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O transplante de medula óssea é uma modalidade de tratamento indicada para doenças relacionadas com a fabricação de células do sangue e com deficiências no sistema imunológico. Os principais beneficiados com o transplante são pacientes com leucemias originárias das células da medula óssea, linfomas, doenças originadas do sistema imune em geral, dos gânglios e do baço, e anemias graves (adquiridas ou congênitas).
Outras doenças, não tão frequentes, também podem ser tratadas com transplante de medula, como as mielodisplasias, doenças do metabolismo, autoimunes e vários tipos de tumores. O transplante de medula óssea pode ser indicado para o tratamento de um conjunto de cerca de 80 doenças.