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Conclusions: SCT is increasingly applied to MF patients.

Though a small with pt and disease characteristics, cytogenetics (CG) and molecular ge-
cohort our data indicate that reduced intensity Treo/Flud conditioning is netics (MG).
feasible. Infections following SCT are common causes of death. Methods: Expression of CD30 was analyzed in BM samples by MFC us-
ing antibodies against CD30, CD45, CD117, CD2 and CD25. MC were
Disclosure: No conflict of interest disclosed.
identified as CD45 positive with bright CD117 expression. Coexpression
of CD2 and/or CD25 defined neoplastic MC, median fluorescence inten-
P519 sities (medFI) of CD30 were determined and related to CD30 medFI in
Specific treatment significantly influences outcome in 32 lymphocytes to derive CD30 index. MC infiltration and CD30exp by IH
pregnancies in polycythemia vera (PV) patients was assessed in 22 pt. CG and MG for KITD816V mutation were done in
44 and 80 pt, respectively. CD30exp detected by MFC was correlated to
Wille K.1, Sadjadian P.1, Horstmann A.1, Kolatzki V.1, Griesshammer M.1 IH-detected CD30exp and to CG and MG.
Johannes Wesling Klinikum Minden, Universittsklinik fr Hmatologie und Results: Pt were 51 female, 42 male, median age 59 years (2087), karyo-
Onkologie, RUB Bochum, Minden, Germany types: 41 normal, 3 aberrant, KITD816V mutation was positive in 74/80
Very limited data are available regarding pregnancy (preg) outcomes in pt. 14/93 pt had concurrent hematological non-mast cell disease (AHN-
patients with PV, less than 40 pregs being reported in the whole literature. MD). Mean (SD) MC infiltration was 20%26% (range, 1.5%-85%) by
Within a European Leukemia Net project we collected 32 pregs in 14 PV IH and 0.4%1.8% (range, 0.01%-17%) by MFC. Mean (SD) CD30 index
patients in our center since 2006. Pregs were categorized in two groups. was 19 20 (range, 3154), mean (SD) CD30exp by IH was 9%17%
Group 1 consisted of pregs receiving treatment according to a suggested (range, 0%-70%). MC infiltration by IH and MFC correlated significantly
algorithm (M. Griesshammer et al., Blood Rev. 2008) (n = 18). Group 2 (p = 0.002, r = 0.819), also KITD816V mutation ratio and MC infiltration
(n = 14) included all pregs without a specific treatment. Most patients in by IH and MFC (p = 0.006, r = 0.669 and p < 0.001, r = 0.554, respective-
group 1 received low dose aspirin during preg and low molecular weight ly). No correlation of MFC CD30 index with age, sex, AHNMD, KIT-
heparin after delivery until the 6th week postpartum. The target hematocrit D816V mutation ratio, grade of MC infiltration or %CD30+ MC by IH
during preg was 40% and therapeutic phlebotomy was performed, if need- was found. Pt with normal vs aberrant karyotype had higher CD30 index
ed. Iron supplementation was not advised during preg. (12.2 6.2 vs 6.3 2.7, p = 0.037). A trend to higher CD30 index in pt
Median age at diagnosis of PV was 28.5 yrs (range 1934), median age with KITD816V mutation was seen (23.9 89.1 vs 10.0 5.8, n.s.).
at delivery 33 yrs (2439). Four out of 14 patients (28.6%) had high-risk Conclusions: Neoplastic MC harbouring aberrant karyotypes displayed
PV due to severe thromboembolic complications either at the time of di- stronger CD30 expression, being by trend also stronger on MC from pt
agnosis or during follow up. These included 2 Budd Chiari syndromes, 1 with KITD816V mutations. CD30 expression on MC should be analyzed
portal vein thrombosis and 1 pulmonary embolism. Overall live birth rate by both IH and MFC in a high number of pt to substantiate our findings
was 47% (15/32). In group 1 live birth was recorded in 13/18 pregs (72%), and to better enable correlation with clinical outcome.
