HLH researchers explore new drugs, gene therapy

Calming a Storm in the Immune System

HLH researchers explore new drugs, gene therapy

Although science has come a long way in understanding HLH, its treatment presents significant challenges. Each step in treatment – getting the raging immune response under control, then replacing the immune system with bone marrow transplantation – wages its own set of assaults on young bodies. Cincinnati Children’s physician-researchers are working to find more effective, less toxic alternatives.

Combination Therapy

One approach is a multicenter clinical trial led by Michael Jordan, MD, of theDivision of Bone Marrow Transplantation and Immune Deficiency. The trial combines two currently used approaches into a unique treatment, “hybrid immunotherapy.” It is designed to improve the first step of treating HLH – quieting the inflammatory response. The Hybrid Immunotherapy for HLH (HIT-HLH) trial is the first-ever U.S.-based trial for the condition and the only one funded by the NIH. It involves 10 centers throughout the U.S. and Canada.

To read the full story click on the link: http://www.cincinnatichildrens.org/professional/resources/research-horizons/archives/2013/winter/calming-storm/

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2nd BMT – DAY +51

Our little warrior is just starting to wake up this morning. He wasn’t wanting to go to sleep last night. He was wanting to tell mommy big stories. He is doing good. Still trying to get him to start eating again. He gets his feeds at night through his NG tube, plus he is getting IV fluids with magnesium for 8 hrs every night. It was really hard for him to try and eat during and after transplant. All the medicines he received for treatment changed they way everything tastes. Very heartbreaking watching him trying to eat, just tasting food he normally liked before transplant would make him shake and gag. But now it is getting better every day. Looks like another beautiful day here in Seattle, hope everyone has a great day. Liam is awake and very busy, just now finishing this post an hour later 🙂

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A great story – Superhero’s, I friend shared this with me. Thanks, Sue

KPTV - FOX 12

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2nd BMT – DAY+ 43

Liam waiting for brothers to arrive ♥

Hunter helping me put Liam to bed ♥

Unspeakable bond between brothers ♥

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2nd BMT – DAY+ 42

Good morning, world!! Thank you, God for blessing me with another day 🙂

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2nd BMT – DAY+ 40

There are not words, that can express the love in this picture…

Papa’s little Buddy…

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2nd BMT – DAY+ 39 – Liam’s ready for the AMA Supercross Main Event :)

Liam’s ready for the AMA Supercross Main Event. He loves his new bicycle from Papa. Noa, Hunter, Ryder and Uncle Markie put it together. It looks like a Motorcycle.

 

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Liam was so excited to leave the hospital!!! :)

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2nd BMT – DAY +34

MOMMY: I love Liams energy and love for life 🙂

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2nd BMT – Day +28 Results- Chimerism Analysis

Day +28 – DNA Chimerism Analysis – April 2013

Post Transplant Peripheral Blood

CD3+ t-cells ~ 16% donor Hunter, 42% donor Noa, 42% Liam
CD33+ myeloid cells ~ 0% donor Hunter, 100% donor Noa, 0% Liam
CD56+ NK cells ~ 0% donor Hunter, 48% donor Noa, 52% Liam
CD19+ B-cells ~ 0% donor Hunter, 90% Noa, 10% Liam

Bone Marrow Results – April 2013

100% donor Noa, 0% Liam , 0% donor Hunter


Liam’s Results from his 1st Bone Marrow Transplant 

DAY +28 – DNA Chimerism Analysis – June 2011

Post Transplant Peripheral Blood

(CD3+) T Cell                          59% donor Hunter origin (41% host – Liam)

(CD33+) Myeloid Cells           100% donor Hunter origin (0% host – Liam)

(CD56+) NK Cells                    100% donor Hunter origin (0%  – host – Liam)

(CD19+) B Cells                       100% donor Hunter origin (0% host – Liam)

My sweet baby boy ♥

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