[CIS PIDD] [cis-pidd] Treatment question for Bare lymphocyte sydrome

[Kate Sullivan]

I am speaking more from theory than experience but the BLS are notoriously hard to transplant and I believe there are data showing the drop off in efficacy related to age is pretty rapid.  I know some people believe that the transplant only works if donor cells can migrate into the thymus to present antigen for selection.  Older thymi don't seem as supportive.

All that said, a DLI seems like step you can take without burning any bridges.  (Presuming a full match)

Kate Sullivan
Sent from my iPhone

On Nov 7, 2013, at 4:47 PM, "Verbsky, James" <jverbsky at mcw.edu> wrote:


> A question regarding treatment option for Bare lymphocyte syndrome

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> We have a 27yo diagnosed with BLS and transplanted with an 8/10 unrelated donor at 10 months of age.  Did well and lost to follow up for about 20 years, then presented with esophageal and vaginal candidiasis, diffuse molluscum, and diffuse warts (2 years ago).  Donor chimerism low, so a repeat BMT with the same donor was done(non T cell depleted).  Tolerated well, with some improvement in warts and molluscum for about 6 months, now worsening again.

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> CD4 counts have ranged from 44-190, never above 200.  No clear bump in response to second transplant.

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> The question is what to do now.

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> 1) Donor lymphocyte infusion?  Patient is 100% engrafted.  She clearly needs some mature CD4 T cells, but at the risk of GVH

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> 2) Any cytokine therapies? (IL2, IL7??)

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> 3) Another transplant with new donor? (past donor is now 70 years old).  Not likely to fix her, but perhaps she ill get a few more mature T cells in.  Concerned about a third transplant that is unlikely to benefit her however.

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> Thanks for your thoughts

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> James Verbsky MD/PhD

> Associate Professor of Pediatrics and Microbiology and Molecular Genetics

> Medical College of Wisconsin

> Milwaukee, WI  53226

> 

> ________________________________

> From: Markus Seidel [markus.seidel at medunigraz.at]

> Sent: Wednesday, November 06, 2013 2:54 PM

> To: CIS-PIDD

> Subject: [cis-pidd] Evans Syndrome, iatrogenous CVID, granulomatous LIP

> 

> Dear Colleagues,

> what would be your suggestions in the therapeutic dilemma in a young adult who

> has been treated successfully with MMF for 5 years for Evans Syndrome, under that time had a CVID-like B-cell phenotype (<100/µL B cells, absence of csmB cells, IgG2 and IgA-def.), did not require Ig-substitution, no severe infections,

> initially had severe pancytopenia, autoimmune hemolytic anemia,

> who then developed granulomatous lymphocytic interstitial pneumonitis early this year (LIP, either as consequence of iatrogenous CVID or occupational dust exposure; no infectious agent identified)

> and when IS was terminated, had recurrence of severe thrombocytopenia (<10.000/µL), neutropenia (600-900/µL), autoantibodies against neutrophils, platelets, pos. Coombs test, but recovered clinically from LIP, which now appears to be stable in Chest CT.

> his mother suffered from granulomatosis with polyangiitis (GPA) – but no other than blood-cell tissue- or nuclear autoantibodies are detectable in the patient.

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> I fear that rituximab or other systemic IS would exacerbate his LIP, which has been stable for 4 months now under rapamycin (the latter did not improve his thrombocytopenia, though); the B cells recovered to >200/µL and >2% csmB cells after discontinuation of MMF, but are now lower again under rapamycin, DNT cells borderline increased 3-5% of CD3;

> Romiplostim works short time (1-2 weeks), but doesn’t look like a very good long term option;

> Splenectomy?, Danazole?, AZT?

> ...SCT?

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> Thanks for your comments or thoughts,

> Sincerely,

> Markus Seidel

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> 

> Markus G. Seidel, M.D., Assoc.Prof.

> Consultant| Dept.of Pediatric Hematology-Oncology | Univ.Clinics of Pediatric and Adolescent Medicine | Medical University Graz | Auenbruggerpl. 34/2 | A-8036 Graz | Austria | T. 0043 316 385 80215| F. 0043 316 385 13717 | Secr. 0043 316 385 13485 |

> Coordinator of the Working Group for Pediatric Immunology of the Austrian Society of Pediatrics and Adolescent Medicine

> 

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