoms such as bleeding and can even cause death. The cause of ITP may be unknown and then it is called primary ITP. Other reasons can be such as acquired immune deficiency syndrome (AIDS) and then the immune system against platelets is triggered, this is called secondary ITP. ITP can also be divided into different groups depending on the duration in each patient. Duration of less than three months is called newly diagnosed ITP, duration between 3 and 12 months is called persistent ITP and duration of more than 12 months is called chronic ITP. ITP can be treated with various therapies. First line therapy is glucocorticoids, and second line therapy is splenectomy, anti-CD20 antibodies rituximab and thrombopoietin receptor agonists such as eltrombopag. Aim: The purpose of the study is to investigate and compare the efficacy of rituximab and eltrombopag patients with chronic ITP. Method: This work is based on a literature search via the database PubMed with the keywords "eltrombopag and immune thrombocytopenia" and "rituximab and immune thrombocytopenia" and then 4 Randomized Controlled Trial studies were selected, 2 studies deal with rituximab and 2 studies deal with eltrombopag. Results: Study 1 showed good efficacy of rituximab compared to placebo against chronic ITP and 28% of patients were able to reach platelet counts above 50 x109/L for more than 6 months after cessation of treatment. Study 2 showed good efficacy of rituximab in chronic ITP and 30.8% of patients were able to reach platelet counts above 50 x109/L for more than 6 months after treatment. Study 3 showed very good efficacy of eltrombopag against chronic ITP and 60% of patients were able to reach platelet counts above 50 x109/L during the treatment period. The effect disappeared after 2 weeks of treatment discontinuation. Study 4 showed very good efficacy of eltrombopag against chronic ITP and 59% of patients were able to reach platelet counts above 50 x109/L during the treatment period. The effect disappeared after 2 weeks of treatment discontinuation. Conclusion: Both rituximab and eltrombopag showed good efficacy against ITP patients, but eltrombopag showed better efficacy against chronic ITP than rituximab. The NNT number showed that more patients need to be treated with rituximab compared to eltrombopag in order for only one of the patients to be able to achieve a platelet count above 50 x109/L, which means a greater chance for those treated with eltrombopag to achieve a platelet count above 50 x109/L. Eltrombopag was able to provide better efficacy and a faster response to increase in platelet count in most patients treated compared to rituximab.

The overall aim of this thesis is to improve knowledge about the prevention of infectious and bleeding complications in patients with hematological malignancies, primarily in those with chronic lymphocytic leukemia (CLL) and myelodysplatic syndrome (MDS). Hypogammaglobulinemia, impaired production of immunoglobulins (Ig), is an established risk factor for infection, but the impact of IgG pure subclass deficiency (IgG subclass deficiency with adequate production of IgG, IgA, and IgM) has been debated. In a retrospective single institution study, we concluded that pure IgG subclass deficiency in CLL patients is rare and is not associated with an increased risk of infection. Hence, routine analysis of IgG subclasses in patients with CLL is not warranted. There is no consensus on recommending vaccination against Streptococcus pneumoniae to CLL patients mainly because comparative studies are lacking. In our randomized trial, the efficacy of a conjugated pneumococcal vaccine on immune response was superior or equal to a polysaccharide vaccine for all pneumococcal serotypes common for the two vaccines. A conjugate pneumococcal vaccine should therefore be included in vaccination programs for patients with CLL. Bronchoalveolar lavage (BAL) is a well-established invasive method to identify the cause of pulmonary infiltrates in immunocompromised patients. In a retrospective trial, we have studied the diagnostic yield of BAL in patients with hematological malignancies. We concluded that BAL is highly useful in either verifying or excluding some of the important respiratory tract infections affecting these patients, particularly invasive pulmonary aspergillosis (IPA) and Pneumocystis jirovecii pneumonia (PJP). However, standardized procedures for BAL sampling should be continually revised to avoid unnecessary microbiological tests. Thrombocytopenia, an adverse prognostic factor in patients with MDS, can be aggravated by azacitidine, first-line treatment for high-risk MDS. Eltrombopag, a thrombopoietin-receptor agonist (TPO-R), alleviates thrombocytopenia in patients with immune thrombocytopenic purpura (ITP). In a phase I clinical trial, we concluded that the combination of eltrombopag and azacitidine in high-risk MDS patients with thrombocytopenia is feasible and well tolerated in doses up to 200 mg eltrombopag daily.