while 5 pregs were unsuccessful due to spontaneous abortion. In contrast, Disclosure: Frauke Bellos: Employment or Leadership Position: angestellt im
outcome was significantly worse in group 2 with 2 live births out of 14 Mnchener Leukmielabor
pregs (14%) [chi square, p = 0,001]. In group 2 we observed 8 (67%) spon- Wolfgang Kern: Employment or Leadership Position: Teilhaber Mnchner Leu-
taneous abortions, and 4 (33%) stillbirths. Interferon alpha was used in 4 kmielabor
cases with 3 live births (75%) and one spontaneous abortion. Concerning
maternal complications, no thromboembolic events were observed. How- P521
ever, 5 bleedings occurred (4 minor and 1 major), all of them in group 1
Two different coagulation factor VIII gene mutations in
which was probably due to the treatment with aspirin and/or low molec-
brothers with hemophilia A: an unexpected and rare finding
ular weight heparin. Most PV patients hematocrit values spontaneously
decreased during preg, thus phlebotomies were mainly performed Krammer-Steiner B.1, Nimtz-Talaska A.2, Toenges R.3, Friday D.3, Steiner M.4
during the first trimester. 1
Klinikum Sdstadt Rostock, Klinik fr Innere Medizin III, Rostock, Germany,
This is the largest series of PV pregs reported in a single center experience 2
Praxis fr Kinder- und Jugendmedizin, Frankfurt/O., Germany, 3Diagenom
so far. The success rate of pregs was significantly better (72% versus 14%, GmbH, Rostock, Germany, 4Medizinisches Labor, Rostock, Germany
respectively) for patients with appropriate management performed ac-
Introduction and Objectives: Inherited hemophilia A in females is rare
cording to current guidelines. No thrombotic events were seen, however,
and female hemophilia A patients are at risk of being misdiagnosed as
an increased bleeding rate was observed.
acquired hemophilia A or as von Willebrand disease type 2N. Here we
Disclosure: No conflict of interest disclosed. report a female patient with moderate factor VIII deficiency due to com-
pound heterozygosity for two different coagulation factor VIII gene mu-
Material and methods: A 41-year-old mother presented her three sons
Assessment of CD30 expression on mast cells in
aged 5, 8, and 12 years with moderately decreased factor VIII activity (20
systemic mastocytosis by immunohistochemistry versus to 24%) for genetic counseling and testing. Genomic DNA was obtained
multiparameter flow cytometry and correlation to clinical from anticoagulated blood. All factor VIII gene exons and flanking re-
parameters gions were amplified and PCR products were purified and sequenced.
Bellos F.1, Sotlar K.2, Jeromin S.1, Haferlach C.1, Haferlach T.1, Kern W.1 Results: Initial mutational screening identified the previously identified
MLL Mnchner Leukmielabor GmbH, Mnchen, Germany, 2Pathologisches
hemizygous factor VIII gene mutation p.Met2183Val (p.Met2164Val) lo-
Institut, Ludwig-Maximilians-Universitt Mnchen, Mnchen, Germany cated in exon 23 in the 12-year-old first son. Unexpectedly, the mutation
could not be demonstrated in his two brothers prompting further full se-
Introduction: Systemic mastocytosis (SM) is a rare disease with variable quencing studies. A hemizygous mutation p.Arg2016Gln (p.Arg1997Gln)
clinics, from indolent to aggressive clinical courses. Immunhistochemi- in exon 19 was identified in the 8-year-old and in the 5-year-old boys.
cal staining (IH) has detected CD30 expression (CD30exp) on neoplastic Investigation of both mutations was conducted in the mother and con-
mast cells (MC) as potential indicator for aggressive disease. CD30exp de- firmed the rare diagnosis of a compound heterozygous female hemophilia
tected by multiparameter flow cytometry (MFC) might improve diagnos- A patient. Her residual factor VIII activity was similar to that of her sons
tics and help correlate CD30exp with clinical parameters giving insights (23%) leading to the diagnosis of mild hemophilia A.
