4.2.4 Haematopoietic stem cell transplantation

Allogeneic stem cell transplantation is the only potentially curative treatment of MDS (Bewersdorf et al. 2020; Skeres & Cutler 2014). In this procedure, the patient receives blood-forming cells (stem cells) from a healthy donor (related or unrelated) to replace their own stem cells following chemotherapy and, in some cases, radiation.

Patients may have additional supportive care requirements to address the immunosuppressive effects and long-term side effects of stem cell transplantation. Issues may include infertility, GVHD, increased risk of infection, iron overload or anaemia, bleeding, mouth ulcers and fatigue.

Timeframe for starting treatment

A referral to a bone marrow transplant specialist should occur once transplantation is considered a potential treatment option for a patient with higher risk MDS and occasionally lower risk MDS.

Early referral to a transplant specialist is recommended for patients under 70 years of age and with higher risk MDS. Age-specific frailty scores and performance status should be considered for such a referral rather than an absolute age cut-off.

Training and experience required of the physician

Documented evidence of the physician’s training and experience (e.g. Fellow of the Royal Australian College of Physicians or equivalent) with adequate training and experience that enables institutional credentialing and agreed scope of practice in bone marrow transplantation.

Documented evidence of the physician’s training and experience, including their specific (sub- specialty) experience with MDS and procedures to be undertaken, should be available.

Health service characteristics

To provide safe and quality care for patients having haematopoietic stem cell transplant, health services should have these features:

  • critical care support
  • 24-hour medical staff availability including access to consultative medical subspeciality services
  • 24-hour operating room access, high dependency unit and intensive care unit
  • bone marrow transplantation unit with ancillary medical and allied health support
  • diagnostic imaging
  • pathology diagnostics
  • ready access to blood banking and transfusion