Table 4 Management of RMD patients undergoing HSCT.

RBC/platelet transfusions

As for haematological patients, monitoring of blood counts is mandatory in ADs (e.g. at every visit and as clinically indicated), including long-term follow up. Platelet and erythrocyte transfusions should be administered according to centre policy and protocols.

Blood products should be irradiated before HSC mobilisation and collection and for a period of time according to institutional and national practice and SOPs, which include consideration of clinical history and immunosuppressive drugs.

G-CSF

In case of grade 3-4 neutropenia, the use of G-CSF may be carefully considered according to the risk/benefit evaluation. Prophylactic administration can be considered (beginning, for example, days +4 to +5 after HSCT infusion), to reduce the duration of neutropenia, according to centre policy.

Infection prophylaxis

Antibacterial prophylaxis: In patients with a high-risk profile for neutropenia, prophylaxis may be considered once ANC <0.5 × 10⁹/L. As per institutional standards. Warning in case of colonization by multi-drug resistant (MDR) pathogens.

Anti-viral prophylaxis: All patients. Start from conditioning until 1-year post-HSCT (level II) AND/OR until CD4⁺ count >0.2 × 10⁹/L. Commonly used are valaciclovir 500 mg bid or aciclovir 800 mg bid.

Anti-pneumocystis and anti-toxoplasma: All patients. To start from conditioning until 6 months post-HSCT (level III) AND/OR until CD4⁺ count >0.2 × 10⁹/L. Co-trimoxazole (TMP/SMX) 480 mg once daily or 960 mg three doses each week. In case of co-trimoxazole allergy, pentamidine inhalation (300 mg once every month) are recommended, dapsone 100 mg daily or atovaquone 1500 mg once daily can be considered [89]. All patients positive for anti-toxoplasma antibodies should receive oral co-trimoxazole for 6 months (level II) [90].

Systemic primary anti-fungal prophylaxis: The management of anti-fungal prophylaxis should be discussed within a MDT meeting (disease specialist, infection-disease specialist, and HSCT specialists). Anti-fungal prophylaxis should be considered in severe neutropenia (ANC <0.5 × 10⁹/L) and/or prolonged neutropenia for at least 100 days post-transplant (level II). Anti-fungal prophylaxis should be evaluated depending on the duration of glucocorticoid use.

Vaccine strategy

Patients should follow national recommendations on vaccination. Measurements of specific antibody titres may be helpful to determine the need for vaccination after HSCT. Recently, ADWP has also provided specific COVID-19 vaccine recommendations in patients with ADs [91]. Re-vaccinations can be considered starting from 3 to 6 months after HSCT following centre policy and EBMT recommendations [18, 92]. Live vaccines are generally contraindicated in immunosuppressed patients [93].

WBC, biochemistry panel, AST, ALT, LDH, bilirubin, CRP

Standard follow-up as haematological patients.

At every visit and as clinically indicated.

CMV, EBV PCR monitoring

Monitor at least during the first 100 days after HSCT, in consideration of immunosuppression. MDT evaluation recommended [94,95,96,97]. CMV antibody positive patients receiving ATG or other serotherapy, or receiving manipulated autografts, are recommended to undergo CMV PCR monitoring for the first 100 days after HSCT (level III). CMV reactivation should be treated according to centre policy. EBV antibody positive patients receiving ATG or other serotherapy, or receiving manipulated autografts, are recommended to undergo EBV PCR screening for the first 100 days after HSCT, with active surveillance for post-transplant lymphoproliferative disease according to local practice (level III). Monitoring for CMV and EBV is important and to be considered also in seronegative patients according to local practise (level III).

Quantitative immunoglobulins

Consider immunoglobulin replacement therapy in case of hypogammaglobulinemia (<4 g/l), due to the risk of recurrent infections, after weighing up the benefits, risks and costs of administration (level III).

Late effects testing appropriate to age

Standard follow-up as haematological patients [98]. Yearly or as clinically indicated.

The occurrence of secondary ADs [46, 99], e.g. TSH levels for autoimmune thyroid disease or platelets for ITP [100, 101], and secondary malignancies must be monitored.

Fertility, pregnancy and menopause

Risk of pregnancy without birth control and/or premature menopause and infertility should be considered after HSCT [102, 103].

Follow-up after HSCT and ongoing responsibility for the patient

All patients should remain under the direct routine care of the HSCT specialist for at least 100 days after HSCT, or longer, if necessary, until clinically stable. Thereafter combined care between HSCT specialist and referring organ/AD specialist with joint annual review as a minimum is recommended (level III). A follow-up of potential infectious complications should be considered mandatory.