into disease biology. We compared CD30exp detected by MFC vs IH on Conclusions: Female cases of hemophilia A are rare. Among the potential
bone marrow (BM) MC of patients (pt) with SM and correlated results genetic mechanisms leading to hemophilia A in females, compound hete-
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Abstracts Oncol Res Treat 2016;39(suppl 3):1160 152

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rozygosity affecting both factor VIII alleles has been demonstrated only in P523
a few cases. Here we report a female patient mother to three hemophilia Platelet transfusion in oncology/hematology patients in
A sons displaying two different missense mutations in exon 19 and exon routine clinical practice in of a tertiary hospital
23, respectively. Compound heterozygosity was confirmed in the mother
causing mild hemophilia A. Berger K.1, Wittmann G.2, Henschler R.3, Rieger C.1, Ostermann H.1
Klinikum der Universitt Mnchen Campus Grosshadern, Medizinische Klinik
Disclosure: No conflict of interest disclosed. und Poliklinik III, Mnchen, Germany, 2Klinikum der Universitt Mnchen,
Abteilung fr Transfusionsmedizin, Zelltherapeutika und Hmostaseologie,
Mnchen, Germany, 3Schweizerisches Rotes Kreuz SRK, Blutspende Zrich,
P522 Zrich, Switzerland
Haemophilia care: Which outcomes should be determined in
clinical routine care and are adequate for access requirements Introduction: There is little information on real life patient data prior and
post transfusion of blood components of clinical hematology and oncol-
Berger K.1, Eichler H.2, Escuriola-Ettinghausen C.3, Holstein K.4, ogy. The objective of this study is to describe patient demographics, indi-
Klamroth R.5, Knigs C.6, Kurnik K.7, Oldenburg J.8, Scholz U.9, cation of platelet transfusion, to compare actively collected hemovigilance
Schramm W.10, Tiede A.11 data and to determine adherence to transfusion guidelines and relative
Klinikum der Universitt Mnchen Campus Grosshadern, Medizinische Klinik effectiveness for patients transfused with platelet components in routine
und Poliklinik III, Mnchen, Germany, 2Universitt und Universittsklinikum des mostly prophylactic use.
Saarlands, Institut fr Klinische Hmostaseologie und Transfusionsmedizin, Methods: Prospective open label, single-center, observational study. In-
Homburg / Saar, Germany, 3HZRM Hmophilie Zentrum Rhein Main GmbH,
clusion of platelet transfusions was consecutive. During the observation
Mrfelden-Walldorf, Germany, 4Universittsklinikum Hamburg-Eppendorf,
Gerinnungsambulanz und Hmophiliezentrum, Hamburg, Germany, 5Vivantes
period the platelet concentrates (plt) transfused were either pathogen in-
Klinikum im Friedrichshain, Zentrum fr Hmophilie und Hmostaseologie, activated (PI) or produced conventionally (CON).
Berlin, Germany, 6Universittsklinikum Frankfurt, Goethe-Universitt Frankfurt, Results: Between March and May 2015 126 patients (pts) received 1207
Klinik fr Kinder- und Jugendmedizin, Schwerpunkt Hmatologie, Onkologie platelet units (586 PI plts, 621 CON plts). 105 patients (pts) signed con-
und Hmostaseologie, Frankfurt am Main, Germany, 7Klinikum der Universitt sent, 11 pts were not able to give informed consent. Subsequently data
Mnchen, Kinderklinik und Kinderpoliklinik im Dr. von Haunerschen for 464 PI plt and 482 CON plt could be documented. Mean age was 53.8
Kinderspital, Mnchen, Germany, 8Universittsklinikum Bonn, Institut fr years. 39% pts were younger than 50 years. 63% of pts were male, 80% of
experimentelle Hmatologie und Transfusionsmedizin, Bonn, Germany,
9 pts were diagnosed with haematological diseases. In total 5 adverse events
Zentrum fr Blutgerinnungsstrungen, MVZ Labor Dr. Reising-Ackermann und
Kollegen, Leipzig, Germany, 10Ludwig-Maximilians Universitt Mnchen, Rudolf- were documented: 1 AE following PI, mild severity not requiring further
Marx-Stiftung, Mnchen, Germany, 11Medizinische Hochschule Hannover, Klinik medical invervention,4 AEs following CON platelet transfusion of moder-
fr Hmatologie, Hmostaseologie, Onkologie und Stammzelltransplantation, ate severity which required further medical intervention. 10% of pts with
Germany underlying haematological diseases received more than 30 transfusions
(36% of all transfused plt). In this group approx. 69% plts were transfused
Background: Stakeholders like the Institute of Quality and Efficiency in
at a transfusion trigger > 10 G/I. 6% of pts received 20 -30 transfusions
Health Care (IQWiG), the Gemeinsame Bundesausschuss (G-BA) and
(14% of all transfused plt) 18% of pts received 1020 transfusions (24% of
payers are increasingly demanding quality oriented patient care. Clinical
all transfused plt), 66% of pts received less than 10 plt transfusions (26%
and patient reported outcomes are in the centre of this context. Therefore
of all transfused plt).
