TCR alpha/beta and B-cell depleted haploidentical haematopoietic stem cell transplantation in severe aplastic anaemia in children: a case report

Ref ID: 18564

Author:

M. Tumino, M. Pillon, E. Calore, B. Buldini, M.V. Gazzola,
R. Destro, S. Varotto, A. Strano, C. Winzler, G. Basso,
C. Messina

Author address:

Hematology Oncology (Padua, IT)

Full conference title:

Annual Meeting of the EBMT, 38th

Abstract:

Background: Allogeneic haematopoietic stem-cell transplantation (AHSCT) from matched sibling donor (MSD) is the therapy of choice for children with severe aplastic anemia (SAA),
although graft-versus-host disease (GvHD) or graft rejection
remain the major concerns. Children with SAA who lack a MSD
and fail to respond to immunosuppressive (IS) treatment should
be considered for alternative donor such as from manipulated
haploidentical parents. We present a case of child with SAA
complicated with lung aspergillosis, treated with haploidentical HSCT (HHCST) with TCR alpha/beta (TCR-a/b) and B cell
depletion.
Methods: A 4-year-old female with SAA not responded to IS
treatment, presented persistent fever due to lung aspergillosis.
In the absence of MSD and prompt available alternative donor,
she was subjected to HHCST TCRa/b and B depleted cell from
maternal PBSC, using immunomagnetic separation with CliniMACS System (Miltenyi Biotec). Depletion of a/b T cells was 4.5
log. The recovery of CD34+ was 74.6%.The number of infused
CD34+,TCRa/b,TCRgamma/delta(TCRg/d) and CD20+ was
11.6X10(6)/kg, 25,092/Kg, 2.57X10(6)/kg, 38,596/kg respectively. Myeloablative conditioning regimen (MCR) included thiotepa (TT) and cyclophosphamide (Cy). IS prophylaxis consisted
of rabbit antithymocyte globulin (rATG) and ciclosporin (CsA) (1
mg/Kg i.v. from day -6 to day -1). Despite initial engraftment of
absolute neutrophilis count (ANC) and platelets (PLTs) (day +12
and +15 respectively), graft rejection occurred at day +20. After
38 days, a second HHCST TCRa/b and B depleted cell from
paternal PBSC was performed. Depletion of a/b T cells was
3.85 log. The recovery of CD34+ was 88.6%. The number of
infused CD34+, TCRa/b, TCRg/d and CD20+ was 19.3X10(6)/
kg, 94,313/Kg, 5.9X10(6)/kg, 106,000/kg respectively. MCR
included TT, Cy and total nodal irradiation. rATG and CsA (3
mg/Kg i.v. from day -6) were used as IS prophylaxis.
Results: Stable ANC and PLTs engraftment occurred from day
+14, with clinical and radiological improvement. Mild acute
GvHD on the gut occurred at day +30, with complete response
to CsA that is gradually tapering. CMV reactivation was controlled with antiviral therapy. Five months later, our patient is still
in good clinical conditions with complete donor engraftment and
immune reconstitution.
Conclusion: HHCST TCRa/b and B depleted cell is feasible in
patients with SAA, due to quick engraftment, reduction of GvHD
without impairing pathogen-specifi c immunity and prompt availability of the donor.

Abstract Number: P665

Conference Year: 2012

Link to conference website: NULL

New link: NULL

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