8 J. E. Vaughn et al.

difference [54]. Treatment-related mortality in both paedi-
atric and adult patients was high, ranging from 16% to
29%, and did not differ between autologous and allo-
geneic HCT recipients [52, 53, 55].
An analysis of American data on HCT for autoimmune
disorders was reported recently by the Center for Interna-
tional Bone Marrow Transplantation Research (CIBMTR),
the Seattle Consortium and two Brazilian centres, which
included two patients with ITP and four with Evans syn-
drome [56]. However, patient characteristics in this study
were reported in conglomerate with other autoimmune
cytopenias or diseases, and results for patients with ITP
or Evans syndrome could not be assessed separately.
Therefore, we reviewed, in addition, data provided to us
by the CIBMTR for this report (Table 1). These patients
had a median follow-up of 41 months. Among the nine
patients, seven received autologous and two allogeneic
HCT, and all experienced platelet count recovery to
>20 9 109/l. Two patients in the autologous group died,
both of intracranial haemorrhage in the immediate peri-
transplant period. The remaining five autologous HCT
recipients and the two allogeneic HCT recipients were
alive at the time of last contact.
Based on the available data, is there a role
for HCT in patients with ITP and Evans
Syndrome?
The answer to this question has remained controversial.
In ITP in particular, the use of HCT has not been readily
implemented because of the availability of multiple alter-
native treatment strategies. However, these more widely
accepted therapies are not without risk. In one study of
150 patients with chronic ITP who underwent splenec-
tomy, 11 died of haemorrhage, and six died of complica-
tions specifically attributed to therapy (three from sepsis,
one from postoperative complications after splenectomy
and two from transfusion-related complications) [57]. This
report antedated the routine use of rituximab and the
approval of the newer TPO receptor agonists. However, as
stated, these agents must be administered continuously to
sustain remissions, once achieved. Also, potential adverse
effects of chronic exposure to TPO receptor agonists may
become more apparent with more extended observations.
Reticulin fibrosis of the marrow may occur, but is reversi-
ble. Patients who require splenectomy or long-term expo-
sure to glucocorticoids, immunosuppressive agents and
rituximab will become profoundly immunocompromised.
Thus, HCT is an attractive consideration, in that ther-
apy is given once with the ultimate goal of curing the
patient and eliminating the need for any additional or
prolonged therapy. Retrospective reviews of results in
patients treated with either autologous or allogeneic HCT
suggest a sustained response rate of approximately 50%
[52–54], higher with the allogeneic than with the autolo-
gous approach. We are still learning how to support
autoimmune patients through HCT, as evidenced by the
higher rates of treatment-related morbidity and mortality
in these patients compared to the general HCT population
[36]. There are several particular risks when dealing with

Table 1 Summary of HCT characteristics and response in nine patients with ITP transplanted with allogeneic or autologous cellsa

Time from Time from HCT

diagnosis Age Follow-Up Platelet platelet recovery
Patient to HCT (years) (years) Sexb Conditioning regimenb Status (years) recovery >20 3 109/l (days)

1 Unk 23 M TLI + Melphalan Death from 0
14 No Not reported

haemorrhage
2 Unk 23 M CY Alive 3
67 Not reported Not reported
3 20 31 F CY Alive 7
63 Yes 11
4 Unk 19 M AraC + Melphalan Death from Death prior Not applicable Not applicable
haemorrhage to stem
cell infusion
5 Unk 23 F Unknown Alive 3
29 Not reported Not reported
6 9 40 M CY + ATG Alive 3
68 Yes 11
7 9 46 F CY Alive 2
05 No Not applicable
8c 4 20 M TBI (550 cGy) + CY Alive 0
96 Yes 8
9c 1 15 M CY + BU + ATG Alive 4
58 Yes 38

a
Data provided by CIBMTR.
b
TLI, total lymphocyte irradiation; CY, cyclophosphamide; AraC, cytosine Arabinoside; ATG, anti-thymocyte globulin; TBI, total body irradiation; BU, busul-
fan; M, male; F, female.
c
These patients received allogeneic transplants. Patient 8 received a CD 34 selected graft with the goal of preventing graft-versus-host disease (GVHD).
Patient 9 received an HLA-matched sibling HCT and methotrexate + tacrolimus as GVHD prophylaxis. Neither patient developed GVHD.

© 2015 International Society of Blood Transfusion

Vox Sanguinis (2016) 110, 5–11