the working group Outcomes of the GTH-Panel Haemophilia reached
Conclusions: The majority of all platelet concentrates in this study was
out to determine relevant outcome parameters in clinical praxis and re-
transfused in a small number of patients. Only a limited number of trans-
search to meet and evaluate the best possible patient care and meet the
fusion reactions were documented. We found a trend to a reduced inci-
payers requirements. This also included assessment clinical routine care
dence of adverse events for the PI treated platelet concentrates. Platelets
and how they could be made assessable for future research initiatives.
were mostly transfused according to current treatment guidelines.
Methods: Literature and desk researches to identify evidence require-
ments from different stakeholders perspectives; Development of a pilot Disclosure: Karin Berger: Expert Testimony: Research Grant CERUs
questionnaire to prioritize clinical and patient reported outcomes (unim- Helmut Ostermann: Expert Testimony: Research Grant CERUS
portant to very important); Questioning of physicians treating haemo-
philia patients (children and adults).
Results: 14 physicians gave feedback on the questionnaire. Focus of the Posterdiskussion
first evaluation was on clinical outcomes. Highly prioritized clinical out- Aggressive Non-Hodgkin-Lymphome
comes were target joints (4 bleeds in one joint during 6 months), inhib-
itor development (influenced by the duration of treatment and intensity,
kind of clotting factor concentrates, mutations), mortality and bleeds. P524
All interviewees scored the aforementioned first three clinical outcomes High efficiency of the pan-PI3K/mTOR inhibitor, PQR-309, in
as very important, 12 physicians graded bleeds (frequency, localization, MCL
severity) as very important, 1 as important. Arthropathy determined by
Kvint R.1, Zimmermann Y.1, Hutter G.1, Hiddemann W.1, Dreyling M.1
clinical joint status, Haemophilia Joint Health Score (HJHS), WFH Phys- 1
Medizinische Klinik III, Klinikum der Universitt Mnchen, Mnchen, Germany
ical Examination Score (Gilbert Score) has been graded as important by 6
and as very important by 8 interviewees. Determination of arthropathy by Introduction: Mantle cell lymphoma (MCL) comprises about 6% of all
radiological joint scores eg Petterson score, magnetic resonance imaging non-Hodgkins lymphoma with a median survival of 35 years. New
and ultrasound were scored of moderate importance, therapy associated emerging strategies especially include inhibitors of the PI3K/mTOR sig-
infections as important (n = 2), very important (n = 10), missing (n = 1). nalling pathway (PI3K, Akt, mTOR) which is constitutively activated in
Osteoporosis has been indicated as an unimportant clinical outcome by 9 MCL and plays a critical role in tumor growth and survival. In this study
interviewees, 1 moderate important, 2 important. we evaluated the efficiency and mode of action of the dual PI3K/mTOR
Conclusions: In a next step the working group outcomes will prioritize (c1+c2)-Inhibitor, PQR-309, in MCL cell lines, as a single agent but also in
the patient reported outcomes to be collected. The groups next objective is combination with others molecular Inhibitors.
to develop a concept how to improve longitudinal outcome data collection Methods: MCL cell lines (Granta 519, Jeko-1, Rec-1 and Mino) were
in Germany. exposed to the panPI3K/ double mTOR inhibitor (PQR-309) as well as
Disclosure: No conflict of interest disclosed. to combination of PQR-309 with PIM- (AZD-199), BET- (JQ1), MEK-
(PD-0325901); BCL2- (ABT199); BTK-(Ibrutinib); RSK-(BI-D1870);
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153 Oncol Res Treat 2016;39(suppl 3):1160 Abstracts

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