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APPENDIX

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L-ASA low dose aspirin

MLD metachromatic leukodystrophyA/

MM multiple myelomaNME new molecular entityNSCLC non-small cell lung cancerNSCT non stem cell transplant

Ph+ ALLPhiladelphia chromosome-positive acute lymphoblastic leukemiaA/ A/ T

PTCL peripheral T-cell lymphoma

R/R relapsed/refractoryRCC renal cell cancerSBS short bowel syndromeSC subcutaneous formulationSCT stem cell transplantUC ulcerative colitisvWD von Willebrand disease A /

AML acute myeloid leukemiaARD acid-related diseasesCD Crohn‘s disease

CHAWI congenital hemophilia A with inhibitors

CML chronic myeloid leukemiaCMV CytomegalovirusCNS central nervous systemCRL complete response letterCTCL cutaneous T-cell lymphoma

cTTP congenital thrombotic thrombocytopenic purpura

DEE developmental and epileptic encephalopathies)

FL front line A

GvHD graft versus host diseaseHAE hereditary angioedemaH2H head to headHCC hepatocellular carcinomaHemA hemophilia AHL Hodgkin's lymphoma

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