This handout is for aplastic anaemia / acquired bone-marrow failure (saa / vsaa — chronic + subacute). Your care team identified this based on: pancytopenia (≥2 lineages cytopenic) on cbc — bone-marrow failure differential (bsh 2024 pmid 38247114).
Other reasons your team may use this plan: isolated or bilineage cytopenia progressing to pancytopenia (dezern blood adv 2021 pmid 34156438); petechiae / mucosal bleeding from severe thrombocytopenia (bsh 2024 pmid 38247114); recurrent / severe infection from neutropenia (bsh 2024 pmid 38247114).
Take these medications exactly as prescribed. Do not stop or change a dose without talking to your provider.
| Medication | Starting dose | How | When | What it does |
|---|---|---|---|---|
| horse antithymocyte globulin (lymphocyte immune globulin, ATGAM) | 40 mg/kg/day IV × 4 consecutive days | IV | daily × 4 days | Horse-ATG backbone of first-line IST — superior to rabbit-ATG (Scheinberg NEJM 2011 PMID 21812672: 6-mo ORR 68% vs 37%, 3-y survival 96% vs 76%). Premedicate (steroid/antihistamine); monitor serum sickness d7-14 (BSH 2024 PMID 38247114). |
| ciclosporin (cyclosporine) | 5 mg/kg/day PO divided BID (trough-adjusted ~200-400 ng/mL) | PO | BID | Calcineurin inhibitor — second pillar of IST with horse-ATG; continue ≥12 mo then taper SLOWLY to limit relapse (BSH 2024 PMID 38247114; RACE PMID 34986284). Monitor trough, renal function, BP. |
| eltrombopag | 150 mg PO once daily (75 mg East-Asian ancestry); from day 1 | PO | once daily | TPO-RA added UPFRONT to hATG + CsA — RACE NEJM 2022 PMID 34986284: 3-mo CR 22% vs 10%, 6-mo ORR 68% vs 41%, faster response (median 3.0 vs 8.8 mo); Townsley NEJM 2017 PMID 28423296 day-1 cohort 6-mo CR 58%/ORR 94%. Monitor LFTs + clonal evolution (BSH 2024 PMID 38247114). |
| rabbit antithymocyte globulin (Thymoglobulin) | 3.5 mg/kg/day IV × 5 days (HSCT conditioning) or salvage IST | IV | daily × 5 days | Inferior to horse-ATG as first-line IST (Scheinberg PMID 21812672) — reserved for salvage IST or as serotherapy within HSCT conditioning where rabbit-ATG is preferred (ASTCT 2024 PMID 39307421). |
| romiplostim | 10 mcg/kg SC weekly, titrate | SC | weekly | Alternative TPO-RA in refractory AA where eltrombopag is not tolerated/available; monitor for clonal evolution (BSH 2024 PMID 38247114). |
| prednisolone | 1 mg/kg/day PO (ATG cover; serum-sickness short course) | PO | daily, tapered | Short corticosteroid course with ATG to attenuate cytokine-release and serum sickness (NOT disease-modifying monotherapy) (BSH 2024 PMID 38247114). |
| filgrastim (G-CSF) | 5 mcg/kg/day SC (short course) | SC | daily | Adjunct in severe infection/sepsis only — does NOT improve survival or response and routine use discouraged (clonal-evolution concern); no substitute for definitive therapy (BSH 2024 PMID 38247114). |
| danazol | 200 mg PO BID-TID | PO | BID-TID | Androgen — historical / supportive role; rational in telomere-biology disorders (dyskeratosis congenita) and where IST/HSCT not feasible; monitor LFTs, virilisation (BSH 2024 PMID 38247114; DeZern PMID 34156438). |
| oxymetholone | 1-5 mg/kg/day PO | PO | daily | Anabolic androgen — supportive option where IST/HSCT unavailable; hepatotoxicity / virilisation monitoring (BSH 2024 PMID 38247114). |
| allogeneic haematopoietic stem-cell transplantation (HLA-matched sibling / matched-unrelated / haploidentical) | — | — | — | Curative for vSAA/SAA — first-line in young patients with HLA-matched sibling (5-y OS >90%); bone-marrow graft + rabbit-ATG conditioning preferred; Fanconi/IBMFS REQUIRES alkylator/irradiation DOSE-REDUCTION to avoid fatal toxicity (ASTCT 2024 PMID 39307421; BSH paeds PMID 29285764). |
| irradiated leucodepleted CMV-safe blood products (pRBC / platelets) | — | — | — | Restrictive thresholds; irradiated + leucodepleted + CMV-safe if potential HSCT candidate; AVOID family-donor products pre-HSCT (alloimmunisation/graft-rejection risk) (BSH 2024 PMID 38247114; ASTCT 2024 PMID 39307421). |
| iron-chelation therapy (transfusional iron overload) | — | — | — | Manage transfusional iron overload in transfusion-dependent AA; weigh cytopenia/marrow effects of chelator choice (BSH 2024 PMID 38247114). |
Plan: Aplastic Anaemia Definitive Therapy (IST triplet + TPO-RA + supportive)
Contact your care team if any of the following happen:
Call 911 or go to the nearest emergency room right away if you have:
Long-term clonal-evolution surveillance (MDS/AML/PNH — periodic marrow + cytogenetics + flow); response reassessment at 3/6/12 mo (re-treat or escalate non-responders); taper ciclosporin slowly to limit relapse; vaccination/fertility counselling; transition paediatric → adult care; lifelong haematology follow-up given late clonal/relapse risk (BSH 2024 PMID 38247114; ASTCT 2024 PMID 39307421)
Guideline: BSH 2024 adult aplastic anaemia guideline (supersedes BSH 2016 Killick) + BSH paediatric amendment + ASTCT 2024 allo-HCT guideline + RACE NEJM 2022 + Camitta criteria