Page 307 - Essential Haematology

Chapter 22 Aplastic anaemia and bone marrow failure / 293

and clonal cytogenetic changes suggest myelodys- horse or rabbit) and is of benefit in approxi-
plasia rather than aplastic anaemia. Some patients mately 50 – 60% of acquired cases. It is usually
diagnosed as having aplastic anaemia develop PNH, given with corticosteroids which reduce the side -

myelodysplasia or acute myeloid leukaemia in effects of ALG including the serum sickness of

subsequent years. This may occur even in patients fever, rash and joint pains which may occur
who have responded well to immunosuppressive approximately 7 days after administration.
therapy. Corticosteroids should not be used alone as they
increase the risk of infection. Typically, if there is
no response to ALG after 4 months a second
Treatment
course may be tried, prepared from another
General species. Overall, up to 80% of patients respond
The cause, if known, is removed (e.g. radiation or to combined ALG.

drug therapy is discontinued). Initial management 2 Ciclosporin Th is is an eff ective agent which
consists largely of supportive care with blood trans- appears particularly valuable in combination
fusions, platelet concentrates and treatment and with ALG and steroids.
prevention of infection. All blood products should 3 Alemtuzumab (Anti - CD52) Th is has proved
be leucodepleted to reduce the risk of alloimmuni- effective in about 50% of pateint in small studies

sation and irradiated to prevent grafting of live and is usually used after ATG has failed.
donor lymphocytes. In severely thrombocytopenic 4 Androgens Th ese are benefi cial in some patients
9
(platelet count < 10 × 10 /L) and neutropenic with FA and acquired aplastic anaemia although
9
patients (neutrophils < 0.5 × 10 /L), management is an overall improved survival in acquired aplastic
similar to the supportive care of patients receiving anaemia has not been proven. Danazol, nan-

intensive chemotherapy with reverse barrier isola- drolone , oxymetholone have all been tried but

tion. An antifibrinolytic agent (e.g. tranexamic side - effects are marked including virilization, salt

acid) may be used in patients with severe prolonged retention and liver damage with cholestatic
thrombocytopenia. Oral antifungal agents and oral jaundice or rarely hepatocellular carcinoma. A
antibiotics are used prophylactically in some units response if any occurs is seen as a rise in haemo-
to reduce the incidence of infection. globin level with neutrophils and platelets
unchanged. If there is no response in 4 – 6 months,
Specifi c androgens should be stopped. If there is a

This must be tailored to the severity of the illness as response the drug should be withdrawn
well as the age of the patient and potential sibling gradually.
stem cell donors. Severity is assessed by the reticu- 5 Stem cell transplantation Allogeneic transplan-
locyte, neutrophil and platelet counts and degree of tation offers the chance of permanent cure. For

marrow hypoplasia. Severe cases have a high mortal- aplastic anaemia conditioning is with cyclophos-
ity in the first 6 – 12 months unless they respond to phamide without irradiation but with ciclosporin,

specific therapy. Less severe cases may have an acute which reduces the risks of graft failure and (with
transient course or a chronic course with ultimate methotrexate) of graft - versus - host disease. Th e
recovery, although the platelet count often remains relative role of SCT versus immunosuppressive
subnormal for many years. Relapses, sometimes therapy in individual patients with aplastic
severe and occasionally fatal, may also occur and anaemia is under constant review. In general
rarely the disease transforms into myelodysplasia, terms, SCT is favoured in younger patients with
acute leukaemia or PNH (see Chapter 6 ). severe aplastic anaemia and a human leucocyte

‘

The following specific ’ treatments are used with antigen (HLA) matching sibling donor. Cure
varying success. rates of up to 80% are obtained. However,
non - myeloablative transplants (see p. 301) are
1 Antilymphocyte or antithymocyte globulin used in selected patients over the age of 40 years.

(ALG or ATG) This is prepared in animals (e.g. SCT using unrelated volunteer donors and