[CIS PIDD] [cis-pidd] conditioning for RMRP SCID

[Nicholas Rider]

Dear Chris,

We have experience with two CHH patients who underwent matched unrelated transplants with reduced intensity conditioning and did not experience clear conditioning-related toxicity.  One child is still living now several years out from BMT, the other died of complications related to autoimmune hemolytic anemia.  As you might expect, those patients were RMRP 70A>G homozygotes.  That said, I think your question is an excellent one given the reported significant risk of hematologic malignancies in CHH.  

I don't think we really know what additional risk a conditioning regimen superimposes upon that baseline risk related to the syndrome.  In your patient with apparent SCID, we certainly don't have enough evidence to suggest that you shouldn't proceed based solely on the biology of the RMRP mutations.  

Sincerely,

Nick

Nicholas L. Rider, D.O.
Texas Children's Hospital
Associate Professor of Pediatrics, Allergy and Immunology
Baylor College of Medicine
Feigin Center, Suite 330
1102 Bates St
Houston TX, 77040

On Mar 19, 2015, at 8:32 PM, Joanne Smart <Joanne.Smart at rch.org.au> wrote:

>  
>  
> Dr Joanne Smart
> BSc. MBBS. PhD. FRACP
> 
> Clinical Allergist & Immunologist
> Department of Allergy and Immunology
> Royal Children's Hospital
> Flemington Rd
> Parkville, VIC 3052
> AUSTRALIA
> 
> Tel: +61 3 9345 5522
> Fax: +61 3 9345 4848
> email: joanne.smart at rch.org.au
> From: John Ziegler <j.ziegler at unsw.edu.au>
> Sent: Thursday, 6 November 2014 4:15 PM
> To: CIS-PIDD
> Subject: RE:[cis-pidd] conditioning for RMRP SCID
>  
> That was VOD attributed to busulphan. Doesn’t suggest unusual sensitivity. VOD was not reported inhttp://www.ncbi.nlm.nih.gov/pubmed/?term=20375313
>  
>  
> From: Christine M. Seroogy, MD [mailto:cmseroogy at pediatrics.wisc.edu] 
> Sent: Thursday, 6 November 2014 3:09 PM
> To: CIS-PIDD
> Subject: [cis-pidd] conditioning for RMRP SCID
>  
>  
>  
> Dear Colleagues,
>  
> We have a new RMRP SCID case  picked up by NBS SCID screening.  We are moving to HSCT with a well-matched cord.  Are any of you aware of increased sensitivity to conditioning in these patients?  The literature does not suggest this, but the recent JAMA publication on NBS SCID screening(
> PMID: 
> 25138334)
> reported  1 of the 2 RMRP SCID infants had complications from conditioning. May simply be age- or conditioning-regimen-related and not the genetic defect.
>  
> Thank you,
>  
> Chris
>  
> 
> Christine M. Seroogy MD,  FAAAAI
> Associate Professor
> University of Wisconsin School of Medicine and Public Health
> Department of Pediatrics
> Division of Allergy, Immunology & Rheumatology
> 1111 Highland Avenue
> 4139 WIMR
> Madison, WI  53705-2275
> phone: 608-263-2652
> fax: 608-263-4226
>  
>  
>  
> 
> 
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